topic
stringlengths 245
1.29k
| doc
stringlengths 52
16.9k
| label
stringclasses 3
values |
|---|---|---|
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 15.0-999.0, Tuberculosis, Pulmonary Adults (≥16 years on 1st March 2009) Males and Nonpregnant females Active Pulmonary Tuberculosis diagnosed by Sputum Smear positivity for Acid fast bacilli (AFB) Diagnosis within one week of into study Not already on antituberculous treatment Not receiving vitamin D replacement or supplementation History of having been treated with antimycobacterial therapy for < 6 months or with < 4 first-line anti-tuberculous drugs Extra pulmonary TB Immune suppressed; with HIV infection, hepatic, renal failure, malignancy, diabetes mellitus Sarcoidosis, hyperparathyroidism Already on or requiring corticosteroids, immunosuppressive agents, thiazide diuretics Breast feeding or pregnant Symptomatic cardiac disease Seriously ill or moribund patients with advanced respiratory impairment (cor pulmonale, hypercapnia, respiratory acidosis, congestive cardiac failure) Allergy/sensitivity to study drugs or their formulations | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 2.0-21.0, Unspecified Childhood Solid Tumor, Protocol Specific Patients must have had histologic verification of malignancy at original diagnosis or relapse except in patients with intrinsic brain stem tumors, patients with optic pathway gliomas, and patients with pineal tumors and elevations of serum or cerebrospinal fluid (CSF) alpha-fetoprotein or beta-human chorionic gonadotropin (HCG) Patients must have either measurable or evaluable disease Patient's current disease state must be one for which there is no known curative therapy or therapy proven to prolong survival with an acceptable quality of life Karnofsky >= 50% for patients > 16 years of age and Lansky >= 50 for patients =< 16 years of age; Note: neurologic deficits in patients with central nervous system (CNS) tumors must have been relatively stable for a minimum of 1 week prior to study enrollment; patients who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score Patients must have fully recovered from the acute toxic effects of all prior anti-cancer chemotherapy Myelosuppressive chemotherapy: patients must not have received myelosuppressive therapy within 3 weeks of enrollment onto this study (6 weeks if prior nitrosourea) Hematopoietic growth factors: at least 14 days after the last dose of a long-acting growth factor (e.g. Neulasta) or 7 days for short-acting growth factor; for agents that have known adverse events occurring beyond 7 days after administration, this period must be extended beyond the time during which adverse events are known to occur; the duration of this interval must be discussed with the study chair Biologic (anti-neoplastic agent): at least 7 days after the last of a biologic agent that is not a monoclonal antibody and enrollment on this study; for agents that have known adverse events occurring beyond 7 days after administration, this period must be extended beyond the time during which adverse events are known to occur; the duration of this interval must be discussed with the study chair Immunotherapy: at least 6 weeks since the completion of any type of immunotherapy, e.g. tumor vaccines Monoclonal antibodies: at least 3 half-lives must have elapsed after treatment with a monoclonal antibody and enrollment on this study Pregnant or breast-feeding women will not be entered on this study; pregnancy tests must be obtained in girls who are post-menarchal; males or females of reproductive potential may not participate unless they have agreed to use an effective contraceptive method Patients receiving chronic systemic corticosteroids are not eligible; patients must have been off systemic corticosteroids for 7 days prior to enrollment Patients who are currently receiving another investigational drug are not eligible Patients who are currently receiving other anti-cancer agents are not eligible Patients who are currently receiving enzyme inducing anticonvulsants are not eligible Patients must not be receiving any of the following potent Cytochrome P450 family 3, subfamily A, polypeptide 4 (CYP3A4) inducers or inhibitors: erythromycin, clarithromycin, ketoconazole, azithromycin, itraconazole, grapefruit juice or St. John's worth Patients who are currently receiving therapeutic anticoagulants (including aspirin, low molecular weight heparin, and others) are not eligible Patients who are currently receiving angiotensin-converting enzyme (ACE) inhibitors are not eligible Patients who are receiving cyclosporine, tacrolimus or other agents to prevent either graft-versus-host disease post bone marrow transplant or organ rejection post-transplant are not eligible for this trial Patients who have an uncontrolled infection are not eligible | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 15.0-65.0, Acute Myeloid Leukemia Patients with CBF positive AML in CR1. CBF AML includes t(8;21)(q22;q22) [AML1(RUNX1)/ETO(CBFα2T1)], inv(16)(q13q22) (CBFβ/MYH11),t(16;16)(p13;q22) (CBFβ/MYH11) Using RT-PCR, FISH, or standard karyotype analysis technique Patients who plan to receive the second cycle of HDAC consolidation chemotherapy years old or older and 65 years or younger Adequate performance status (Karnofsky score of 70 or more) Adequate hepatic and renal function (AST, ALT, and bilirubin < 3.0 x upper normal limit, and creatinine < 2.0 mg/dL) Adequate cardiac function (left ventricular ejection fraction over 40% on heart scan or echocardiography) Signed and dated informed consent must be obtained from patient Presence of significant active infection Presence of uncontrolled bleeding Any coexisting major illness or organ failure Patients with psychiatric disorder or mental deficiency severe as to make compliance with the treatment unlike, and making informed consent impossible Nursing women, pregnant women, women of childbearing potential who do not want adequate contraception Patients with a diagnosis of prior malignancy unless disease-free for at least 5 years following therapy with curative intent (except curatively treated nonmelanoma skin cancer, in situ carcinoma, or cervical intraepithelial neoplasia) | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 18.0-80.0, Depression, Myalgia Capable of providing informed consent American Society Anesthesiologists (ASA) rating I-III Subjects age 18-80 capable of providing consent Subjects undergoing electroconvulsive therapy using succinylcholine as the sole neuromuscular blocking agent Subjects who have scored ≥23 on the Mini-Mental State Examination Subjects who have had a recent thrombotic event, myocardial infarction or stroke or episode of Congestive Heart Failure (CHF) within less than 3 months Subjects who have had a recent cardiovascular surgery within the last 3 months Subjects with active Gastrointestinal bleeding Subjects who have asthma, itching or allergic type reaction following aspirin or other NSAID administration Subjects with a known hypersensitivity to ibuprofen Subjects with heart failure, bleeding disorders or kidney failure Subjects taking aspirin, Angiotensin converting enzyme (ACE) inhibitors, or anticoagulants within one month Subjects with any devices used to treat pain (intrathecal pumps, spinal cord stimulators etc) Subjects with a history of fibromyalgia or chronic myositis Subjects who are pregnant | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 18.0-64.0, Tuberculosis, Pulmonary Provide signed written informed consent for study participation, including HIV testing (if HIV serostatus is not known or the last documented negative is more than four weeks prior to enrolment). 2. Be eighteen (18) to 64 (inclusive) years of age. 3. Have a body weight (in light clothing and with no shoes) between 40 and 90 kg, inclusive. 4. Have newly diagnosed, previously untreated, uncomplicated, sputum smear-positive, pulmonary TB. 5. Have a chest X-ray which, in the opinion of the Investigator, is compatible with TB. 6. Is sputum positive on direct microscopy for acid-fast bacilli (at least 1+ on the scale (Appendix 3). 7. Is able to produce an adequate spot sputum sample, indicating an overnight sputum volume of at least 10 mL. 8. Female patients of childbearing potential must have a negative serum pregnancy test, and consent to practice two effective methods of birth control when not abstaining from sexual intercourse, unless she and her partner(s) are surgically sterile or she is post-menopausal with no menses for the last 12 months. Preferably, contraceptive measures should be continued until completion of TB treatment, but at least until one month after last dose of IMP, unless she and her partner(s) are sterile (that is, women who have had a bilateral oophorectomy or hysterectomy or have been postmenopausal for at least 12 consecutive months). Two of the following methods may be used, but only one may be hormonal: tubal ligation, vaginal diaphragm, intrauterine device, condom, oral contraceptives, contraceptive implant, combined hormonal patch, combined injectable contraceptive or depot-medroxyprogesterone acetate, partner(s) has had a vasectomy. 9. Male participants must agree to use an adequate method of contraception when not abstaining from sexual intercourse throughout participation in the trial and for 12 weeks after last dose, unless he has had bilateral orchidectomy. 10. A Karnofsky score of at least 60 (requires occasional assistance but is able to care for most of his/her needs, see Appendix 5) Poor general condition where any delay in treatment cannot be tolerated per discretion of Investigator. 2. Treatment with any drug active against MTB within the 3 months prior to Visit 1 (this includes, but is not limited to INH, EMB, RIF, PZA, amikacin, cycloserine, rifabutin, streptomycin, kanamycin, para-aminosalicylic acid, rifapentine, thioacetazone, capreomycin, fluoroquinolone, thioamides, metronidazole). 3. Sputum isolate is resistant to RIF as detected by rapid assay from native sputum 4. A history of allergy to the IMP or related substances. 5. Clinically significant evidence of extrathoracic TB (miliary TB, abdominal TB, urogenital TB, osteoarthritic TB, TB meningitis), as judged by the investigator. 6. A history of previous TB. 7. Evidence of serious lung conditions other than TB or uncontrolled obstructive bronchial disease. 8. Laboratory parameters done at, or within 14 days prior to, screening Serum amino aspartate transferase (AST) and/or serum alanine aminotransferase (ALT) activity >3 times the upper limit of normal Serum total bilirubin level >2.5 times the upper limit of normal Serum creatinine level >2 times the upper limit of normal Complete blood count with hemoglobin level <7.0 g/dL Platelet count <50,000/mm3 Serum potassium <3.5 meq/L 9. History, presence, or evidence of a neuropathy or epilepsy. 10. Clinically relevant change s in the ECG such as atrioventricular (AV) block, prolongation of the QRS complex over 120 milliseconds, or of either the QTcF or QTcB interval over 450 milliseconds on the screening ECG. 11. A history of, or current clinically relevant cardiovascular disorder such as myocardial infarction, heart failure, coronary heart disease, hypertension, arrhythmia, or tachyarrhythmia. Family history of sudden death of unknown or cardiac-related cause, or of prolonged QTc interval. Concomitant use of any drug known to prolong QTc interval (including amiodarone, bepridil chloroquine, chlorpromazine, cisapride, cisapride, clarithromycin, disopyramide dofetilide, domperidone, droperidol, erythromycin, halofantrine, haloperidol, ibutilide, levomethadyl, lumefantrine, mesoridazine, methadone, pentamidine, pimozide, procainamide, quinidine, sotalol, sparfloxacin, terfenadine, thioridazine). 12. Diabetics using insulin. 13. Evidence of clinically significant metabolic, gastrointestinal, neurological, psychiatric or endocrine diseases, malignancy, or other abnormalities (other than the indication being studied). 14. Any disease or condition in which the use of the standard TB drugs or any of their components is contraindicated, including but not limited to allergy to any TB drug, their components or to the IMPs. 15. Any disease or condition in which any of the medicinal products listed in the section pertaining to prohibited medication (see 4.10.4) is used. 16. Known or suspected, current or history of within the past 2 years, alcohol or drug abuse, that is, in the opinion of the investigator, sufficient to compromise the safety or cooperation of the patient. Opiates prescribed for cough relief are not counted as drug abuse. 17. Prior administration of SQ109. 18. Is pregnant, breast-feeding, or planning to conceive or father a child within one month of cessation of treatment. 19. Use of any drugs or substances within 30 days prior to dosing known to be strong inhibitors or inducers of cytochrome P450 enzymes (including xenobiotics, quinidine, tyramine, ketoconazole, testosterone, quinine, gestodene, metyrapone, phenelzine, doxorubicin, troleandomycin, cyclobenzaprine, erythromycin, cocaine, furafylline, cimetidine, dextromethorphan). Exceptions may be made for subjects that have received 3 days or less of one of these drugs or substances, if there has been a wash-out period equivalent to at least 5 half-lives of that drug or substance. 20. Use of any therapeutic agents within 30 days prior to dosing known to alter any major organ function (e.g., barbiturates, opiates, phenothiazines, cimetidine). 21. Use of systemic glucocorticoids within three months prior to dosing. 22. HIV infection with helper/inducer T lymphocyte (CD4 cell) count of 250 10-6/L. 23. Receiving antiretroviral therapy (ART) | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 14.0-999.0, Leukemia Must be >14 years of age 2. Must be diagnosed with refractory or relapsed AML or ALL. 3. Must have adequate organ function as demonstrated by the following: o Alanine Aminotransferase (ALT) (SGOT) and/or Aspartate Aminotransferase (AST) (SGPT) equal to or less than 1.5x upper limit of normal o Serum creatinine equal to or less than 2.5 mg/dL 4. Eastern Cooperative Oncology Group (ECOG) performance status (PS) of 0, 1, or 2 5. At least 2 weeks from prior leukemia-directed treatment to starting treatment drug (except for hydroxyurea, which is allowed if clinically indicated but should be stopped after 2 weeks of receiving study drug, and glucocorticoids, which are allowed but should be stopped upon starting treatment drug). 6. Treatment-related toxicities from prior therapies must have resolved to Grade equal to or less than 1 (except for peripheral neuropathy, which should resolve to grade equal to or less than 2) 7. No active malignancies with the exception of basal cell or squamous cell carcinoma of the skin, or carcinoma "in situ" of the cervix or breast. 8. Females of childbearing potential (FCBP)(A female of childbearing potential is a sexually mature woman who: 1) has not undergone a hysterectomy or bilateral oophorectomy; or 2) has not been naturally postmenopausal for at least 24 consecutive months (i.e., has had menses at any time in the preceding 24 consecutive months) must have negative pregnancy test. FCBP and males participating in the study must agree to use a reliable form of contraception or to practice complete abstinence from heterosexual intercourse while participating in the study and for at least 28 days after discontinuation from the study. If pregnancy or a positive pregnancy test does occur in a study subject, treatment with the study drug must be immediately discontinued Known positive status for HIV, or known active hepatitis A, B, or C infection. 2. Any serious medical condition or psychiatric illness that would prevent, (as judged by the treating physician) the subject from signing the informed consent form or any condition, including the presence of laboratory abnormalities, which places the subject at unacceptable risk if he/she were to participate in the study or confounds the ability to interpret data from the study. 3. Pregnant or lactating females. 4. Acute promyelocytic leukemia 5. Concurrent use of strong inducers or strong inhibitors of cytochrome P450 3A4 (CYP3A4). Strong inducers are rifampin and St. John's Worth. Strong inhibitors are HIV-antivirals, clarythromycin, itraconazole, ketoconazole, nefazodone, and telithromycin. 6. Participating in any other research trial | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 18.0-80.0, Myasthenia Gravis Ability to comprehend and willing to sign an Informed Consent Form (ICF) Ability to understand written and oral English language Males and females between 18 and 80 years of age, inclusive Patient's signs and symptoms not better explained by another disease process Established diagnosis of MG defined as clinical evidence of muscle weakness and positive AChR-binding antibody titer (>0.02 nmol/L) Myasthenia Gravis Foundation of America (MGFA) clinical classification II or III Stable MG disease for 4 weeks prior to randomization Ability to refrain from IVIg treatments during the course of the study Ability to refrain from cholinesterase-inhibitors (e.g. pyridostigmine) for 12 hours before each dose Ability to perform all elements of the QMG History of chronic degenerative, psychiatric, or neurologic disorder other than MG that can produce weakness or fatigue Other major chronic or debilitating illnesses within six months prior to study entry Hepatic insufficiency (defined as ALT or AST > 3x ULN, or total bilirubin > 3 mg/dL) Renal insufficiency (defined as serum creatinine > 2.5 mg/dL or receiving dialysis) Other myasthenic syndromes (e.g. Lambert Eaton syndrome; inherited myasthenic syndrome) Female patients who are premenopausal and are: (a) pregnant on the basis of a serum pregnancy test, (b) breast-feeding, or (c) not using an effective method of double barrier (1 hormonal plus 1 barrier method or 2 simultaneous barrier methods) birth control (birth control pills, male condom, female condom, intrauterine device, Norplant, tubal ligation, or other sterilization procedures) Receipt of IVIg or plasmapheresis treatment within 6 weeks prior to the first dose of study drug Changes to immunosuppressive treatments (i.e., prednisone) within 6 weeks prior to the first dose of study drug Rituxan treatment within 3 months prior to study entry Participation in any other investigational study drug or device trial in which receipt of an investigational study drug or device occurred within 30 days prior to dosing | 1 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 40.0-999.0, Myalgia Hypercholesterolemia Hyperlipidemia diagnosis of primary hypercholesterolemia (heterozygous familial and nonfamilial) Fredrickson types IIa or IIb history of statin-associated myalgia, as defined by being unable to tolerate two previous statins due to muscle pain, aches, weakness, or cramping that begins or increases during statin therapy and stops when statin therapy is discontinued. History of statin-associated myalgia will be captured on the historical questionnaire on statin-associated myalgia LDL-C > 110 mg/dL and triglycerides < 500 mg/dL at Prescreening prescreening hemoglobin value of ≥10 g/dL for females and ≥12 g/dL patient agrees to stop all other antihyperlipidemic agents (including but not limited to niacin, probucol, ezetimibe, fibrates and derivatives, bile acid-sequestering agents, other 3-hydroxy-3-methylglutaryl-coenzyme A(HMG-CoA) reductase inhibitors, fish oils, flaxseed oil, and red yeast rice) patient agrees to stop all Coenzyme Q10 supplements if taking other nonexcluded medications, patients must be on a stable dose for 4 weeks before screening history of chronic pain and currently experiences chronic pain unrelated to statins that requires chronic use of pain medications, has been diagnosed with fibromyalgia or has severe neuropathic pain requires the chronic use of pain medications, including acetaminophen, non-steroidal anti-inflammatory medications, narcotics, and other analgesics vitamin D insufficiency (current insufficiency is defined as Vitamin D3 < 20 ng/mL [50 nmol/L] measured at Prescreening hypothyroidism or abnormal thyroid function test as confirmed by thyroid-stimulating hormone ≥ 5 mcIU/mL and free thyroxine (T4) < 0.7 ng/dL at Prescreening history of rhabdomyolysis (defined as evidence of organ damage with creatinine kinase(CK) > 10,000 IU/L) history of liver disease history of significant renal dysfunction as defined by serum creatinine clearance < 30 mL/min Nephrotic-range proteinuria HbA1C >9% at Prescreening CK levels >5 times the upper limit of normal at Prescreening | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 30.0-999.0, Pterygium Bevacizumab VEGF Anti-VEGF Patients who are diagnosed with primary pterygium and plan for pterygium excision with bare sclera by single surgeon. (G.N.) Patients with pterygium who understand and can follow the study protocol Patients of age more than 30 years Patients who have corneal melt, corneal epitheliopathy, abnormal corneal epithelial wound healing Patients who are pregnancy or lactation Patients who have a history of allergy to bevacizumab Patients who have a history of allergy to steroid eye drops | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 18.0-70.0, Myasthenia Gravis diagnosed as myasthenia gravis (MG) by clinical diagnosis QMG scores ≥7 at the time of enrollment basic treatment drugs are mainly glucocorticoids, and the efficacy of glucocorticoid therapy was not sufficient receiving blood purification therapy or immunoglobulin therapy within 8 weeks before the study QMG swallowing function score ≥ 2 points or QMG vital capacity = 3 abnormal hepatic functions uncontrolled diabetes patients hyperkalemia patients immuno-inhibitors are forbidden due to malignancy, history of malignancy or history of HIV infection patients who are allergic to Tacrolimus or macrolide antibiotics receiving other immuno-inhibitors within 12 weeks | 1 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 0.0-999.0, Very Low Birth Weight Baby Pneumonia A cluster was eligible to participate if it Is located in Kintampo North or South Districts (this is the core study area for KHRC) Is primarily rural (in practice, this excludes Kintampo, which is a small city of approximately 40,000 people) Is operationally feasible (in practice, this excluded a handful very small, isolated clusters that would have presented extraordinary logistical challenges) Is home to women who primarily deliver at one of our four staffed birth facilities (in practice this excluded one village on the edge of the study area, in which women travel to another district for deliveries). A woman will be eligible to participate in the study if she Is in the first or second trimester of pregnancy (gestational age ≤ 24 weeks gestation; this is to ensure that the intervention is actually delivered prior to 27 weeks) Is carrying a live singleton fetus (twins will be excluded) Is the primary cook in her household or compound; and Is a non-smoker | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 16.0-999.0, Fracture of Cervical Spine Fracture Dislocation of Cervical Spine The investigators will enroll consecutive alert, stable adults presenting to the study hospital EDs with potential c-spine injury after sustaining acute blunt trauma. These will be patients presenting with neck pain or with c-spine immobilization on an ambulance stretcher. Patient will be determined based on these at the time of arrival in the ED "Potential c-spine injury after sustaining acute blunt trauma" will patients with either: i) neck pain with any mechanism of injury (subjective complaint by the patient of any pain in the posterior aspect of the neck), and/or ii) presentation by ambulance with c-spine immobilization after injury (typically backboard and collar) "Alert" is defined as a Glasgow Coma Scale103 score of 15 (converses, fully oriented, and follows commands) "Stable" refers to normal vital signs as defined by the Revised Trauma Score31 (systolic blood pressure 90 mm Hg or greater and respiratory rate between 10 and 24 breaths per minute) "Acute" refers to injury within the past 48 hours Patients under the age of 16 years Patients who do not satisfy the definition of "potential c-spine injury" as defined above (for example, patients with neither neck pain nor arriving with ambulance c-spine immobilization will be excluded) Patients with Glasgow Coma Scale score less than 15 Patients with unstable vital signs (systolic BP < 90; respiratory rate less than 10 or more than 24) Patients whose injury occurred more than 48 hours previously Patients with penetrating trauma from stabbing or gunshot wound Patients with acute paralysis (paraplegia, quadriplegia) Patients with known vertebral disease (ankylosing spondylitis, rheumatoid arthritis, spinal stenosis, or previous cervical spine surgery) Patients who return for reassessment of the same injury, or Patients referred from another hospital | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 1.0-999.0, Neuroblastoma Patients must have histologically proven neuroblastoma and confirmation of refractory or recurrent disease with histologic confirmation at diagnosis or at the time of recurrence/progression Patients must be age > 12 months and ≤ 21 at initial diagnosis Life expectancy must be more than 3 months If measurable disease, this must be demonstrated by residual abnormal tissue at a primary or metastatic site measuring more than 1 cm in any dimension by standardized imaging (CT or MRI); tumor must be accessible for biopsy. Patients with bone marrow only disease expected to be > 75% are eligible to enroll Current disease state must be one for which there is currently no known curative therapy Lansky or KarnofskyScore must be more than 50 Patients without bone marrow metastases must have an ANC > 750/μl and platelet count > 50,000/μl Adequate liver function must be demonstrated, defined as Total bilirubin ≤ 1.5 x upper limit of normal (ULN) for age AND SGPT (ALT) < 10 x upper limit of normal (ULN) for age Patients who have received any chemotherapy within the last 7 days prior to enrollment and 14 days prior to study treatment start date Patients who have received any radiotherapy within the last 30 days must have another site of disease to follow Patients receiving anti-tumor therapy for their disease or any investigational drug concurrently Patients with serious infection or a life-threatening illness (unrelated to tumor) that is > Grade 2 (NCI CTCAE V4.0), or active, serious infections requiring parenteral antibiotic therapy Patients with any other medical condition, including malabsorption syndromes, mental illness or substance abuse, deemed by the Investigator to be likely to interfere with the interpretation of the results or which would interfere with a patient's ability to sign or the legal guardian's ability to sign the informed consent, and patient's ability to cooperate and participate in the study | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 55.0-85.0, Alzheimer's Disease age 55 to 85 years; 2. should have a mild-to-moderate AD diagnosis (MMSE 10-26 inclusive); 3. should have evidence of elevated cortical amyloid by PET using florbetapir F18 (Amyvid) [i.e. a positive scan], assessed qualitatively according to the Amyvid product label. 4. if on anti-dementia treatment should be on stable treatment for at least 2 months (i.e. cholinesterase inhibitor and/or Memantine or Axona); 5. stable on all other medications for at least 30 days prior to screen; 6. should be fluent in English; 7. should be physically able to participate by medical history, clinical exam and tests; 8. should have a study partner to accompany them to scheduled visits clinically relevant arrhythmias; 2. a resting pulse less than 50; 3. active cancer other than non-melanoma skin cancers; 4. use of another investigatory drug within 2 months of screening; 5. significant stroke or head trauma by history or MRI; 6. contraindication for having a MRI; 7. diagnostic and Statistical Manual of Mental Disorders-IV for a current major psychiatric disorder; 8. sensitivity to yeast or yeast products; 9. impaired kidney function as measured by a Glomerular Filtration Rate less than 60 milliliters/min; 10. preexisting fluid retention, pulmonary infiltrates, or congestive heart failure; 11. history of moderate-to-severe lung disease; 12. history of moderate-to-severe liver disease; 13. pregnant women, or any women who feel they are likely to become pregnant during the study; 14. prisoners | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 0.385-74.0, Gastric Cancer Recurrent gastric cancer histologically confirmed as being adenocarcinoma 2. Age of 20 to 74 years with either gender 3. ECOG Performance Status of 0 to 2 4. Lesions confirmed on imaging within 28 days before registration (not required measurable lesions as defined in version 1.1) 5. Post-gastrectomy adjuvant chemotherapy including S-1 for at least 12 weeks including interruption period 6. Less than 6 months treatment-free interval from completion of adjuvant therapy 7. In case with receiving neoadjuvant chemotherapy, the total dose of CDDP does not exceed 120mg/m2 8. Treatment-naïve recurrent gastric cancer 9. Life expectancy of at least 3 months after registration 10. Written informed consent 11. Adequate major organ functions within 14 days before registration Positive HER2 status 13. Previous treatment with platinum agents after curative surgery 14. Previous history of serious hypersensitivity to fluoropyrimidines or platinum agents 15. Previous history of adverse reactions suggestive of dihydropyrimidine dehydrogenase (DPD) deficiency 16. More than one cancer at the same time or more than one cancer at different times separated by a 5-year disease-free interval. However, multiple active cancers do not carcinoma in situ or skin cancer which is determined to have been cured as a result of treatment. 17. Obvious infection or inflammation (pyrexia ≥ 38.0˚C) 18. Active hepatitis 19. Heart disease that is serious or requires hospitalization, or history of such disease within past year 9) Concurrent illness that is serious or requires hospitalization (intestinal paralysis, intestinal obstruction, interstitial pneumonia or pulmonary fibrosis, poorly controlled diabetes mellitus, renal failure, liver disorders, or hepatic cirrhosis) 10) Being treated or in need of treatment with phenytoin or warfarin potassium 11) Chronic diarrhea (watery stool or ≥ 4 times/day) 12) Active gastrointestinal hemorrhage 13) Body cavity fluids requiring drainage or other treatment 14) Clinical suspicion or previous history of metastases to brain or meninges 15) Women who are pregnant, breastfeeding, or potentially (hoping to become) pregnant 16) Unwillingness to practice contraception 17) Poor oral intake 18) Psychiatric disorders which are being or may need to be treated with psychotropics 19) Otherwise determined by investigators or site principal investigators to be unsuitable for participation in study | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 0.0-45.0, Anemia, Sickle Cell Complex and Transfusion-dependent Hemoglobinopathies Thalassemia Diamond-Blackfan Anemia Bone Marrow Failure Syndromes Alpha-Thalassemia Beta-Thalassemia The following are established to identify subjects with hemoglobinopathies, hematologic or bone marrow failure syndromes who have a high predicted morbidity and are at risk for early mortality Patients with alpha or beta thalassemia major Patients with Diamond-Blackfan anemia and other bone marrow failure syndromes, characterized by severe chronic anemia Patients with other complex and transfusion-dependent hemoglobinopathies, including sickle cell disease Patients with sickle disease who have one or more of the following Overt or silent stroke Neurocognitive impairment Pain crises 2 or more episodes per year for past year One or more episodes of acute chest syndrome Osteonecrosis involving 1 or more joints Patients with cirrhosis, extensive bridging hepatic fibrosis, or active hepatitis are excluded from enrollment Uncontrolled infection or severe concomitant diseases, which in the judgment of the Principal Investigator, indicate that the patient could not tolerate reduced intensity transplantation Severe impairment of functional performance as evidenced by a Karnofsky score <70% (patients ≥16 years old) or Lansky (children <16 years old) score <70% Renal insufficiency (GFR <50 ml/min/1.73 m2) Subjects with a positive human immunodeficiency virus (HIV) antibody test result Subjects who are pregnant, as indicated by a positive serum human chorionic gonadotrophin (HCG) test Subjects whose only donor is pregnant at the time of intended transplant Subjects of childbearing potential who are not practicing adequate contraception as defined by the investigator at the site Allogeneic hematopoietic stem cell transplant within the previous 1 year Subjects must not have had previous radiation therapy that would preclude total body irradiation (TBI) (as determined by a radiation therapist) | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 0.0-21.0, Adenovirus Anesthesia Anxiety Anxiolysis Autism Autistic Disorder Bacterial Meningitis Bacterial Septicemia Benzodiazepine Bipolar Disorder Bone and Joint Infections Central Nervous System Infections Convulsions Cytomegalovirus Retinitis Early-onset Schizophrenia Spectrum Disorders Epilepsy General Anesthesia Gynecologic Infections Herpes Simplex Virus Infantile Hemangioma Infection Inflammation Inflammatory Conditions Intra-abdominal Infections Lower Respiratory Tract Infections Migraines Pain Pneumonia Schizophrenia Sedation Seizures Skeletal Muscle Spasms Skin and Skin-structure Infections Treatment-resistant Schizophrenia Urinary Tract Infections Withdrawal Sepsis Gram-negative Infection Bradycardia Cardiac Arrest Cardiac Arrhythmia Staphylococcal Infections Nosocomial Pneumonia Neuromuscular Blockade Methicillin Resistant Staphylococcus Aureus Endocarditis Neutropenia Headache Fibrinolytic Bleeding Pulmonary Arterial Hypertension CMV Retinitis Hypertension Chronic Kidney Diseases Hyperaldosteronism Hypokalemia Heart Failure Hemophilia Heavy Menstrual Bleeding Insomnia Children (< 21 years of age) who are receiving understudied drugs of interest per standard of care as prescribed by their treating caregiver Failure to obtain consent/assent (as indicated) Known pregnancy as determined via interview or testing if available | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 12.0-45.0, Acne Vulgaris Male or female subjects 12 to 45 years (inclusive) of age in good general health Subjects must have both on the face: A) A minimum of 17 but not more than 60 inflammatory lesions (papules/pustules), including nasal lesions. And B) A minimum of 20 but not more than 150 non-inflammatory lesions (open/closed comedones), including nasal lesions An ISGA score of 2 or greater at baseline Females of childbearing potential and women who are less than 2 years from their last menses must agree to use the contraception The ability and willingness to follow all study procedures and attend all scheduled visits The ability to understand and sign a written informed consent form (Written informed consent must be obtained also from the parent or guardian in case of subject under 20 years of age at the time of given consent) Have any nodule-cystic lesions at baseline Are pregnant or breast-feeding Have a history or presence of regional enteritis, inflammatory bowel disease (e.g., ulcerative colitis, pseudomembranous colitis, chronic diarrhea or antibiotic-associated colitis) or similar symptoms Used any of the following agents on the face within the previous 2 weeks:Topical antibiotics (or systemic antibiotics);Topical anti-acne medications (e.g., BPO, azelaic acid, resorcinol, salicylates);Abradants, facials, or peels containing glycolic or other acids;Masks, washes or soaps containing BPO, sulfacetamide sodium, or salicylic acid;Non-mild facial cleansers (e.g., facial scrub, cleansers containing agents with anti-inflammatory action); Moisturizers that contain retinol, salicylic acid, or α or β-hydroxy acids;Astringents and toner (Subjects are allowed to enroll in this study, if the subject has been on treatment for more than 2 consecutive weeks prior to start of investigational product use) Used the following agents on the face or performed the following procedure within the previous 4 weeks:Topical corticosteroids (Use of inhaled, intra-articular, or intra-lesional steroids other than for facial acne is acceptable);Facial procedure (such as chemical or laser peel, microdermabrasion, blue light treatment, etc.) Used systemic retinoids within the previous 6 months or topical retinoids on the face within the previous 6 weeks Received treatment with estrogens, androgens, or anti-androgenic agents within the previous 12 weeks (Subjects who have been treated with the above agents for more than 12 consecutive weeks prior to start of investigational product are allowed to enrol as long as they do not expect to change dose, drug, or discontinue use during the study) Used any medication that in the opinion of the investigator may affect this clinical study or evaluation of the study Plan to use medications that are reported to exacerbate acne (e.g., mega-doses of certain vitamins, such as vitamin D [>2000 IU/day] and vitamin B12 [>1 mg/day], corticosteroids*, androgens, haloperidol, halogens [e.g., iodide and bromide], lithium, hydantoin, and phenobarbital). *: except the using of topical corticosteroids (e.g., inhaled, intra-articular, or intra-lesional steroids) other than for facial acne | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 30.0-70.0, Diabetes Age ≥ 30 and ≤ 70 years balanced gender ratio (50: 50) BMI 20-25 kg/m² (normal weight subjects) BMI 25-35 kg/m² (obese subjects) acute illness within the last 2 weeks before the examination autoimmune diseases and disorders immune compromised (leukocytes <5000/μl) renal insufficiency (creatinine> 1.5 mg / dl) heart disease, condition after heart attack anemia (Hb <12 g / l, controls at each examination), blood donation within 4 weeks before the examination participation in another study within 2 months before the examination wear a metal or magnetic objects on or in the body claustrophobia use of immunomodulatory drugs (cortisol, antihistamines, aspirin) | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 18.0-999.0, Myasthaenia Gravis years and older, with life expectancy of greater than 1 year MG of class II to IVa inclusive Acetylcholine receptor (AChR) or muscle specific kinase (MuSK) antibody positive Stable dose (defined as no dose changes) not exceeding the maximum doses given in Section 5.6.1 of the following therapy(ies) prior to screening: Cholinesterase inhibitor(pyridostigmine or equivalent) for at least 2 weeks prior to screening and no immunosuppressants; Cholinesterase inhibitor (pyridostigmine or equivalent) for at least 2 weeks prior to screening and/or only one of the following: prednisone (up to 40 mg/day or equivalent) for at least 1 month prior to screening; azathioprine for at least 6 months prior to screening; mycophenolate for at least 6 months prior to screening, or cyclosporine for at least 3 months prior to screening; or Cholinesterase inhibitor (pyridostigmine or equivalent) for at least 2 weeks prior to screening and/or prednisone (up to 20 mg/day or equivalent) for at least 1 month prior to screening and only one of the following: azathioprine for at least 6 months prior to screening, mycophenolate for at least 6 months prior to screening, or cyclosporine for at least 3 months prior to screening Quantitative Myasthenia Gravis (QMG) score of 8 or greater, with at least 4 points derived from signs other than ocular A female subject is eligible to participate if she is: Of non-childbearing potential; Of childbearing potential and NOT pregnant or nursing, has a negative serum pregnancy test at screening, and agrees to one of the following: Complete abstinence from penile-vaginal intercourse, when this is the female's preferred and usual lifestyle, for the period from consent into the study until 16 weeks after the last dose of investigational product; or Consistent and correct use of one of the following acceptable methods of birth control for the period from consent into the study until 16 weeks after the last dose of investigational product: Oral contraceptives (either combined or progesterone only), Injectable progesterone, Implants of etonogestrel or levonorgestrel, Estrogenic vaginal ring, Percutaneous contraceptive patches, Intrauterine device (IUD) or intrauterine system (IUS) with a documented failure rate of less than 1% per year, Male partner sterilization (vasectomy with documentation of azoospermia) prior to the female subject's entry into the study; this male must be the sole partner for the subject, Double barrier method: condom and an occlusive cap (diaphragm or cervical/vault caps) with a vaginal spermicidal agent (foam/gel/film/cream/suppository) Capable of giving written informed consent, which includes compliance with the requirements and restrictions listed in the consent form Single QTc less than 450 msec; or QTc less than 480 msec in subjects with Bundle Branch Block AST and ALT less than 2xULN; alkaline phosphatase and bilirubin less or = to 1.5xULN (isolated bilirubin greater than 1.5xULN is acceptable if bilirubin is fractionated and direct bilirubin less than 35%) Participated in a clinical trial and has received an investigational product within 30 days, 5 half-lives or twice the duration of the biological effect of the investigational product (whichever is longer) prior to screening or planning to take any investigational drug for the planned duration of study participation (6 months after the last dose of study drug) Presence or previous history of thymoma Thymectomy within 12 months Clinically significant (in the opinion of investigator) abnormal laboratory values Pregnant females as determined by positive (serum) hCG test at screening or prior to dosing, or lactating females or planning to become pregnant within 16 weeks after last dose of investigational product History of sensitivity to any of the study medications, or components thereof or a history of drug or other allergy that, in the opinion of the investigator or GSK Medical Monitor, contraindicates their participation May require (in the opinion of investigator) treatment with IVIg and/or plasmapheresis during the 24 week treatment period Have received IVIg and/or plasmapheresis within 90 days prior to screening Have received any other biopharmaceutical agent (except IVIg as described in #8) in the 364 days prior to screening Have received treatment with any B cell targeted therapy (e.g., rituximab, belimumab), at any time | 1 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 0.0-999.0, Nervousness Life Experiences All Danish-speaking expected gynaecological cancer patients, who are offered open surgery at Odense University Hospital, who can speak for themselves and have the opportunity to have close relative to follow before anaesthesia and who accept participation Senility | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 12.0-20.0, HPV16 Anitbody Levels Post Vaccination HPV18 Antibody Levels Post Vaccination The following will be reviewed at the time of enrollment, and where noted, confirmed at the clinic visit, immediately prior to blood draw Subject is aged 12-17 years as of August 31, 2011 Subject received at least one dose of HPV vaccine, Cervarix (GSK, UK), from October 1, 2008 through October 31, 2009 as a part of the PATH HPV vaccine demonstration project in Nakasongola, Uganda Signed and dated informed written consent form (with both parent and subject s signatures) received (confirmed at clinic visit) Subject is afebrile, in good apparent health (confirmed at clinic visit) Subject assents to participate (confirmed at clinic visit) Subject is able to comply with the study protocol (confirmed at clinic visit) Subject plans to stay in Nakasongola for the duration of enrolment, which is expected to be about week between signed consent and blood draw The following will be reviewed at the time of enrollment, and where noted, confirmed at the clinic visit Prior HPV vaccination outside the PATH HPV vaccine demonstration project period Subject is known to be pregnant or lactating at the time of the scheduled blood draw (confirmed at clinic visit) Subject has an apparent moderate or severe acute illness or has fever (confirmed at clinic visit) Clinical history of bleeding disorders such as haemophilia, thrombocytopenia, or anticoagulant therapy Investigational drug or investigational vaccine or licensed vaccine administered during the period from 30 days before the date of the scheduled blood draw (confirmed at clinic visit) Subject receives immunoglobulins and/or any blood products during the period from 30 days before the date of the scheduled blood draw (confirmed at clinic visit) Subject or subject s parents refused to sign written consent Subject does not assent at the time of the blood draw | 1 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 18.0-999.0, Intrinsic Aging of Skin Solar Elastosis Male or female patients who meet all of the following are eligible for this study Eighteen years of age or older Moderate to severe lateral orbital rhytids at maximum smile (score of [2] or [3] by physician assessment) Negative pregnancy test for females of childbearing potential Time and ability to complete the study and comply with instructions Understanding of the study and contents of the informed consent Patients who meet any of the following are not eligible for this study Previous treatment to the glabellar area, forehead or lateral orbital rhytids with Dysport® or Botox® Cosmetic or other botulinum toxin within 180 days of entry into the study. Botulinum toxin treatment of areas other than the lateral orbital rhytids at any time during the study Patients with an ongoing treatment-related AE from any Dysport® or Botox® Cosmetic or botulinum toxin study Inability to substantially lessen lateral orbital lines by physically spreading them apart Permanent or semi-permanent dermal fillers in the lateral orbital rhytids at any time Ablative skin resurfacing on the lateral orbital rhytids at any time preceding the study or planning to during the current study Upper eyelid blepharoplasty or brow lift at any time preceding the study or planning to during the current study Non-ablative treatments in the lateral orbital region for skin dyschromias (e.g. Intense Pulsed Light, light-emitting diodes) at any time during the current study Non-ablative dermal treatment in the lateral orbital area for skin tightening (e.g. radiofrequency treatments at any time preceding the current study or planned to have this done during the current study Retinoid, microdermabrasion, or prescription-level glycolic acid treatments to the lateral orbital area within 2 weeks prior to study participation or during the current study | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 3.0-75.0, Accelerated Phase Chronic Myelogenous Leukemia Adult Acute Lymphoblastic Leukemia in Remission Adult Acute Myeloid Leukemia in Remission Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities Adult Acute Myeloid Leukemia With Del(5q) Adult Acute Myeloid Leukemia With Inv(16)(p13;q22) Adult Acute Myeloid Leukemia With t(15;17)(q22;q12) Adult Acute Myeloid Leukemia With t(16;16)(p13;q22) Adult Acute Myeloid Leukemia With t(8;21)(q22;q22) Adult Grade III Lymphomatoid Granulomatosis Adult Nasal Type Extranodal NK/T-cell Lymphoma Anaplastic Large Cell Lymphoma Angioimmunoblastic T-cell Lymphoma Aplastic Anemia Burkitt Lymphoma Childhood Acute Lymphoblastic Leukemia in Remission Childhood Acute Myeloid Leukemia in Remission Childhood Chronic Myelogenous Leukemia Childhood Diffuse Large Cell Lymphoma Childhood Grade III Lymphomatoid Granulomatosis Childhood Immunoblastic Large Cell Lymphoma Childhood Myelodysplastic Syndromes Childhood Nasal Type Extranodal NK/T-cell Lymphoma Chronic Myelomonocytic Leukemia Chronic Phase Chronic Myelogenous Leukemia Congenital Amegakaryocytic Thrombocytopenia Diamond-Blackfan Anemia Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue Hepatosplenic T-cell Lymphoma Juvenile Myelomonocytic Leukemia Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable Nodal Marginal Zone B-cell Lymphoma Paroxysmal Nocturnal Hemoglobinuria Peripheral T-cell Lymphoma Polycythemia Vera Post-transplant Lymphoproliferative Disorder Previously Treated Myelodysplastic Syndromes Primary Myelofibrosis Recurrent Adult Acute Lymphoblastic Leukemia Recurrent Adult Acute Myeloid Leukemia Recurrent Adult Burkitt Lymphoma Recurrent Adult Diffuse Large Cell Lymphoma Recurrent Adult Diffuse Mixed Cell Lymphoma Recurrent Adult Diffuse Small Cleaved Cell Lymphoma Recurrent Adult Grade III Lymphomatoid Granulomatosis Recurrent Adult Hodgkin Lymphoma Recurrent Adult Immunoblastic Large Cell Lymphoma Recurrent Adult Lymphoblastic Lymphoma Recurrent Adult T-cell Leukemia/Lymphoma Recurrent Childhood Acute Lymphoblastic Leukemia Recurrent Childhood Acute Myeloid Leukemia Recurrent Childhood Anaplastic Large Cell Lymphoma Recurrent Childhood Grade III Lymphomatoid Granulomatosis Recurrent Childhood Large Cell Lymphoma Recurrent Childhood Lymphoblastic Lymphoma Recurrent Childhood Small Noncleaved Cell Lymphoma Recurrent Cutaneous T-cell Non-Hodgkin Lymphoma Recurrent Grade 1 Follicular Lymphoma Recurrent Grade 2 Follicular Lymphoma Recurrent Grade 3 Follicular Lymphoma Recurrent Mantle Cell Lymphoma Recurrent Marginal Zone Lymphoma Recurrent Mycosis Fungoides/Sezary Syndrome Recurrent Small Lymphocytic Lymphoma Recurrent/Refractory Childhood Hodgkin Lymphoma Refractory Chronic Lymphocytic Leukemia Refractory Hairy Cell Leukemia Refractory Multiple Myeloma Secondary Acute Myeloid Leukemia Secondary Myelodysplastic Syndromes Secondary Myelofibrosis Severe Combined Immunodeficiency Severe Congenital Neutropenia Shwachman-Diamond Syndrome Splenic Marginal Zone Lymphoma T-cell Large Granular Lymphocyte Leukemia Waldenstrom Macroglobulinemia Wiskott-Aldrich Syndrome Diagnosis of a histology documented hematologic malignancy or marrow disorder BONE Acquired bone marrow failure disorders aplastic anemia, paroxysmal nocturnal hemoglobinuria (PNH) * Primary allogeneic HSCT is appropriate for selected patients with severe aplastic anemia; however, patients with aplastic anemia must have failed at least one cycle of standard immunosuppressive therapy with calcineurin inhibitor plus anti-thymocyte globulin (ATG) if a fully matched donor is available * Patients with PNH should not be eligible for a myeloablative HSCT Hereditary bone marrow failure disorders Diamond-Blackfan Anemia, Shwachman-Diamond Syndrome, Kostmann Syndrome, congenital Amegakaryocytic Thrombocytopenia; Fanconi Anemia or related chromosomal breakage syndrome, Dyskeratosis Congenital are excluded from this study die to their poor deoxyribonucleic acid (DNA) repair capacity * Fanconi anemia or related chromosomal breakage syndrome: positive chromosome breakage analysis using diepoxybutane (DEB) or mitomycin C if applicable * Dyskeratosis Congenita: diagnosis is supported by using either telomerase RNA component (TERC) gene mutation in autosomal dominant Dyskeratosis Congenita or X-linked DKC1 gene mutation Other non-malignant hematologic or immunologic disorders that require transplantation * Quantitative or qualitative congenital platelet disorders (including but not limited to congenital amegakaryocytopenia, absent-radii syndrome, Glanzmann's thrombasthenia) * Quantitative or qualitative congenital neutrophil disorders (including but not limited to chronic granulomatous disease, congenital neutropenia) *Congenital primary immunodeficiency syndrome, Wiskott-Aldrich syndrome, CD40 ligand deficiency, T-cell deficiencies) ACUTE Subjects must be ineligible for or unable to receive a conventional myeloablative transplantation Resistant or recurrent disease after at least one standard combination chemotherapy OR first remission patients at high risk of relapse * Acute myeloid leukemia (AML) antecedent myelodysplastic syndrome, secondary AML, high risk cytogenetic abnormalities or normal cytogenetics with high-risk molecular mutations (e.g., fms-like tyrosine kinase3-internal tandem duplication [Flt3-ITD] mutation) * Acute lymphocytic leukemia (ALL) high or standard risk ALL (CML) Chronic phase (intolerant or unresponsive to imatinib and/or other tyrosine kinase inhibitors), second chronic phase or accelerated phase who are ineligible for conventional myeloablative transplantation AND (MDS) Myelofibrosis (with/without splenectomy) with intermediate to high risk features Uncontrolled central nervous system (CNS) disease (for hematologic malignancies) Karnofsky (adult) or Lansky (for =< 16 years) performance status < 50% Diffusing capacity of the lung for carbon monoxide (DLCO) < 40% predicted, corrected for hemoglobin and/or alveolar ventilation Left ventricular ejection fraction < 40% Bilirubin >= 3 X upper limit of normal Liver alkaline phosphatase >= 3 x upper limit of normal Serum glutamic oxaloacetic transaminase (SGOT) or serum glutamic pyruvate transaminase (SGPT) >= 3 x upper limit of normal Child's class B and C liver failure Calculated creatinine clearance < 40 cc/min by the modified Cockroft-Gault formula for adults or the Schwartz formula for pediatrics Patients who have received maximally allowed doses (given in 2 Gy fractionations, or equivalent) of previous radiation therapy to various organs as follows: * Mediastinum: adult -40, pediatric (=<18 yrs) | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 18.0-80.0, Myasthenia Gravis Must be between 18 and 80 years of age Established diagnosis of myasthenia based on: the presence of fatigable weakness of ocular, oropharyngeal, and/or limb muscles AND the presence of abnormal acetylcholine receptor binding antibodies ≥ 0.4 nmol/l Patients of childbearing potential must agree to use a medically acceptable form of contraception defined by consistent use of oral contraceptive medications or history of tubal ligation or men who are in sexual relationship with such women during and for at least 8 weeks following completion of the study Patient or designee must have the ability to self-inject investigational product If thymectomized, the procedure must have been performed at least one year prior to screening Dose of current anticholinesterase drugs must be constant for 2 weeks prior to screening If taking prednisone, dose must be stable for ≥4 weeks prior to screening exclusively ocular MG (MGFA Class I) severe respiratory and/ or swallowing muscle weakness (MGFA Class Vb or V) presence of thymoma Must not have received plasm exchange or IVIG within 4 weeks of screening Must not have received immuno-modulating agents within the 4 weeks of screening, including Azathioprine (Imuran), Cyclosporine (Sandimmune, Neoral), Mycophenolate mofetil (CellCept), GM-CSF (Filgrastim; Neupogen; pegfilgrastim, sargramostim), or any other chronic immunosuppressive agent History of tuberculosis or evidence of latent tuberculosis (positive PPD skin test or a chest X-ray with evidence of tuberculosis) vital capacity of less than 1.2 liters or on supplemental oxygen therapy severe comorbidities including lung disease, stroke, congestive heart failure of any severity, myocardial infarction, EKG abnormalities, uncontrolled hypertension - (sitting systolic BP <80 or > 160 mm Hg or diastolic BP > 100 mm Hg, unstable angina pectoris, hepatic or renal disease, insulin-dependent diabetes mellitus, history of cancer (other than in-situ cervical cancer or resected, cutaneous basal cell or squamous cell carcinoma), open cutaneous ulcers, known hepatitis B surface antigen (HbsAg) or hepatitis C virus (HCV) positive, or any other concurrent medical condition, which would make it unsafe for subjects to participate in the trial or interfere with the interpretation of the results Laboratories values which, at the time of the screening visit or at any time during the study that in the opinion of the Investigator would preclude participation in the study including: serum creatinine > 2.5 mg/dL, serum potassium < 3.5 mmol/L or > 5.5 mmol/L, serum aspartate transaminase (AST), alanine transaminase (ALT), or alkaline phosphatase (ALP)> 3 times the upper limit of normal, platelet count < 100,000/mm3, WBC count < 3,000 cells/mm3, Hemoglobin, hematocrit, or red blood cell count outside 30% of the upper or lower limits of normal Receipt of a live vaccine within 3 months of screening | 1 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 0.5-65.0, Neuromuscular Disease Confirmed diagnosis of neuromuscular disease Clinical evidence of impaired pulmonary function Above or below age range No clinical evidence of impaired pulmonary function No diagnosis of a neuromusuclar disease Presence of an acute illness at time of study Participating in other research studies involving investigational drugs Diagnosis of a primary pulmonary disease Use of tobacco products | 1 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 18.0-999.0, Skin Aging Elastosis Senilis Male or female patients who meet all of the following are eligible for this study Eighteen years of age or older Moderate to severe vertical glabellar lines at maximum frown (score of [2] or [3] by physician assessment) Negative pregnancy test for females of childbearing potential Time and ability to complete the study and comply with instructions Understanding of the study and contents of the informed consent Patients who meet any of the following are not eligible for this study Previous treatment to the glabellar area with Dysport® or Botox® Cosmetic or other botulinum toxin within 90 days of entry into the study. Botulinum toxin treatment of areas other than the glabellar area at any time during the study Patients with an ongoing treatment-related AE from any Dysport® or Botox® Cosmetic or botulinum toxin study Inability to substantially lessen glabellar lines by physically spreading them apart Soft tissue augmentation of the glabellar area (e.g. collagen-type implants, or hyaluronic acid fillers) at any time during the current study Permanent or semi-permanent dermal fillers in the glabellar area at any time Ablative skin resurfacing on the glabellar area at any time preceding the study or planning to during the current study Upper eyelid blepharoplasty or brow-lift at any time preceding the study or planning to during the current study Non-ablative treatments in the glabellar area for skin dyschromias (e.g. Intense Pulsed Light, light-emitting diodes) at any time during the current study Non-ablative dermal treatment in the glabellar area for skin tightening (e.g. radiofrequency treatments at any time preceding the current study or plan to have this done during the current study) | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 8.0-999.0, Amblyopia Age ≥8 years 2. Amblyopia associated with strabismus and/or anisometropia 3. Amblyopic eye visual acuity of 20/50 400 4. Sound eye visual acuity of ≥20/25 5. For 8 to 17 year olds, current amblyopia treatment of at least 2 hours of daily patching for at least 4 weeks during the pre-enrollment period with no improvement in best-corrected amblyopic eye visual acuity (<5 letters or 1 logMAR line between 2 consecutive visual acuity measurements at least 4 weeks apart while on current treatment) 6. For ≥18 year olds, history of prior amblyopia treatment with patching 7. Wearing optimal optical correction with stable amblyopic eye visual acuity (<5 letters or 1 logMAR line of improvement during 2 consecutive visual acuity measurements at least 4 weeks apart) 8. Complete eye examination within 6 months prior to enrollment 9. Available for at least 6 months of follow-up, have access to a phone, and willing to be contacted by clinical staff 10. Likely to comply with prescribed treatment and unlikely, if applicable, to continue to improve with 2 hours of daily patching alone Myopia more than -6.00 D spherical equivalent 2. Presence of associated findings that could cause reduced visual acuity 3. Previous intraocular or refractive surgery 4. Strabismus surgery planned within 22 weeks 5. Current vision therapy or orthoptics 6. Treatment with topical atropine within the past 4 weeks 7. Presence of cardiac condition, asthma, obstructive pulmonary disease, seizure disorder, urinary incontinence, and/or peptic ulcer disease receiving concurrent NSAIDs 8. History of gastrointestinal bleeding from peptic ulcer disease 9. Known psychological problems 10. Known skin reaction to patch or bandage adhesives for 8 to 17 year olds 11. Known allergies or contraindications to the use of acetylcholinesterase inhibitors 12. Prior acetylcholinesterase inhibitor treatment 13. Current use of medication for the treatment of ADHD or psychological disorders 14. Inability to swallow pills equivalent in size to the 5 mg donepezil tablet 15. Females who are pregnant, lactating, or intending to become pregnant within the next 6 months | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 0.0-21.0, Neuroblastoma Age: 0-21 years at the time of diagnosis Diagnosis: histologic verification at either the time of original diagnosis or a previous relapse of high risk neuroblastoma Disease Status: Neuroblastoma that is in remission Greater than 30 days from completion of cytotoxic and biologic therapy and less than 120 days from previous therapy A negative urine pregnancy test is required for female subjects of child bearing potential (onset of menses or ≥13 years of age) Both male and female post-pubertal study subjects need to agree to use one of the more effective birth control methods during treatment and for six months after treatment is stopped. These methods total abstinence (no sex), oral contraceptives ("the pill"), an intrauterine device (IUD), levonorgestrol implants (Norplant), or medroxyprogesterone acetate injections (Depo-provera shots). If one of these cannot be used, contraceptive foam with a condom is recommended ANC > 500/μl and platelet count >50,000/μl Organ Function Requirements: Subjects must have adequate liver function as defined by AST and ALT <10x upper limit of normal Serum bilirubin must be ≤ 2.0 mg/dl Lansky score < 60% BSA (m2) of <0.25 Investigational Drugs: Subjects who are currently receiving another investigational drug are excluded from participation Anti-cancer Agents: Subjects who are currently receiving other anticancer agents are not eligible. Subjects must have fully recovered from the effects of prior chemotherapy (hematological and bone marrow suppression effects) Infection: Subjects who have an uncontrolled infection are not eligible until the infection is judged to be well controlled in the opinion of the investigator Subjects who, in the opinion of the investigator, may not be able to comply with the safety monitoring requirements of the study, or in whom compliance is likely to be suboptimal, should be excluded | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 18.0-64.0, HIV Dementia Men and women 18 years of age or older and less than 65 years of age. Able to read and understand English. HIV infected on stable ART regimen for 5 months or greater, and not likely to change regimen for the duration of the study. Significant dementia but able to give consent (International HIV Dementia Scale score <10). Significant cognitive slowing on screening, defined as 1 SD below normal (t-score >60) on the Conner's CPT-II reaction time test. Beck Depression Inventory score <16. Documented HIV-1 RNA PCR <50 copies/mL and documented CD4 count >200 within 3 months of entry visit. Baseline CBC and chemistry panel Grade 1 or normal. For females of reproductive potential (women who have not been post-menopausal for at least 24 consecutive months, i.e., who have had menses within 24 months prior to study entry), or women who have not undergone surgical sterilization, specifically hysterectomy or bilateral oophorectomy or tubal ligation) will require a negative serum or urine pregnancy test within 48 hours prior to entry. NOTE: Subject reported history is considered acceptable documentation of hysterectomy, bilateral oophorectomy, tubal ligation, tubal micro-inserts, menopause, and vasectomy/azoospermia. All subjects must agree not to participate in the conception process (e.g., active attempt to become pregnant or to impregnate, sperm donation, in vitro fertilization), and if participating in sexual activity that could lead to pregnancy, the subject/partner must use at least two reliable methods of contraception, (condoms, without a spermicidal agent; a diaphragm or cervical cap without spermicide; an IUD; or hormone-based contraceptive), for 2 weeks before study treatment, while receiving study treatment, and for 6 weeks after receiving study treatment. Ability and willingness of subject to provide informed consent Inability to give informed consent. No proxy consent allowed. Uncontrolled hypertension (SBP >140 and DBP >90 at screening and baseline). Untreated hypogonadism, hypothyroidism or hyperthyroidism. Initiation of antidepressants, thyroid medication or anabolic steroids within 6 weeks of screening visit. Pregnancy or breast feeding. Clinically significant EKG abnormalities at screening. History of coronary artery disease, atherosclerotic disease, left ventricular hypertrophy, cardiomegaly, syncope, congestive heart failure, myocardial infarction, pacemaker, clinically significant arrhythmia, angina, history of treatment for arrhythmia. Brain related opportunistic infections, stroke, intracranial lesions/disease, or meningitis. History of epilepsy. Untreated depression. Uncontrolled diabetes (glucose <70 or >200 at screening). Use of interferon or ribavirin during study and for 1 month prior to screening. History of bipolar disorder, Alzheimer's dementia, ALS, Parkinson's disease or other medical dementias other than HIV-related. History of schizophrenia, mania or other serious mental illness. History of methylphenidate allergy. Other serious concurrent medical illness other than HIV. History of radiation therapy to the brain or brain injury. History of crystal methamphetamine, cocaine, LSD use or other recreational substance use other than marijuana within the 12 months prior to screening and for the duration of the study. Plan to use marijuana or marinol during the entire study duration and one month prior to screening visit. Plan to drink 7 or more alcoholic drinks per week during the 4 weeks prior to screen visit and for the duration of the study. History of alcohol dependence, alcohol abuse, cocaine addiction, crystal methamphetamine addiction or other recreational drug addiction or treatment for addiction or dependence within 5 years of screening visit. If subject is using prescription narcotics, should be on a stable dose for at least 1 month prior to screening and throughout the study. Previous use of Concerta™, Ritalin™, atomexitine or Adderall™ or other psychostimulants (e.g. modafinil, armodafinil) for 3 months prior to screening visit and for duration of the study. History of tic disorders in the past 3 months or any history of Tourette's syndrome. Greater than 10% below ideal body weight (IBW for men = 50 kg + 2.3 kg per inch over 5 feet of height, and IBW for women = 45.5 kg + 2.3 kg per inch over 5 feet of height) Uncontrolled migraine headaches. History of short gut syndrome, Meckel's diverticulum, or cystic fibrosis. Family history of sudden cardiac death in a young relative. History of fainting with exercise. History of attention deficit disorder will be excluded, defined as a score greater than 6 on the Childhood ADHD Symptoms Scale. - | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 0.0-999.0, Aplastic Anemia de Novo Myelodysplastic Syndrome Myelodysplastic Syndrome Previously Treated Myelodysplastic Syndrome Patients with the diagnosis of MDS (low, int-1 by International Prognostic Scoring System [IPSS], or hypocellular) who are either previously treated or untreated are eligible for this trial Patients with the diagnosis of aplastic anemia who are either previously treated or untreated are eligible if they are not currently candidates for an allogeneic stem cell transplant Patients must have been off of cytotoxic, immunosuppressive (except steroids), or targeted therapy for at least 2 weeks prior to entering this study, and have recovered from the toxic effects of that therapy to grade 1 or less Bilirubin < 2 mg/dL Aspartate aminotransferase (AST) < 3 x upper limit of normal (ULN) Creatinine < 2.5 x ULN Eastern Cooperative Oncology Group (ECOG) performance status of =< 2 Women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation; should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician immediately A negative urine pregnancy test is required within 1 week for all women of childbearing potential prior to enrolling on this trial Patient must have the ability to understand the requirements of the study and signed informed consent; a signed informed consent by the patient or his legally authorized representative is required prior to their enrollment on the protocol Pregnant women are excluded from this study; because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with the study agents, breastfeeding should be discontinued if the mother is treated on this study Known human immunodeficiency virus (HIV) infection Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements Patient with documented hypersensitivity to any of the component medications | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 0.0-90.0, Alzheimer's Disease Clinical diagnosis of all cause dementia and probable Alzheimer's disease (AD) Mini-Mental State Examination (MMSE) score of 14-26 (inclusive) Cognitive impairment present for at least 6 months Age ≤90 years Modified Hachinski ischaemic score of ≤4 Females, if of childbearing potential, must use adequate contraception and maintain this use throughout participation in the study Patient is able to read, understand, and provide written informed consent Has one or more identified caregivers who are able to verify daily compliance with study drug and provide information on safety and tolerability; the caregiver(s) must also give consent to participate Currently taking an taking an acetylcholinesterase inhibitor and/or memantine; the subject must have been taking such medication(s) for ≥3 months. The dosage regimen must have remained stable for ≥6 weeks and it must be planned to remain stable throughout participation in the study Able to comply with the study procedures Significant central nervous system disorder other than Alzheimer's disease Patients in whom baseline MRI is contraindicated such as metal implants in head (except dental), pacemaker, and cochlear implant Significant focal or intracranial pathology that would lead to a diagnosis other than probable Alzheimer's disease Clinical evidence or history of stroke, transient ischemic attack, significant head injury or other unexplained or recurrent loss of consciousness Epilepsy Major depressive disorder, schizophrenia or other psychotic disorders, bipolar disorder, substance (including alcohol) related disorders Resides in a hospital or continuous care facility History of swallowing difficulties Pregnant or breastfeeding History of significant hematological abnormality or current acute or chronic clinically significant abnormality | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 16.0-999.0, Gastrointestinal Bleeding adult patients with acute significant upper or lower gastrointestinal bleeding where the responsible clinician is substantially uncertain as to the appropriateness of antifibrinolytic agents in the patient The fundamental criterion is the responsible clinician's 'uncertainty' as to whether or not to use an antifibrinolytic agent in a particular patient with upper or lower gastrointestinal bleeding There are no other exclusions | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 0.0-999.0, Breast Cancer Karnofsky ≥ 70 2. Provision of informed consent 3. Pathological confirmation of breast cancer and of other metastases. 4. Pathological confirmation of triple negative or Her-2 positive or with more than 4 axillary lymph node metastasis 5. The patients have finished the chemotherapy of Anthracycline and/or Taxane.And it's no more than 28 days from accepting the last chemotherapy. 6. Laboratory PLT ≥ 100*109/L WBC ≥ 4000/mm3 HGB ≥ 10g/dl GOT,GPT,ALP ≤ 2*ULN TBIL,DBIL,CCr ≤ 1.5*ULN Pregnant or lactation woman 2. Bilateral breast cancer, inflammatory breast cancer or carcinoma in situ 3. Accepted neoadjuvant treatment including chemotherapy, radiotherapy and endocrine therapy 4. History of organ transplantation 5. With mental disease 6. With severe infection or active gastrointestinal ulcers 7. With severe liver disease (such as cirrhosis), kidney disease, respiratory disease or diabetes 8. Disease-free period of other malignant tumor is less than 5 years(except cured basal cell skin cancer and cervical carcinoma in situ) 9. With heart disease 10. Experimental drug allergy | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 9.0-18.0, Transplantation Systemic Lupus Erythematosus Systemic Immune Disease Female gender Age ≥ 9 years and < 18 years Weight ≥ 25 kg Solid organ transplantation: kidney, liver, heart, lung, intestinal or combined transplant; or systemic lupus erythematosus or other systemic immune disease Transplantation or diagnosis of lupus or diagnosis of systemic immune disease since more than 6 months Immunosuppressant treatment by anti-metabolites or calcineurin inhibitors, with or without associated corticosteroids Minimum required period of 3 months considered as stable after transplantation or without relapse of lupus according to physician evaluation In case of sexual activity (assessed by auto-declaration): onset less than one year before Written informed consent signed by the investigator and the legal representatives of the patient, and assent by the patient Male gender Pregnancy Age < 9 years or ≥ 18 years Previous HPV vaccination Immunosuppressive treatment by anti-TNF (adalimumab, etanercept, infliximab) or monoclonal antibodies (rituximab, anakinra, abatacept) during the last 3 months Active malignancy Active opportunistic infection HIV infection Concurrent clinical trial | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 16.0-50.0, Cesarean Section Pregnancy cesarean section via transverse skin incision informed consent vertical skin incision | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 16.0-29.0, Acne Vulgaris Capable of understanding and willing to provide signed and dated written voluntary informed consent (and any local or national authorization requirements) before any protocol specific procedures are performed. 2. Male or female aged from 16 to 29 years, inclusive, at time of consent. No more than 50% of the subjects at each site can be enrolled under the age of 20. 3. Mild facial acne vulgaris, characterized by at least 12 facial inflammatory lesions (papules and pustules) and/or noninflammatory lesions (open and closed comedones) on the face. 4. Able to complete the study and to comply with study instructions. 5. Sexually active females of childbearing potential participating in the study must agree to use a medically acceptable method of contraception while receiving protocol-assigned product. A woman of childbearing potential is defined as one who is biologically capable of becoming pregnant; including perimenopausal women who are less than 2 years from their last menses. Acceptable contraceptive methods the following Hormonal contraception, including oral, injectable, or implantable methods started at least 2 months prior to screening. If hormonal contraception was started less than 2 months prior to screening, then a form of nonhormonal contraception should be added until the third continuous month of hormonal contraception has been completed Two forms of reliable nonhormonal contraception, to the use of either an intrauterine device plus a reliable barrier method or 2 reliable barrier methods. Reliable barrier methods condoms or diaphragms. A cervical cap is also a reliable barrier method, provided that the female subject has never given birth naturally. The combined use of a condom and spermicide constitute 2 forms of acceptable nonhormonal contraception, provided that they are both used properly. The use of spermicide alone and the improper use of condoms are inferior methods of contraception. Subjects with surgical sterilization, including tubal sterilization or partner's vasectomy, must use a form of nonhormonal contraception. A barrier method or sterilization plus spermatocide is acceptable Women who are not currently sexually active must agree to use a medically accepted method of contraception should she become sexually active while participating in the study Female who is pregnant, trying to become pregnant, or breast feeding. 2. Has active or chronic skin allergies. 3. Has a history of acute or chronic disease that might interfere with or increase the risk of study participation. 4. Had skin cancer treatment in preceding 12 months. 5. Has damaged skin on facial areas (eg, sunburn, tattoo, or scar) 6. Had any medical procedure (eg, laser resurfacing, chemical peel, or plastic surgery) on facial areas in preceding 12 months. 7. Had any cosmetic procedure (eg, microdermabrasion) on facial areas within 8 weeks of the baseline visit. 8. Has any dermatological disorder that in the opinion of the investigator may interfere with the accurate evaluation of the subject's facial appearance. 9. Received any investigational drug or procedure within 28 days of the baseline visit or is scheduled to receive an investigational drug (other than the study products) or procedure during the study. 10. Currently using any medication that in the opinion of the investigator may affect the evaluation of the study products or place the subject at undue risk (including but not limited to asthma medications, oral steroids, rifampin, anticonvulsants, and St John's wart). 11. Has a history of known or suspected intolerance to any of the ingredients of the study products (ie, benzoyl peroxide). 12. Considered unable or unlikely to attend the necessary visits. 13. Live in the same household as currently enrolled subjects. 14. Employee of the investigator, a contract research organization, or Stiefel Laboratories who is involved in the study, or an immediate family member (partner, offspring, parents, siblings or sibling's offspring) of an employee involved in the study | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 16.0-29.0, Acne Vulgaris Capable of understanding and willing to provide signed and dated written voluntary informed consent (and any local or national authorization requirements) before any protocol specific procedures are performed. 2. Male or female aged from 16 to 29 years, inclusive, at time of consent. No more than 50% of the subjects at each site can be enrolled under the age of 20. 3. Mild facial acne vulgaris, characterized by at least 24 facial inflammatory lesions (papules and pustules) and/or noninflammatory lesions (open and closed comedones) on the face. 4. Able to complete the study and to comply with study instructions. 5. Sexually active females of childbearing potential participating in the study must agree to use a medically acceptable method of contraception while receiving protocol-assigned product. A woman of childbearing potential is defined as one who is biologically capable of becoming pregnant; including perimenopausal women who are less than 2 years from their last menses. Acceptable contraceptive methods the following Hormonal contraception, including oral, injectable, or implantable methods started at least 2 months prior to screening. If hormonal contraception was started less than 2 months prior to screening, then a form of nonhormonal contraception should be added until the third continuous month of hormonal contraception has been completed Two forms of reliable nonhormonal contraception, to the use of either an intrauterine device plus a reliable barrier method or 2 reliable barrier methods. Reliable barrier methods condoms or diaphragms. A cervical cap is also a reliable barrier method, provided that the female subject has never given birth naturally. The combined use of a condom and spermicide constitute 2 forms of acceptable nonhormonal contraception, provided that they are both used properly. The use of spermicide alone and the improper use of condoms are inferior methods of contraception. Subjects with surgical sterilization, including tubal sterilization or partner's vasectomy, must use a form of nonhormonal contraception. A barrier method or sterilization plus spermatocide is acceptable Women who are not currently sexually active must agree to use a medically accepted method of contraception should she become sexually active while participating in the study Female who is pregnant, trying to become pregnant, or breast feeding. 2. Has active or chronic skin allergies. 3. Has a history of acute or chronic disease that might interfere with or increase the risk of study participation. 4. Had skin cancer treatment in preceding 12 months. 5. Has damaged skin on facial areas (eg, sunburn, tattoo, or scar) 6. Had any medical procedure (eg, laser resurfacing, chemical peel, or plastic surgery) on facial areas in preceding 12 months. 7. Had any cosmetic procedure (eg, microdermabrasion) on facial areas within 8 weeks of the baseline visit. 8. Has any dermatological disorder that in the opinion of the investigator may interfere with the accurate evaluation of the subject's facial appearance. 9. Received any investigational drug or procedure within 28 days of the baseline visit or is scheduled to receive an investigational drug (other than the study products) or procedure during the study. 10. Currently using any medication that in the opinion of the investigator may affect the evaluation of the study products or place the subject at undue risk (including but not limited to asthma medications, oral steroids, rifampin, anticonvulsants, and St John's wart). 11. Has a history of known or suspected intolerance to any of the ingredients of the study products (ie, benzoyl peroxide). 12. Considered unable or unlikely to attend the necessary visits. 13. Live in the same household as currently enrolled subjects. 14. Employee of the investigator, a contract research organization, or Stiefel Laboratories who is involved in the study, or an immediate family member (partner, offspring, parents, siblings or sibling's offspring) of an employee involved in the study | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 2.0-18.0, Recurrent Malignant Solid Neoplasm Recurrent Melanoma Recurrent Primary Central Nervous System Neoplasm Recurrent Thyroid Gland Carcinoma Refractory Malignant Solid Neoplasm Refractory Primary Central Nervous System Neoplasm Thyroid Gland Medullary Carcinoma Patients must have a body surface area >= 0.44 m^2 when enrolling on dose level -1; patients must have a body surface area >= 0.35 m^2 when enrolling on dose level 1, 2, or 3 PART A: Patients with relapsed or refractory solid tumors (excluding medullary thyroid cancer) including CNS tumors and malignant melanoma are eligible; patients must have had histologic verification of malignancy at original diagnosis or relapse except in patients with intrinsic brain stem tumors, optic pathway gliomas, or patients with pineal tumors and elevations of cerebrospinal fluid (CSF) or serum tumor markers including alpha-fetoprotein or beta-human chorionic gonadotropin (HCG) Part B: Patients with medullary thyroid cancer (MTC), with or without bone marrow involvement, will be eligible for Part B; these patients will be enrolled at dose level 2, the recommended phase 2 dose determined in the dose escalation part of the study Patients must have either measurable or evaluable disease Patient's current disease state must be one for which there is no known curative therapy or therapy proven to prolong survival with an acceptable quality of life Karnofsky >= 50% for patients > 16 years of age and Lansky >= 50 for patients =< 16 years of age Note: neurologic deficits in patients with CNS tumors must have been relatively stable for at least 7 days prior to study enrollment; patients who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score Patients must have fully recovered from the acute toxic effects of all prior anti-cancer chemotherapy Myelosuppressive chemotherapy: at least 21 days after the last dose of myelosuppressive chemotherapy (42 days if prior nitrosourea) Hematopoietic growth factors: at least 14 days after the last dose of a long-acting growth factor (e.g. Neulasta) or 7 days for short-acting growth factor; for agents that have known adverse events occurring beyond 7 days after administration, this period must be extended beyond the time during which adverse events are known to occur; the duration of this interval must be discussed with the study chair Pregnant or breast-feeding women will not be entered on this study; pregnancy tests must be obtained in girls who are post-menarchal; males or females of reproductive potential may not participate unless they have agreed to use two methods of birth control a medically accepted barrier method of contraceptive method (e.g., male or female condom) and a second effective contraceptive method of birth control during protocol therapy and for at least 4 months after the last dose of XL184; abstinence is an acceptable method of birth control Patients receiving corticosteroids who have not been on a stable or decreasing dose of corticosteroid for at least 7 days prior to enrollment are not eligible Patients who are currently receiving another investigational drug are not eligible Patients who are currently receiving other anti-cancer agents are not eligible Patients who are receiving cyclosporine, tacrolimus or other agents to prevent graft-versus-host disease post bone marrow transplant are not eligible for this trial Patients must not be receiving any of the following potent cytochrome P450 family 3, subfamily A, polypeptide 4 cytochrome (CYP3A4) inducers or inhibitors: erythromycin, clarithromycin, ketoconazole, azithromycin, itraconazole, grapefruit juice or St. John's wort Patients who are receiving systemic treatment anticoagulation are not eligible; patients receiving prophylactic systemic anticoagulation will be allowed as long as PT/INR requirements are met Patients must not have received enzyme-inducing anticonvulsants within 14 days prior to enrollment | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 13.0-18.0, Sexual and Reproductive Health Problems Nicaragua adolescents aged 13-18 living in the 33 town districts of Managua with a population number that varies between 1400 and 4500 inhabitants and with more than 50 % poor people. Bolivia and Ecuador: Students from conveniently selected secondary schools in Cochabamba (Bolivia) and Cuenca (Ecuador) | 2 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 12.0-65.0, Myasthenia Gravis 12 to 65 years； 2. Myasthenia gravis: 1. Patients who are diagnosed as generalized or ocular myasthenia gravis 2. have experienced extended thymectomy (including thymic hyperplasia and thymoma)， no significant complications in 6 months after operation , and does not received any immunosuppressants or glucocorticoids treatments. 3. do not applied in plasmapheresis or immunoglobulins treatment during 3 months . 4. women of child-bearing period do not have a plan of pregnant for at least 3 year. 5. Written consent of the patient, after informing The liver , kidney or glycometabolic function is abnormal 2. Seriously complications, such as infection or symptom in central nervous system, 3. The patients who suffering from malignancy or a history of malignancy, a variety of sexually transmitted diseases and HIV infection, tuberculosis infection, and other condition which need to prohibit the use of immunosuppressive patients. 4. Be allergic to leflunomide, azathioprine 5. Pregnant or suckling period woman 6. Accompanied with mental disorders and have difficult to communication 7. Experienced myasthenia crisis in 3 months. 8. suffering from clear cardiopulmonary functional and brain abnormalities 9. Have a history of refractory hypertension or peptic ulcer . 10. One of the white blood cells, hemoglobin, and platelet count obvious abnormalities | 1 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 2.0-25.0, Niemann-Pick Disease, Type C1 Aged greater than or equal to 2 and less than or equal to 25 years old at time of enrollment, either gender and any ethnicity. 2. Diagnosis of NPC1 based upon one of the following: 1. Two NPC1 mutations; 2. Positive filipin staining and at least one NPC1 mutation; 3. Vertical supranuclear gaze palsy (VSNGP) in combination with either: i. One NPC1 mutation, or ii. Positive filipin staining and no Niemann-Pick Type 2 (NPC2) mutations. 3. Patients with at least one neurological manifestation of NPC1. For example, but not limited to, hearing loss, vertical supranuclear gaze palsy, ataxia, dementia, dystonia, seizures, dysarthria, or dysphagia. 4. Ability to travel to the National Institutes of Health Clinical Center (NIH CC) repeatedly for evaluation and follow-up. 5. If taking miglustat, the patient must have been taking a constant dose of the medication for no less than 3 months prior to baseline evaluation and must be willing to maintain that dose level for the duration of the trial. 6. Willing to discontinue all non-prescription supplements, with the exception of an age-appropriate multivitamin. 7. Women of reproductive age must be willing to use an effective method of contraception for the duration of the trial. 8. Willing to participate in all aspects of trial design including serial blood and cerebrospinal fluid (CSF) collections Aged below 2 or above 25 years of age at enrollment in the trial. 2. Subjects will be excluded if their weight would result in an endotoxin level that would exceed 0.2 EU/kg for either the saline or drug dosing. 3. Severe manifestations of NPC1 that would interfere with the patient's ability to comply with the requirements of this protocol. 4. Neurologically asymptomatic patients. 5. Patients who have received any form of cyclodextrin in an attempt to treat NPC1. Treatment with another drug preparation for another medical indication that contains cyclodextrin as an excipient, will not a patient. 6. History of hypersensitivity reactions to cyclodextrin or components of the formulation. 7. Pregnancy or breastfeeding at any time during the study. 8. Patients with suspected infection of the CNS or any systemic infection. 9. Spinal deformity that would impact the ability to perform a lumbar puncture 10. Skin infection in the lumbar region 11. Neutropenia, defined as an absolute neutrophil count (ANC) of less than 1,500. 12. Thrombocytopenia (a platelet count of less than 75,000 per cubic millimeter). 13. Evidence of disturbed circulation of CSF. 14. Contraindication for anesthesia. 15. Prior use of anticoagulants or history/presence of a bleeding disorder with increased risk of clinical bleeding or an international normalized ratio (INR) greater than 2. 16. Patients with clinical evidence of acute liver disease having symptoms of jaundice or right upper quadrant pain. 17. Presence of anemia defined as two standard deviations below normal for age and gender. 18. For subjects 18 years of age and older, the epidermal growth factor receptor (eGFR) is automatically calculated and reported by the NIH CC laboratory utilizing the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) Creatinine 2009 equation. We will subjects greater than or equal to 18 years of age if eGFR is less than or equal to 60 mL/min/1.73 m2. For subjects < 18 12 years of age, we will utilize the national kidney disease education program (NKDEP) calculator (http://www.nkdep.nih.gov/lab-evaluation/gfr-calculators/children-conventional-unit.sh tml). Results are reported as > 75 mL/min/1.73 m2 or lower. We will subjects < 18 years of age if eGFR is less than or equal to 75 15 mL/min/1.73 m2 19. Hematuria on a single urinalysis, as defined by the American Urological Association (AUA) as five or more red blood cells per high-power field (or > 25/micro L) on microscopic evaluation of urinary sediment from a properly collected urinalysis specimen. The patient will not be excluded if 2 subsequent urine specimens are negative for hematuria as defined by the AUA. 20. Proteinuria (1+ protein on urinalysis) unless evaluated and classified as benign by patient s primary medical provider or by NIH nephrology consult or in the context of normal urine protein creatinine ratio and in the absence of clinical symptoms (edema, hypertension). 21. Active pulmonary disease, oxygen requirement or clinically significant history of decreased blood oxygen saturation, pulmonary therapy, or requiring active suction. 22. Patients unable to complete a behavioral audiologic evaluation including pure-tone threshold assessment (500 Hz to 8000 Hz) to monitor for ototoxicity and for whom otoacoustic emissions (OAEs) cannot be reliably obtained at baseline. 23. Patients with ongoing seizures, that are not stable in frequency, type or duration over a 2 month period prior to enrollment, requiring change in dose of antiepileptic medication (other than adjustment for weight) over a 2 month period prior to enrollment, or requiring 3 or more antiepileptic medications to control seizures. 24. Patients, who in the opinion of the investigators are unable to comply with the protocol or have specific health concerns that would potentially increase the risk of participation | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 0.0-65.0, Uterine Cervical Neoplasms Cervical Cancer Uterine Cervical Cancer FIGO stage: ⅠB～ⅡA, cervical cancer Age≤60 years; female, Chinese women Initial treatment is radical hysterectomy + pelvic lymph node dissection Pathological diagnosis: cervical squamous cell invasive carcinoma Pathologic examination and meet the following one of the indications of adjuvant therapy: ① lymph node metastasis, ② parametrial invasion, ③ ≥ 2/3 deep stromal invasion, ④ histopathological grading in poorly differentiated (G2 to G3), ⑤ lymphatic vascular space involvement, ⑥ tumor diameter> 4cm Laboratory tests: WBC≥4×10(9)/L, NEU≥2×10(9)/L, PLT≥80×10(9)/L, serum bilirubin≤ 1.5 times the upper limit of normal, transaminase≤ 1.5 times the upper limit of normal, BUN, Cr≤ normal Performance status: Karnofsky score≥60 No prior treatment Receiving extensive resection of uterus (type III) plus pelvic lymph nodes wide resection; pathologically diagnosed with cervical squamous cell carcinoma Provide written informed consent With severe or uncontrolled internal disease, unable to receive surgery and/or unsuitable for radiotherapy or chemotherapy History of organ transplantation, immune diseases History of serious mental illness, a history of brain dysfunction Drug abuse or a history of drug abuse Suffering from other malignancies Concurrently participating in other clinical trials Unable or unwilling to sign informed consents Unable or unwilling to abide by protocol | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 3.0-18.0, Pontine Glioma Subjects must have radiographically proven diffuse intrinsic pontine glioma and confirmation of residual disease after initial therapy or at the time of recurrence/progression as confirmed by MRI of the brain Subjects must be age ≥3 years and ≤ 18 years Diffuse intrinsic pontine glioma with measurable disease after receiving radiotherapy either concurrent with or followed by ≤ 2 prior courses of chemotherapy Measurable disease as defined by: Measurable tumor >10mm by MRI Karnofsky performance status (PS) 60-100% (for patients > 16 years of age) OR Lansky PS 60-100% (for patients ≤ 16 years of age) Body surface area > 0.67 m2 and ≤ 2.21 m2 Life expectancy of at least 2 months A negative urine pregnancy test is required for female participants of child bearing potential (≥13 years of age or after onset of menses) Acceptable liver function as defined by: 1. Bilirubin ≤ 1.5 times upper limit of normal 2. AST (SGOT), ALT (SGPT) and Alkaline phosphatase ≤ 2.5 times upper limit of normal Creatinine clearance or radioisotope GFR ≥ 70 mL/min OR serum creatinine based on age as follows Concurrent anti-cancer therapy (chemotherapy, radiation therapy, surgery, immunotherapy, hormonal therapy, biologic therapy) other than the ones specified in the protocol. Patients must have discontinued the above cancer therapies for generally about 3 weeks (8 weeks for radiotherapy) prior to the first dose of study medication, as well as recovered from toxicity (to ≤ than grade 2 except for alopecia) induced by previous treatments Currently receiving another investigational medicinal product Uncontrolled concurrent illness including, but not limited to: 1. Active, uncontrolled bacterial, viral, or fungal infections requiring systemic therapy 2. Diarrhea of any cause ≥ CTCAE grade 2 3. Psychiatric illness/social situations that would compromise patient safety or limit compliance with study requirements including maintenance of a compliance/pill diary 4. Any kind of malabsorption syndrome significantly affecting gastrointestinal function Pregnant or nursing female patients. NOTE: If a female patient becomes pregnant or suspects that she is pregnant while participating in this study, she should stop taking study drug and immediately inform her treating physician immediately Prior therapy with a Hedgehog inhibitor Unwillingness or inability to comply with procedures required in this protocol Serious nonmalignant disease (e.g., hydronephrosis, liver failure, or other conditions) that could compromise protocol objectives in the opinion of the investigator and/or the sponsor History of Congestive Heart Failure (CHF) or ventricular arrhythmia requiring medication | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 0.0-999.0, Rare Disorders Undiagnosed Disorders Disorders of Unknown Prevalence Cornelia De Lange Syndrome Prenatal Benign Hypophosphatasia Perinatal Lethal Hypophosphatasia Odontohypophosphatasia Adult Hypophosphatasia Childhood-onset Hypophosphatasia Infantile Hypophosphatasia Hypophosphatasia Kabuki Syndrome Bohring-Opitz Syndrome Narcolepsy Without Cataplexy Narcolepsy-cataplexy Hypersomnolence Disorder Idiopathic Hypersomnia Without Long Sleep Time Idiopathic Hypersomnia With Long Sleep Time Idiopathic Hypersomnia Kleine-Levin Syndrome Kawasaki Disease Leiomyosarcoma Leiomyosarcoma of the Corpus Uteri Leiomyosarcoma of the Cervix Uteri Leiomyosarcoma of Small Intestine Acquired Myasthenia Gravis Addison Disease Hyperacusis (Hyperacousis) Juvenile Myasthenia Gravis Transient Neonatal Myasthenia Gravis Williams Syndrome Lyme Disease Myasthenia Gravis Marinesco Sjogren Syndrome(Marinesco-Sjogren Syndrome) Isolated Klippel-Feil Syndrome Frasier Syndrome Denys-Drash Syndrome Beckwith-Wiedemann Syndrome Emanuel Syndrome Isolated Aniridia Axenfeld-Rieger Syndrome Aniridia-intellectual Disability Syndrome Aniridia - Renal Agenesis - Psychomotor Retardation Aniridia - Ptosis - Intellectual Disability - Familial Obesity Aniridia - Cerebellar Ataxia - Intellectual Disability Aniridia - Absent Patella Aniridia Peters Anomaly - Cataract Peters Anomaly Potocki-Shaffer Syndrome Silver-Russell Syndrome Due to Maternal Uniparental Disomy of Chromosome 11 Silver-Russell Syndrome Due to Imprinting Defect of 11p15 Silver-Russell Syndrome Due to 11p15 Microduplication Syndromic Aniridia WAGR Syndrome Wolf-Hirschhorn Syndrome 4p16.3 Microduplication Syndrome 4p Deletion Syndrome, Non-Wolf-Hirschhorn Syndrome Autosomal Recessive Stickler Syndrome Stickler Syndrome Type 2 Stickler Syndrome Type 1 Stickler Syndrome Mucolipidosis Type 4 X-linked Spinocerebellar Ataxia Type 4 X-linked Spinocerebellar Ataxia Type 3 X-linked Intellectual Disability - Ataxia - Apraxia X-linked Progressive Cerebellar Ataxia X-linked Non Progressive Cerebellar Ataxia X-linked Cerebellar Ataxia Vitamin B12 Deficiency Ataxia Toxic Exposure Ataxia Unclassified Autosomal Dominant Spinocerebellar Ataxia Thyroid Antibody Ataxia Sporadic Adult-onset Ataxia of Unknown Etiology Spinocerebellar Ataxia With Oculomotor Anomaly Spinocerebellar Ataxia With Epilepsy Spinocerebellar Ataxia With Axonal Neuropathy Type 2 Spinocerebellar Ataxia Type 8 Spinocerebellar Ataxia Type 7 Spinocerebellar Ataxia Type 6 Spinocerebellar Ataxia Type 5 Spinocerebellar Ataxia Type 4 Spinocerebellar Ataxia Type 37 Spinocerebellar Ataxia Type 36 Spinocerebellar Ataxia Type 35 Spinocerebellar Ataxia Type 34 Spinocerebellar Ataxia Type 32 Spinocerebellar Ataxia Type 31 Spinocerebellar Ataxia Type 30 Spinocerebellar Ataxia Type 3 Spinocerebellar Ataxia Type 29 Spinocerebellar Ataxia Type 28 Spinocerebellar Ataxia Type 27 Spinocerebellar Ataxia Type 26 Spinocerebellar Ataxia Type 25 Spinocerebellar Ataxia Type 23 Spinocerebellar Ataxia Type 22 Spinocerebellar Ataxia Type 21 Spinocerebellar Ataxia Type 20 Spinocerebellar Ataxia Type 2 Spinocerebellar Ataxia Type 19/22 Spinocerebellar Ataxia Type 18 Spinocerebellar Ataxia Type 17 Spinocerebellar Ataxia Type 16 Spinocerebellar Ataxia Type 15/16 Spinocerebellar Ataxia Type 14 Spinocerebellar Ataxia Type 13 Spinocerebellar Ataxia Type 12 Spinocerebellar Ataxia Type 11 Spinocerebellar Ataxia Type 10 Spinocerebellar Ataxia Type 1 With Axonal Neuropathy Spinocerebellar Ataxia Type 1 Spinocerebellar Ataxia - Unknown Spinocerebellar Ataxia - Dysmorphism Non Progressive Epilepsy and/or Ataxia With Myoclonus as a Major Feature Spasticity-ataxia-gait Anomalies Syndrome Spastic Ataxia With Congenital Miosis Spastic Ataxia - Corneal Dystrophy Spastic Ataxia Rare Hereditary Ataxia Rare Ataxia Recessive Mitochondrial Ataxia Syndrome Progressive Epilepsy and/or Ataxia With Myoclonus as a Major Feature Posterior Column Ataxia - Retinitis Pigmentosa Post-Stroke Ataxia Post-Head Injury Ataxia Post Vaccination Ataxia Polyneuropathy - Hearing Loss - Ataxia - Retinitis Pigmentosa - Cataract Muscular Atrophy - Ataxia - Retinitis Pigmentosa - Diabetes Mellitus Non-hereditary Degenerative Ataxia Paroxysmal Dystonic Choreathetosis With Episodic Ataxia and Spasticity Olivopontocerebellar Atrophy - Deafness NARP Syndrome Myoclonus - Cerebellar Ataxia - Deafness Multiple System Atrophy, Parkinsonian Type Multiple System Atrophy, Cerebellar Type Multiple System Atrophy Maternally-inherited Leigh Syndrome Machado-Joseph Disease Type 3 Machado-Joseph Disease Type 2 Machado-Joseph Disease Type 1 Leigh Syndrome Late-onset Ataxia With Dementia Infection or Post Infection Ataxia GAD Ataxia Hereditary Episodic Ataxia Gliadin/Gluten Ataxia Friedreich Ataxia Fragile X-associated Tremor/Ataxia Syndrome Familial Paroxysmal Ataxia Exposure to Medications Ataxia Episodic Ataxia With Slurred Speech Episodic Ataxia Unknown Type Episodic Ataxia Type 7 Episodic Ataxia Type 6 Episodic Ataxia Type 5 Episodic Ataxia Type 4 Episodic Ataxia Type 3 Episodic Ataxia Type 1 Epilepsy and/or Ataxia With Myoclonus as Major Feature Early-onset Spastic Ataxia-neuropathy Syndrome Early-onset Progressive Neurodegeneration - Blindness - Ataxia - Spasticity Early-onset Cerebellar Ataxia With Retained Tendon Reflexes Early-onset Ataxia With Dementia Childhood-onset Autosomal Recessive Slowly Progressive Spinocerebellar Ataxia Dilated Cardiomyopathy With Ataxia Cataract - Ataxia - Deafness Cerebellar Ataxia, Cayman Type Cerebellar Ataxia With Peripheral Neuropathy Cerebellar Ataxia - Hypogonadism Cerebellar Ataxia - Ectodermal Dysplasia Cerebellar Ataxia - Areflexia - Pes Cavus - Optic Atrophy - Sensorineural Hearing Loss Brain Tumor Ataxia Brachydactyly - Nystagmus - Cerebellar Ataxia Benign Paroxysmal Tonic Upgaze of Childhood With Ataxia Autosomal Recessive Syndromic Cerebellar Ataxia Autosomal Recessive Spastic Ataxia With Leukoencephalopathy Autosomal Recessive Spastic Ataxia of Charlevoix-Saguenay Autosomal Recessive Spastic Ataxia - Optic Atrophy - Dysarthria Autosomal Recessive Spastic Ataxia Autosomal Recessive Metabolic Cerebellar Ataxia Autosomal Dominant Spinocerebellar Ataxia Due to Repeat Expansions That do Not Encode Polyglutamine Autosomal Recessive Ataxia, Beauce Type Autosomal Recessive Ataxia Due to Ubiquinone Deficiency Autosomal Recessive Ataxia Due to PEX10 Deficiency Autosomal Recessive Degenerative and Progressive Cerebellar Ataxia Autosomal Recessive Congenital Cerebellar Ataxia Due to MGLUR1 Deficiency Autosomal Recessive Congenital Cerebellar Ataxia Due to GRID2 Deficiency Autosomal Recessive Congenital Cerebellar Ataxia Autosomal Recessive Cerebellar Ataxia-pyramidal Signs-nystagmus-oculomotor Apraxia Syndrome Autosomal Recessive Cerebellar Ataxia-epilepsy-intellectual Disability Syndrome Due to WWOX Deficiency Autosomal Recessive Cerebellar Ataxia-epilepsy-intellectual Disability Syndrome Due to TUD Deficiency Autosomal Recessive Cerebellar Ataxia-epilepsy-intellectual Disability Syndrome Due to KIAA0226 Deficiency Autosomal Recessive Cerebellar Ataxia-epilepsy-intellectual Disability Syndrome Autosomal Recessive Cerebellar Ataxia With Late-onset Spasticity Autosomal Recessive Cerebellar Ataxia Due to STUB1 Deficiency Autosomal Recessive Cerebellar Ataxia Due to a DNA Repair Defect Autosomal Recessive Cerebellar Ataxia - Saccadic Intrusion Autosomal Recessive Cerebellar Ataxia - Psychomotor Retardation Autosomal Recessive Cerebellar Ataxia - Blindness - Deafness Autosomal Recessive Cerebellar Ataxia Autosomal Dominant Spinocerebellar Ataxia Due to a Polyglutamine Anomaly Autosomal Dominant Spinocerebellar Ataxia Due to a Point Mutation Autosomal Dominant Spinocerebellar Ataxia Due to a Channelopathy Autosomal Dominant Spastic Ataxia Type 1 Autosomal Dominant Spastic Ataxia Autosomal Dominant Optic Atrophy Ataxia-telangiectasia Variant Ataxia-telangiectasia Autosomal Dominant Cerebellar Ataxia, Deafness and Narcolepsy Autosomal Dominant Cerebellar Ataxia Type 4 Autosomal Dominant Cerebellar Ataxia Type 3 Autosomal Dominant Cerebellar Ataxia Type 2 Autosomal Dominant Cerebellar Ataxia Type 1 Autosomal Dominant Cerebellar Ataxia Ataxia-telangiectasia-like Disorder Ataxia With Vitamin E Deficiency Ataxia With Dementia Ataxia - Oculomotor Apraxia Type 1 Ataxia - Other Ataxia - Genetic Diagnosis - Unknown Acquired Ataxia Adult-onset Autosomal Recessive Cerebellar Ataxia Alcohol Related Ataxia Multiple Endocrine Neoplasia Multiple Endocrine Neoplasia Type II Multiple Endocrine Neoplasia Type 1 Multiple Endocrine Neoplasia Type 2 Multiple Endocrine Neoplasia, Type IV Multiple Endocrine Neoplasia, Type 3 Multiple Endocrine Neoplasia (MEN) Syndrome Multiple Endocrine Neoplasia Type 2B Multiple Endocrine Neoplasia Type 2A Atypical Hemolytic Uremic Syndrome Atypical HUS Wiedemann-Steiner Syndrome Breast Implant-Associated Anaplastic Large Cell Lymphoma Autoimmune/Inflammatory Syndrome Induced by Adjuvants (ASIA) Hemophagocytic Lymphohistiocytosis Behcet's Disease Alagille Syndrome Inclusion Body Myopathy With Early-onset Paget Disease and Frontotemporal Dementia (IBMPFD) Lowe Syndrome Pitt Hopkins Syndrome 1p36 Deletion Syndrome Jansen Type Metaphyseal Chondrodysplasia Cockayne Syndrome Chronic Recurrent Multifocal Osteomyelitis CRMO Malan Syndrome Hereditary Sensory and Autonomic Neuropathy Type Ie VCP Disease Hypnic Jerking Sleep Myoclonus Mollaret Meningitis Recurrent Viral Meningitis CRB1 Leber Congenital Amaurosis Retinitis Pigmentosa Rare Retinal Disorder KCNMA1-Channelopathy Primary Biliary Cirrhosis ZMYND11 Transient Global Amnesia Glycogen Storage Disease Alstrom Syndrome White Sutton Syndrome DNM1 EIEE31 Myhre Syndrome Recurrent Respiratory Papillomatosis Laryngeal Papillomatosis Tracheal Papillomatosis Refsum Disease Nicolaides Baraitser Syndrome Leukodystrophy Tango2 Cauda Equina Syndrome Rare Gastrointestinal Disorders Achalasia-Addisonian Syndrome Achalasia Cardia Achalasia Icrocephaly Syndrome Anal Fistula Congenital Sucrase-Isomaltase Deficiency Eosinophilic Gastroenteritis Idiopathic Gastroparesis Hirschsprung Disease Rare Inflammatory Bowel Disease Intestinal Pseudo-Obstruction Scleroderma Short Bowel Syndrome Sacral Agenesis Sacral Agenesis Syndrome Caudal Regression Scheuermann Disease SMC1A Loss of Function Epilepsy Diagnosis of a rare disease, a disease of unknown prevalence, undiagnosed or an unaffected carrier of a rare/uncommon disease Diagnosis of a disease which is not rare | 2 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 18.0-85.0, Pruritus Patients w/ histologically confirmed mycosis fungoides stage IB to IVA eligible to receive oral vorinostat Patients w/ stage IB to IV reporting pruritus Patients age 18-85 years, of any race, sex, and ethnicity Life expectancy > 24 weeks Patient must have performance status of ≤2 on the ECOG Performance Scale Patients w/ a min. of 3 weeks since their last systemic treatment Women who are not pregnant, lactating, or of childbearing potential Female patients w/ reproductive potential must use an adequate contraceptive method during treatment and for three months after completing treatment Male patient w/ reproductive potential, agrees to use an adequate method of contraception for the duration of the study and for 30 days beyond the duration of study Patients, or legal representative must to be willing to adhere to the protocol, and sign an Informed Patient Consent Form prior to entry into the study Patients w/ a recent cardiac history, such as a myocardial infarct within the last year, uncontrolled angina, severe uncontrolled ventricular arrhythmias, clinically significant pericardial disease, or electrocardiographic evidence of acute ischemic or active conduction system abnormalities Patients w/ a history of liver damage (2.5 x normal ALT, AST), leukopenia, or thrombocytopenia Women who are pregnant or nursing a child Patients w/ severe emotional, behavioral or psychiatric problems that, in the opinion of the investigator, would result in poor compliance with the treatment regimen Patients who have received and histone deacetylase inhibitor within the last 6 months Patients receiving valproic acid will be excluded unless there has been a wash-out period of 30 or more days Patients who will have received systemic therapy, radiation therapy or phototherapy within 3 weeks prior to initial dosing with study drugs or who has not recovered from adverse events due to agents administered more than 3 weeks earlier QTc prolongation greater than 500ms Patient w/ a "currently active" second malignancy, other than non-melanoma skin cancer and carcinoma in situ of the cervix, should not be enrolled Patients are not considered to have a "currently active" malignancy if they have completed therapy for a prior malignancy, are disease free from prior malignancies for >5 years or are considered by their physician to be at less than 30% risk of relapse | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 1.0-50.0, Refractory and/or Relapsed Metastatic Solid Tumors Presence of a suitable related HLA-haploidentical bone marrow donor.a. The donor and recipient must be identical at at least one allele of each of the following genetic loci: HLA-A, HLA-B, HLA-Cw, HLA-DRB1, and HLA-DQB1. A minimum match of 5/10 is therefore required, and will be considered sufficient evidence that the donor and recipient share one HLA haplotype. 1 year-50 years Patients must have a confirmed histopathologic diagnosis and be classified as high risk defined by having an expected survival of < 10%. Examples Neuroblastoma or ganglioneuroblastoma Failure to achieve at least a PR after induction therapy with COG ANBL0532 or standard chemotherapy Refractory to induction chemotherapy with COG ANBL0532 or standard chemotherapy Patients with high risk disease as defined in Appendix 1 whose autologous peripheral blood stem cell product is contaminated with neuroblastoma or who do not have an autologous product available Patients with high risk disease as defined in Appendix 1 who do not meet requirements/organ function requirements for myeloablative conditioning. Patients with >5 identified lesions on the end of induction (COG ANBL0532 or standard chemotherapy) MIBG scan Stage 4 rhabdomyosarcoma Metastatic Ewing Sarcoma Osteosarcoma with metastatic disease beyond the lungs and/or with lung metastases not amenable to resection Desmoplastic small round cell tumor | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 0.0-999.0, Postmenopausal Women Osteoporosis women osteoporosis 2. patients with a diagnosis of Postmenopausal over 6 Months if they had a bone mineral density T-score of < -2.5 at the mean lumbar spine (L1-4), femoral neck, or total, or a T-score of < -1.0 with radiologic evidence of at least one vertebral fracture. Menopause was defined as no natural menses for at least 1 year and a serum FSH level > 40 IU/L, with a reported hysterectomy 3. low levels of 25(OH)D > 9 ng/mL 4. patients who give written consent of agreement to voluntarily participate in the clinical study 5. patients who can read and understand written instructions patients who had contraindications to oral bisphosphonates, such as esophageal strictures 2. ALT, AST ≥ 2×UNL and Serum Creatinine ≥ 1.5×UNL 3. low levels of 25(OH)D (less than 9 ng/mL). 4. Previous use of oral bisphosphonates and vitamin D were allowed, but a washout period was needed, depending on the duration of treatment. Two-year washout periods were needed for bisphosphonate users and 3-6-month periods were required for vitamin D users of > 200 IU. 5. drug administration after diagnosing as alcoholic or psychical disease 6. patients whom the investigators judge as improper to participate in this clinical trial 7)13.patients who have experience to participate in other clinical trial within 30 days prior to study participation | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 5.0-30.0, Osteoporosis Cystic Fibrosis age 2-30 years clinically stable condition regular menses in females low Bone Mineral Apparent Density for age (defined as BMAD Z-score ≤-2.0 if age ≤18 years or ≤-2.5 if age >18 years) two or more episodes of hypercalcemia and/or hypercalciuria contraindications to 25-OH vitamin D or alendronate treatment recent transplantation other diseases or medications (glucocorticoids excepted) associated with bone loss | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 16.0-75.0, Metastatic Ocular Melanoma Metastatic Uveal Melanoma Measurable metastatic ocular melanoma. 2. Confirmation of diagnosis of metastatic ocular melanoma by the Laboratory of Pathology of the National Cancer Institute (NCI). 3. Patients with 3 or fewer brain metastases that are less than 1 cm in diameter and asymptomatic are eligible. Lesions that have been treated with stereotactic radiosurgery must be clinically stable for 1 month after treatment for the patient to be eligible. Patients with surgically resected brain metastases are eligible. 4. Greater than or equal to 16 years of age and less than or equal to age 75. 5. Able to understand and sign the Informed Consent Document 6. Willing to sign a durable power of attorney 7. Clinical performance status of Eastern Cooperative Oncology Group (ECOG) 0 or 1 8. Life expectancy of greater than three months 9. Patients of both genders must be willing to practice birth control from the time of enrollment on this study and for up to four months after receiving the treatment. 10. Serology Seronegative for human immunodeficiency virus (HIV) antibody. (The experimental treatment being evaluated in this protocol depends on an intact immune system. Patients who are HIV seropositive can have decreased immune-competence and thus be less responsive to the experimental treatment and more susceptible to its toxicities.) Seronegative for hepatitis B antigen, and seronegative for hepatitis C antibody. If hepatitis C antibody test is positive, then patient must be tested for the presence of antigen by reverse transcription-polymerase chain reaction (RT-PCR) and be hepatitis C virus (HCV) ribonucleic acid (RNA) negative. 11. Women of child-bearing potential must have a negative pregnancy test because of the potentially dangerous effects of the treatment on the fetus. 12. Hematology Absolute neutrophil count greater than 1000/mm^3 without the support of filgrastim White blood cell (WBC) greater than or equal to 3000/mm^3 Platelet count greater than or equal 100,000/mm^3 Hemoglobin > 8.0 g/dl 13. Chemistry Serum alanine aminotransferase (ALT)/aspartate aminotransferase (AST) less than or equal to 3.5 times the upper limit of normal Serum creatinine less than or equal to 1.6 mg/dl Total bilirubin less than or equal to 2.0 mg/dl, except in patients with Gilbert's Syndrome who must have a total bilirubin less than 3.0 mg/dl. 14. More than four weeks must have elapsed since any prior systemic therapy at the time the patient receives the preparative regimen, and patients toxicities must have recovered to a grade 1 or less (except for toxicities such as alopecia or vitiligo). Note: Patients may have undergone minor surgical procedures within the past 3 weeks, as long as all toxicities have recovered to grade 1 or less or as specified in the criteria Women of child-bearing potential who are pregnant or breastfeeding because of the potentially dangerous effects of the treatment on the fetus or infant. 2. Active systemic infections, coagulation disorders or other active major medical illnesses of the cardiovascular, respiratory or immune system, as evidenced by a positive stress thallium or comparable test, myocardial infarction, cardiac arrhythmias, obstructive or restrictive pulmonary disease. 3. Any form of primary immunodeficiency (such as Severe Combined Immunodeficiency Disease). 4. Concurrent opportunistic infections (The experimental treatment being evaluated in this protocol depends on an intact immune system. Patients who have decreased immune competence may be less responsive to the experimental treatment and more susceptible to its toxicities). 5. Concurrent systemic steroid therapy. 6. History of severe immediate hypersensitivity reaction to any of the agents used in this study. 7. The following patients will be excluded from the high-dose aldesleukin arm (but may be eligible for cells alone arm): 1. History of coronary revascularization or ischemic symptoms 2. Documented left ventricular ejection fraction (LVEF) of less than or equal to 45%. Testing is required in patients with Clinically significant atrial and/or ventricular arrhythmias including but not limited to: atrial fibrillation, ventricular tachycardia, second or third degree heart block Age greater than or equal to 60 years old 3. Clinically significant patient history which in the judgment of the Principal Investigator would compromise the patients ability to tolerate aldesleukin | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 0.0-70.0, High Risk Hematologic Malignancies Age 0-70 years 2. Karnofsky or Lansky Performance status >70% 3. High risk hematologic malignancy 4. Acute myeloid leukemia (AML) with one or more of the following 4a.Poor risk cytogenetics, including -5, 5q-, -7, 7q-, t(9;22); complex cytogenetics (>3 abnormalities); or normal cytogenetics with Flt3 internal tandem duplication (ITD), in first or subsequent complete remission (CR). 4b. Relapsed or primary refractory AML with <10% blasts in the peripheral blood. 4c. Subjects in first complete remission (CR1) who required two cycles of induction to achieve remission may be included at the discretion of the treating physician. 4d. Standard risk or intermediate risk cytogenetics in second or subsequent CR (enrolled at the discretion of the treating physician). 5. Acute lymphoblastic leukemia (ALL) with one of the following 5a. Second or subsequent CR 5b. Any partial remission (PR) (no circulating blasts) 5c. High-risk ALL in first CR including (Ph+, t(4:11), complex karyotype, hypodiploidy (<44 chromosomes), or positive minimal residual disease (MRD) after induction 6. Myelodysplasia, intermediate -2 (score 1.5-2.0) or high risk (score >2.5) by the International Prognostic Score System. 7. Myeloproliferative Disorders (include chronic myelomonocytic leukemia (CMML), agnogenic myeloid metaplasia (AMM) or Idiopathic Myelofibrosis, and JMML) with excess blasts (>5%) 8. Chronic myeloid leukemia (CML) with one of the following 8a. Second or subsequent chronic phase 8b. Accelerated phase 8c. blast crisis 9. Non-Hodgkin's lymphoma (NHL) meeting one of the following 9a. Relapse after autologous stem cell transplantation with evidence of responsive disease. 9b. Subject with chemosensitive relapse who have no option for autologous stem cell transplantation due to blood or marrow involvement or failure to mobilize autologous stem cells or are not considered eligible for autologous transplant by their treating physician. 9c. Hodgkin's Lymphoma: relapse after autologous hematopoietic cell transplantation (HCT), chemo-refractory disease 9d. Multiple myeloma: per National Comprehensive Cancer Network (NCCN) guidelines. Updated annually at: www.nccn.org 10. No suitable human leukocyte antigen (HLA)-identical sibling donor. 11. No identified 8/8 (based upon A, B, C, DR beta 1 (DRB1) loci) allele matched unrelated donor, or unable to wait sufficient time to procure a 8/8 allele matched unrelated donor 12. Available HLA 3-5/6 matched genotypically haploidentical partially matched related donor 13. Female subjects must be surgically sterile, postmenopausal (minimum 1 year without menses), or agree to use approved form of contraception from the time of signing the informed consent form through Day +100. Male subjects must also agree to use an approved form of birth control for either themselves or their partner, as appropriate, from the time of signing the informed consent form through Day +100. 14. Able to provide informed consent and have signed an approved consent form that conforms to federal and institutional guidelines Available HLA identical matched sibling donor (unless having failed a prior allogeneic transplant from an HLA identical matched sibling) 2. Recipient HLA antibodies against donor HLA 3. Any of the following organ dysfunctions: 1. Cardiac left ventricular ejection fraction <40%, symptomatic coronary artery disease, or uncontrolled arrhythmias 2. Pulmonary forced expiratory volume at one second (FEV1) or diffusion capacity of lung for carbon monoxide (DLco)<40% or need for use of supplemental oxygen 3. Renal calculated or measured glomerular filtration rate (GFR) <30 ml/min, dialysis requirement, or prior renal transplant 4. Hepatic bilirubin > 2.0, alanine aminotransferase (ALT) > 2.5 X upper limit of normal (ULN), cirrhosis 4. Subjects with active or uncontrolled bacterial, viral, or fungal infections requiring systemic therapy. 5. Subjects who have tested positive for HIV. 6. Pregnant women, nursing mothers or women of child-bearing potential who are unwilling to use medically accepted methods of contraception | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 18.0-80.0, Myasthenia Gravis AChR Ab positive myasthenia gravis (acetylcholine receptor antibody). 2. Age 18-80 years. 3. MGFA Classification II-IV (The scale used to determine the severity of symptoms of MG). 4. Receiving > or equal 30mg of Prednisone per day. 5. No new MG-specific treatments in prior 3 months. 6. Willingness to participate in study protocol. 7. QMG > 10 (quantitative myasthenia gravis score: the sum of grades given for symptoms of MG). 8. Treatment with any immunomodulator > than or equal to 3 months prior to trial initiation IgA deficiency (a major class of immunoglobulins found in serum and external body secretions such as saliva, tears, and sweat as well as in the gastrointestinal, respiratory, and genitourinary tracts). 2. Previous thromboembolic events, including deep vein thrombosis, stroke and myocardial infarction 3. MGFA Class I, IV (if patient requires hospitalization) or V 4. History of thymoma 5. Thymectomy in previous year or planning to undergo thymectomy in next six months 6. Pregnancy or lactation; unwillingness to avoid pregnancy 7. Serious concurrent medical, neurological or psychiatric condition that would interfere with IGSC administration or subsequent clinical assessments 8. Unwillingness or incapacity to participate, agree to necessary follow-up visits, or give written and informed consent 9. Patients who have had an anaphylactic or severe systemic reaction to the administration of human immune globulin or to components of Hizentra, such as polysorbate 80, or patients with hyperprolinemia because it contains the stabilizer L-proline 10. Cholinesterase inhibitor no more than 240 mg/day 11. Body weight greater than 120 kg | 1 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 13.0-21.0, Obstructive Sleep Apnea Syndrome Polycystic Ovary Syndrome Sleep Apnea Female Ages 13-21 PCOS BMI >95%ile (Obese group) or <85%ile (Lean group) Controls: ages 18-21, regular menses, BMI <85%ile Breastfeeding Pregnant Use of any steroid preparations (including hormonal contraception), medications known to alter insulin secretion and/or action within 3 month (including Metformin) | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 18.0-95.0, Heart Rate Control in ICD Patients With Heart Failure Age ≥ 18 years. Patients with stable chronic heart failure implanted with mono-cameral or bicameral ICD with a home monitoring remote control. Moderate to severe left ventricular dysfunction (FE ≤ 40%). Any cause of heart failure was allowed apart congenital heart disease. Bicameral ICD programmed in DDD or AAI/DDD with AV interval < 300 msec. Rest ECG heart rate ≥70 bpm; Sinus rhythm. In therapy with low-dose of beta-blocker (bisoprolol 1,25-2,5 mg) and with the maximum dose tolerated of angiotensin-converting enzyme inhibitor or blockade of angiotensin II receptor, mineralocorticoid antagonist, antiplatelet and lipid-lowering therapy, unless contraindicated Inability of providing informed consent; Age < 18 years. State of pregnancy or lactation. Recent (<2 months) myocardial infarction; Contraindications to beta-blockers and ivabradine; Rest ECG heart rate < 70 bpm; No sinus rhythm. Administration of non-dihydropyridinic calcium channels antagonists, digitalis, class I antiarrhythmic drugs, strong inhibitors of cytochrome P450 3A4 at the time of enrollment | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 18.0-999.0, Heart Failure With Reduced Ejection Fraction Age ≥ 18 years; CHF with New York Heart Association class II-IV; left ventricular ejection fraction ≤ 35%; on beta blockers Potassium > 5.2 mmol/l; estimated glomerular filtration rate < 30 ml/min/1.73 m2; systolic blood pressure <100 mmHg or > 180 mmHg; history of intolerance to recommended target doses of angiotensin converting enzyme inhibitors or angiotensin receptor blockers Other protocol-defined inclusion/ | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 18.0-999.0, Myasthenia Gravis has the capacity to understand and sign an informed consent form years or older diagnosis of MG based on clinical features has detectable serum autoantibodies to AChR has a clinical indication for the use of TPE to treat MG unable or unwilling to comply with study procedures that multiple venipunctures weighs less than 50Kg has a contraindication to treatment with TPE (e.g. clinically significant bleeding disorder) has muscle specific tyrosine kinase or low-density lipoprotein receptor-related protein 4 (LRP4) antibody positive MG has prior or current history of thymoma had a thymectomy in the past 6 months has received rituximab in the past 12 months has another coexisting autoimmune disease that is not clinically controlled or may preclude accurate study assessments according to the judgment of the PI has current signs or symptoms of severe, progressive, or uncontrolled renal, hepatic, hematological, gastrointestinal, endocrine, pulmonary, cardiac, or central nervous system disease has participated in an interventional clinical trial with a novel therapeutic agent in the past 6 months | 1 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 18.0-100.0, All Diagnosed Health Conditions ADD/ADHD Alopecia Areata Ankylosing Spondylitis Asthma Atopic Dermatitis Eczema Beta Thalassemia Bipolar Disorder Breast Cancer Celiac Disease Cervical Cancer Chronic Inflammatory Demyelinating Polyneuropathy Chronic Kidney Diseases Chronic Obstructive Pulmonary Disease Colon Cancer Colorectal Cancer Crohn's Disease Cystic Fibrosis Depression Diabetes Mellitus Duchenne Muscular Dystrophy Endometriosis Epilepsy Facioscapulohumeral Muscular Dystrophy G6PD Deficiency General Anxiety Disorder Hepatitis B Hereditary Hemorrhagic Telangiectasia HIV/AIDS Human Papilloma Virus Huntington's Disease Idiopathic Thrombocytopenic Purpura Insomnia Kidney Cancer Leukemia Lung Cancer Lupus Nephritis Lymphoma Melanoma Multiple Myeloma Multiple Sclerosis Myositis Myotonic Dystrophy Ovarian Cancer Pancreatic Cancer Parkinson's Disease Polycystic Kidney Diseases Prostate Cancer Psoriasis Psoriatic Arthritis Rosacea Scleroderma Sickle Cell Anemia Sickle Cell Trait Sjogren's Syndrome Skin Cancer Spinal Muscular Atrophy Systemic Lupus Erythematosus Thrombotic Thrombocytopenic Purpura Trisomy 21 Ulcerative Colitis All adults 18-100 years old Able to provide proof of diagnosis Live in USA Younger than 18 years old Receipt of blood products within 30 days of study blood collection Receipt of investigational drug within 30 days of study blood collection | 1 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 0.0-120.0, Other Specified Inflammatory Disorders of Skin or Subcutaneous Tissue Pyoderma Gangrenosum Erosive Pustular Dermatosis of the Scalp Sweet's Syndrome Behcet's Disease Bowel-associated Dermatosis-arthritis Syndrome Pustular Psoriasis Acute Generalized Exanthematous Pustulosis Keratoderma Blenorrhagicum Sneddon-Wilkinson Disease IgA Pemphigus Amicrobial Pustulosis of the Folds Infantile Acropustulosis Transient Neonatal Pustulosis Neutrophilic Eccrine Hidradenitis Rheumatoid Neutrophilic Dermatitis Neutrophilic Urticaria Still's Disease Erythema Marginatum Unclassified Periodic Fever Syndromes / Autoinflammatory Syndromes Dermatitis Herpetiformis Linear IgA Bullous Dermatosis Bullous Systemic Lupus Erythematosus Inflammatory Epidermolysis Bullosa Aquisita Neutrophilic Dermatosis of the Dorsal Hands (Pustular Vasculitis) Small Vessel Vasculitis Including Urticarial Vasculitis Erythema Elevatum Diutinum Medium Vessel Vasculitis History of NMID or active disease Informed consent No consent to either part of the study | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 12.0-999.0, Metastatic Melanoma Stage III Cutaneous Melanoma AJCC v7 Stage IIIA Cutaneous Melanoma AJCC v7 Stage IIIB Cutaneous Melanoma AJCC v7 Stage IIIC Cutaneous Melanoma AJCC v7 Stage IV Cutaneous Melanoma AJCC v6 and v7 Patients must have metastatic melanoma or stage III in-transit, subcutaneous, or regional nodal disease (Turnstile I) Patients must have a lesion amenable to resection for the generation of TIL (Turnstile I) Patients must receive a magnetic resonance imaging (MRI)/computed tomography (CT)/positron emission tomography (PET) of the brain within 6 months of signing informed consent; if new lesions are present, patient must have definitive treatment; principal investigator (PI) or his designee should make final determination regarding enrollment (Turnstile I) Clinical performance status of Eastern Cooperative Oncology Group (ECOG) 0-2 within 30 days of signing informed consent (Turnstile I) Patients previously treated with immunotherapy, targeted therapy, or no therapy (treatment naive) will be eligible (Turnstile I) Patients receiving cytotoxic agents will be evaluated by the PI or his designee for suitability (Turnstile I) Patients with a negative pregnancy test (urine or serum) must be documented within 14 days of screening for women of childbearing potential (WOCBP); a WOCBP has not undergone a hysterectomy or who has not been naturally postmenopausal for at least 12 consecutive months (i.e. who has not had menses at any time in the preceding 12 consecutive months) (Turnstile I) Patients must have adequate TIL available (Turnstile II); pre-rapid expansion procedure (Pre-REP) TIL generated in the similar clinical trial 2004-0069 may also be utilized for Turnstile II Patients must have at least one biopsiable measurable metastatic melanoma, lesion > or = to 1 cm (Turnstile II) Patients may have brain lesions =< 1 cm each; the PI or designee will approve the treatment (Turnstile II) Active systemic infections requiring intravenous antibiotics, coagulation disorders or other major medical illnesses of the cardiovascular, respiratory or immune system; PI or his designee shall make the final determination regarding appropriateness of enrollment (Turnstile I) Primary immunodeficiency and need for chronic steroid therapy, however prednisone is allowed at < 10 mg/day (Turnstile I) Patients who are pregnant or nursing (Turnstile I) Presence of a significant psychiatric disease, which in the opinion of the principal investigator or his designee, would prevent adequate informed consent (Turnstile I) Has had prior systemic cancer therapy within the past four weeks or v-raf murine sarcoma viral oncogene homolog B (B-RAF) or mitogen-activated protein kinase (MEK) inhibitors within 7 days at the time of the start of the lymphodepletion regimen (Turnstile II) Women who are pregnant will be excluded because of the potentially dangerous effects of the preparative chemotherapy on the fetus (Turnstile II) Any active systemic infections requiring intravenous antibiotics, coagulation disorders or other major medical illnesses of the cardiovascular, respiratory or immune system, such as abnormal stress thallium or comparable test, myocardial infarction, cardiac arrhythmias, obstructive or restrictive pulmonary disease; PI or his designee shall make the final determination regarding appropriateness of enrollment (Turnstile II) Any form of primary or secondary immunodeficiency; must have recovered immune competence after chemotherapy or radiation therapy as evidenced by lymphocyte counts (> 500/mm^3), white blood cell (WBC) (> 3,000/mm^3) or absence of opportunistic infections (Turnstile II) Require steroid therapy or steroid-containing compounds, or have used systemic steroids in the past 4 weeks, or have used topical or inhalational steroids in the past 2 weeks prior to lymphodepletion; the exception being patients on chronic physiologic dose of steroid (Turnstile II) Presence of a significant psychiatric disease, which in the opinion of the principal investigator or his designee, would prevent adequate informed consent or render immunotherapy unsafe or contraindicated (Turnstile II) | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 55.0-85.0, Alzheimer's Disease Dementia Ages ≥55 and ≤85 years Informed consent form (ICF) signed by the subject or legally acceptable representative before any study-specific procedures for the subject are performed and an ICF signed by the support person/caregiver before any study-specific procedures for the support person/caregiver are performed Clinical diagnosis of dementia due to probable AD consistent with established by a workgroup of the National Institute on Aging and the Alzheimer's Disease Association Clinical decline within 12 months before screening and onset of symptoms at least 12 months or longer before screening, which may any documented cognition, functional, or other objective assessment or the clinical judgment of the investigator or the subject's referring physician that the subject has experienced a clinical decline within the last 12 months Magnetic resonance imaging (MRI) or computed tomography (CT) scan performed within 12 months before screening, with findings consistent with the diagnosis of dementia due to AD without any other clinically significant comorbid pathologies. If an MRI or CT scan is unavailable or occurred greater than 12 months before screening, this assessment should be completed and the findings confirmed before the subject enters the run-in period (Day -14) (copy of the report will be available at the study site) Mini-Mental State Examination (MMSE) score ≥14 and ≤24 at screening and confirmed on Day 1 prior to randomization (fluctuations of ±2 points are acceptable on Day 1/baseline) Clinical Dementia Rating Global score (CDR-GS) ≥1 (at least mild dementia) at screening and confirmed on Day 1 prior to randomization Modified Hachinski Ischemic Scale (mHIS) score ≤4 at screening Fertile, sexually active subjects (men and women) must use an effective method of contraception during the study. Female subjects and the female partner of male subjects must be surgically sterile (hysterectomy or bilateral tubal ligation), postmenopausal for at least 1-year, or willing to practice adequate methods of contraception if of childbearing potential (defined as consistent use of combined effective methods of contraception [including at least 1 barrier method]) Reliable and capable support person/caregiver, who if not living in the same household, interacts with the subject approximately 4 times per week and will be available to attend clinic visits in person when possible Exposure to an experimental drug, experimental biologic or experimental medical device within 2 months (60 days) before screening Prior participation in an amyloid vaccination clinical study at any time in the past or completion of a passive amyloid vaccination study within 6 months before screening Inability to swallow a tablet In the judgment of the investigator, inability of the subject or the support person/caregiver to complete a 26-week study Inability to be ≥75% compliant with single-blind study drug Inability to adequately cooperate or complete the cognitive testing procedures or any study assessment Residence in a skilled nursing facility Untreated vitamin B12 or folate deficiency (if treated, must be stably treated for at least 6 months before screening) Clinically significant (in the judgment of the investigator) abnormal serum electrolytes (sodium, potassium, magnesium) after repeat testing Clinically significant untreated hypothyroidism (if treated, thyroid-stimulating hormone level and thyroid supplementation dose must be stable for at least 6 months before screening) | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 55.0-85.0, Alzheimer's Disease Dementia Ages ≥55 and ≤85 years Informed consent form (ICF) signed by the subject or legally acceptable representative before any study-specific procedures for the subject are performed and an ICF signed by the support person/caregiver before any study-specific procedures for the support person/caregiver are performed Clinical diagnosis of dementia due to probable AD consistent with established by a workgroup of the National Institute on Aging and the Alzheimer's Disease Association Clinical decline within 12 months before screening and onset of symptoms at least 12 months or longer before screening, which may any documented cognition, functional, or other objective assessment or the clinical judgment of the investigator or the subject's referring physician that the subject has experienced a clinical decline within the last 12 months Magnetic resonance imaging (MRI) or computed tomography (CT) scan performed within 12 months before screening, with findings consistent with the diagnosis of dementia due to AD without any other clinically significant comorbid pathologies. If an MRI or CT scan is unavailable or occurred greater than 12 months before screening, this assessment should be completed and the findings confirmed before the subject enters the run-in period (Day -14) (copy of the report will be available at the study site) Mini-Mental State Examination (MMSE) score ≥14 and ≤24 at screening and confirmed on Day 1 prior to randomization (fluctuations of ±2 points are acceptable on Day 1/baseline) Clinical Dementia Rating Global score (CDR-GS) ≥1 (at least mild dementia) at screening and confirmed on Day 1 prior to randomization Modified Hachinski Ischemic Scale (mHIS) score ≤4 at screening Fertile, sexually active subjects (men and women) must use an effective method of contraception during the study. Female subjects and the female partner of male subjects must be surgically sterile (hysterectomy or bilateral tubal ligation), postmenopausal for at least 1-year, or willing to practice adequate methods of contraception if of childbearing potential (defined as consistent use of combined effective methods of contraception [including at least 1 barrier method]) Reliable and capable support person/caregiver, who if not living in the same household, interacts with the subject approximately 4 times per week and will be available to attend clinic visits in person when possible Exposure to an experimental drug, experimental biologic or experimental medical device within 2 months (60 days) before screening Prior participation in an amyloid vaccination clinical study at any time in the past or completion of a passive amyloid vaccination study within 6 months before screening Inability to swallow a tablet In the judgment of the investigator, inability of the subject or the support person/caregiver to complete a 26-week study Inability to be ≥75% compliant with single-blind study drug Inability to adequately cooperate or complete the cognitive testing procedures or any study assessment Residence in a skilled nursing facility Untreated vitamin B12 or folate deficiency (if treated, must be stably treated for at least 6 months before screening) Clinically significant (in the judgment of the investigator) abnormal serum electrolytes (sodium, potassium, magnesium) after repeat testing Clinically significant untreated hypothyroidism (if treated, thyroid-stimulating hormone level and thyroid supplementation dose must be stable for at least 6 months before screening) | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 40.0-65.0, Adult Lymphoblastic Lymphoma Disease ALL in complete remission (CR) at the time of transplant. Remission is defined as "less than 5.0% bone marrow lymphoblasts by morphology," as determined by a bone marrow aspirate obtained within 2 weeks of study registration Philadelphia chromosome positive ALL is allowed Lymphoid blastic crisis of CML will be included (provided that patients achieve CR) Age Equal or above age 40 and up to 65 years. If younger than 40, there must be comorbidities which preclude the patient to undergo CyTBI conditioning regimen Organ Function All organ function testing should be done within 28 days of study registration Cardiac: Left ventricular ejection fraction (LVEF) ≥ 50% by MUGA (Multi Gated Acquisition) scan or echocardiogram Pulmonary: FEV1 (Forced expiratory volume in 1 second) and FVC (Forced vital capacity) ≥ 50% predicted, DLCO (alveolar diffusion capacity for carbon monoxide) (corrected for hemoglobin) ≥ 50% of predicted Renal: The estimated creatinine clearance (CrCl) must be equal or greater than 60 mL/min/1.73 m2 as calculated by the Cockcroft-Gault Formula: CrCl = (140-age) x weight (kg) x 0.85 (if female)/72 x serum creatinine (mg/dL) Hepatic Non-compliant to medications No appropriate caregivers identified HIV1 (Human Immunodeficiency Virus-1) or HIV2 positive Active life-threatening cancer requiring treatment other than ALL Uncontrolled medical or psychiatric disorders Uncontrolled infections, defined as positive blood cultures within 72 hours of study entry, or evidence of progressive infection by imaging studies such as chest CT scan within 14 days of registration Active central nervous system (CNS) leukemia Preceding allogeneic HSCT Receiving intensive chemotherapy within 21 days of registration. Maintenance type of chemotherapy will be allowed | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 7.0-999.0, Type 1 Diabetes Type 2 Diabetes for A subject is eligible for the user evaluation study if all of the following are met: 1. Subject is 7 years or older at time of screening 2. Subject is current insulin pump user for at least 3 months 3. Subject has the following CGM experience as determined by the Investigator Has experience and is able to insert/change sensor by herself/himself and Has experience and can recharge the transmitter and Has experience and can read sensor data in real-time on her/his pump screen 4. Subject/legal representative has signed a Patient Informed Consent and is willing to comply with the study procedures; 5. Subject is willing to complete study questionnaires throughout the study 6. Must have the following clinical diagnosis: 1. Type 1 diabetes, for a minimum of 6 months prior to enrollment for A subject is excluded from the user evaluation if any of the following are met: 1. Female subject has a positive urine pregnancy screening test. 2. Female subject who plans to become pregnant during the course of study. If a woman becomes pregnant during participation, she will be withdrawn 3. Subject has any condition that, in the opinion of the Investigator or qualified Investigational Centre staff, may preclude him/her from participating in the study and completing study related procedures. 4. Subject has impaired vision or hearing problems that could compromise the handling of the device as determined by Investigator or qualified Investigational Centre staff 5. Subject is unable to tolerate tape adhesive in the area of sensor placement 6. Subject has any unresolved adverse skin condition in the area of sensor placement (e.g. psoriasis, rash, Staphylococcus infection) 7. Subject has travel plans which would make it difficult for the subject to attend on-site study visits as scheduled 8. Subject is actively participating in an investigational study (drug or device) wherein he/she has received treatment from an investigational study drug or investigational study device in the last 2 weeks (CEP267 User Evaluation is not included in this | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 18.0-999.0, Breast Cancer Nos Metastatic Recurrent Women Aged 18 years and over With an invasive breast cancer diagnosed by cytology or histology Tumors cT0 to cT3, CN0-3 No clinical evidence of metastasis at the time of Untreated including scored for breast cancer surgery in progress Patient receiving a social security system Patient mastering the French language Free and informed consent for additional biological samples, different questionnaires and collecting information on resource usage Metastatic breast cancer Local recurrence of breast cancer History of cancer within 5 years prior to entry into the trial other than basal cell skin or carcinoma in situ of the cervix Already received treatment for breast cancer ongoing Blood transfusion performed for less than six months Persons deprived of liberty or under supervision (including guardianship) | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 18.0-70.0, Metastatic Melanoma Measurable metastatic melanoma with at least one lesion that is resectable for TIL generation and at least one other lesion that can be measured by criteria. 2. Confirmation of diagnosis of metastatic melanoma by the Laboratory of Pathology of NCI. 3. Patients with 3 or fewer brain metastases that are less than 1 cm in diameter and asymptomatic are eligible. Lesions that have been treated with stereotactic radiosurgery must be clinically stable for 1 month after treatment for the patient to be eligible. Patients with surgically resected brain metastases are eligible. 4. Greater than or equal to 18 years of age and less than or equal to 70 years of age. 5. Ability of subject to understand and the willingness to sign the Informed Consent Document 6. Willing to sign a durable power of attorney. 7. Clinical performance status of ECOG 0, 1 or 2. 8. Patients of both genders must be willing to practice birth control from the time of enrollment on this study and for up to four months after treatment. 9. Serology Seronegative for HIV antibody. (The experimental treatment being evaluated in this protocol depends on an intact immune system. Patients who are HIV seropositive can have decreased immune-competence and thus are less responsive to the experimental treatment and more susceptible to its toxicities.) Seronegative for hepatitis B antigen, and seronegative for hepatitis C antibody. If hepatitis C antibody test is positive, then patient must be tested for the presence of antigen by RT-PCR and be HCV RNA negative. 10. Women of child-bearing potential must have a negative pregnancy test because of the potentially dangerous effects of the treatment on the fetus. 11. Hematology Absolute neutrophil count greater than 1000/mm(3) without the support of filgrastim WBC greater than or equal to 3000/mm(3) Platelet count greater than or equal to 100,000/mm(3) Hemoglobin > 8.0 g/dl 12. Chemistry Serum ALT/AST less than or equal to 2.5 times the upper limit of normal Serum Creatinine less than or equal to 1.6 mg/dl Total bilirubin less than or equal to 1.5 mg/dl, except in patients with Gilbert s Syndrome who must have a total bilirubin less than 3.0 mg/dl. 13. More than four weeks must have elapsed since any prior systemic therapy at the time the patient receives the preparative regimen, and patients toxicities must have recovered to a grade 1 or less (except for toxicities such as alopecia or vitiligo). Patients must have progressive disease after prior treatment. Note: Patients may have undergone minor surgical procedures within the past 3 weeks, as long as all toxicities have recovered to grade 1 or less. 14. Subjects must be co-enrolled in 03-C-0277 Women of child-bearing potential who are pregnant or breastfeeding because of the potentially dangerous effects of the treatment on the fetus or infant. 2. Any form of primary immunodeficiency (such as Severe Combined Immunodeficiency Disease). 3. Concurrent opportunistic infections (The experimental treatment being evaluated in this protocol depends on an intact immune system. Patients who have decreased immune competence may be less responsive to the experimental treatment and more susceptible to its toxicities). 4. Active systemic infections, (e.g.: requiring anti-infective treatment), coagulation disorders or any other active major medical illnesses. 5. Concurrent systemic steroid therapy. 6. History of severe immediate hypersensitivity reaction to any of the agents used in this study. 7. History of coronary revascularization or ischemic symptoms. 8. Any patient known to have an LVEF less than or equal to 45% 9. Documented LVEF of less than or equal to 45%, note: testing is required in patients with Age greater than or equal to 65 years old Clinically significant atrial and or ventricular arrhythmias including but not limited to: atrial fibrillation, ventricular tachycardia, second or third degree heart block or history of ischemic heart disease or chest pain. 10. Patients who are receiving other investigational agents 11. Documented FEV1 less than or equal to 60% predicted tested in patients with A prolonged history of cigarette smoking (20 pk/year of smoking within the past 2 years) Symptoms of respiratory dysfunction | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 18.0-999.0, Thyroid Cancer Newly diagnosed with a first occurrence of thyroid cancer <2-4 weeks of diagnosis (i.e., histologically confirmed thyroid cancer (papillary, follicular, or medullary type; TNM classification system) Willing to participate in the EG meetings >18 years Alert and capable of giving free and informed consent Able to speak and read English or French Anaplastic thyroid cancer Karnofsky Performance Status (KPS) score <60 (rated by the Research Coordinator (RC) or referring physician) or expected survival <6 months according to clinical judgment | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 18.0-65.0, Chronic Pain Women Clinical diagnosis of chronic pelvic pain More than eighteen years Non-menstrual or noncyclic pelvic pain Duration of pain of at least 6 months Duration of pain less than 6 months Women who were pregnant in the last 12 months | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 18.0-999.0, Coronary Artery Stenosis Age ≥ 18 years Patient with an indication for PCI including angina (stable or unstable), silent ischemia (in absence of symptoms a visually estimated target lesion diameter stenosis of ≥70%, a positive non-invasive stress test, or FFR ≤0.80 must be present), or recent STEMI. For STEMI the time of presentation to the first treating hospital, whether a transfer facility or the study hospital, must be >24 hours prior to randomization and enzyme levels (CK-MB or Troponin) demonstrating that either or both enzyme levels have peaked Non-target vessel PCI are allowed prior to randomization depending on the time interval and conditions as follows: a. During Baseline Procedure: i. PCI of non-target vessels performed during the baseline procedure itself immediately prior to randomization if successful and uncomplicated defined as: <50% visually estimated residual diameter stenosis, TIMI Grade 3 flow, no dissection ≥ NHLBI type C, no perforation, no persistent ST segment changes, no prolonged chest pain, no TIMI major or BARC type 3 bleeding. b. Less than 24 hours prior to Baseline Procedure: i. Not allowed (see #3). c. 24 hours-30 days prior to Baseline Procedure: i. PCI of non-target vessels 24 hours to 30 days prior to randomization if successful and uncomplicated as defined above. ii. In addition, in cases where non-target lesion PCI has occurred 24-72 hours prior to the baseline procedure, at least 2 sets of cardiac biomarkers must be drawn at least 6 and 12 hours after the non-target vessel PCI. If cardiac biomarkers are initially elevated above the local laboratory upper limit of normal, serial measurements must demonstrate that the biomarkers are falling. d. Over 30 days prior to Baseline Procedure: iii. PCI of non-target vessels performed greater than 30 days prior to procedure whether or not successful and uncomplicated Patient or legal guardian is willing and able to provide informed written consent and comply with follow-up visits and testing schedule. Angiographic (visual estimate) Treatment of up to three de novo target lesions, maximum of one de novo target lesion per vessel Target lesion(s) must be located in a native coronary artery with visually estimated diameter of ≥2.5 mm to ≤4.25 mm and diameter stenosis ≥50% to <100% Lesion must be ≤28 mm long and can be covered by a single study stent with maximum length of 33 mm (note: multiple focal stenoses may be considered as a single lesion and be enrolled if they can be completely covered with one stent) TIMI flow 2 or 3 If more than one target lesion will be treated, the RVD and lesion length of each must meet the above criteria Planned procedures after the baseline procedure in either the target or non-target vessels STEMI within 24 hours of initial time of presentation to the first treating hospital, whether at a transfer facility or the study hospital or in whom enzyme levels (either CK-MB or Troponin)have not peaked PCI within the 24 hours preceding the baseline procedure and randomization Non-target lesion PCI in the target vessel within 12 months of the baseline procedure History of stent thrombosis Cardiogenic shock (defined as persistent hypotension (systolic blood pressure <90 mm/Hg for more than 30 minutes) or requiring pressors or hemodynamic support, including IABP Known LVEF <30% Subject is intubated Relative or absolute contraindication to DAPT for 12 months (including planned surgeries that cannot be delayed, or subject is indicated for chronic oral anticoagulant treatment) Hemoglobin <10 g/dL | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 18.0-999.0, Refractory Generalized Myasthenia Gravis Key Male or female patients ≥18 years old Diagnosis of MG made by the following tests: 1. Positive serologic test for anti-AChR Abs as confirmed at screening, and 2. One of the following: 1. History of abnormal neuromuscular transmission test demonstrated by single-fiber electromyography (SFEMG) or repetitive nerve stimulation, or 2. History of positive anticholinesterase test, e.g. edrophonium chloride test, or 3. Subject has demonstrated improvement in MG signs on oral cholinesterase inhibitors, as assessed by the treating physician MGFA Clinical Classification Class II to IV at screening MG-ADL total score must be ≥6 at screening and Randomization (Day 1) Subjects who have: 1. Failed treatment with at least two immunosuppressive agents. Or, 2. Failed treatment with at least one immunosuppressive agent and require chronic plasma exchange or IVIg Key History of thymoma or other neoplasms of the thymus History of thymectomy within 12 months prior to screening MGFA Class I or MG crisis at screening (MGFA Class V) Use of rituximab within 6 months prior to screening Use of IVIg or PE within 4 weeks prior to Randomization (Day 1) | 1 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 13.0-999.0, Preeclampsia Eclampsia HELLP Syndrome Toxemia Hypertensive Disorder of Pregnancy Women who were medically diagnosed with preeclampsia or a related hypertensive disorder of pregnancy (HDP) such as eclampsia or HELLP syndrome Female or Male relatives of affected women Women who have never had preeclampsia or hypertensive disorders of pregnancy, but who would like to serve as "controls" in research studies of preeclampsia An authorized representative may complete the questionnaire and provide consent for a woman who had preeclampsia, but has died or is disabled as a result Those unable to provide consent Prisoners Individuals unable to speak English | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 14.0-65.0, Dental Caries Two class V carious lesions which do not require an invasive treatment Size and form of the lesions: the lesions must both be fully visible and assessable and accessible Patients must be able and willing to observe good oral hygiene throughout the study -≥ 20 teeth Permanent dentition and ≤ 65 years Willing and able to attend the on-study visits Willing and able to understand all study-related procedures Written informed consent before participation in the study Negative pregnancy test for women of childbearing potential The two study lesions must not be on adjacent teeth No adjacent restoration on study tooth surface Fluoride varnish application < 6 months prior to study treatment Patient suffers from diabetes Evidence of tooth erosion (due to excessive acidic drink consumption or reflux) History of head and neck illnesses (e.g. head/neck cancer) Any pathology or concomitant medication affecting salivary flow or dry mouth Any metabolic disorders affecting bone turnover Concurrent participation in another clinical trial with an investigational drug or device or participation in another clinical trial with an investigational drug or device within 30 days prior to study entry Pregnant and lactating woman | 2 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 8.0-999.0, Diet Modification Chronic Urticaria Urticaria +/ angioedema present for a minimum of 6 weeks Agree to be on anti-histamine prn Age older than 8 years old, with no upper age limit Causes such as parasite infestation, microbial and viral infections, autoimmune disease, or other pathology that could account for the U/A/P should be excluded Skin prick test and/or radioallergosorbent (RAST) test are negative for all food allergens tested, or patient had achieved no symptomatic improvement after strict avoidance of all skin test-positive foods Pregnant or breast feeding women Patients who is taking central nervous system (CNS) acting agents (including tranquilizers, antidepressants, sedatives, hypnotics or antiepileptics) at any time | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 0.0-999.0, Colorectal Cancer Screening centres participating in the Polish Colonoscopy Screening Platform (PCSP) between 2012 and 2013, in which no routine video recording of screening colonoscopies is performed Screening centres which will sign informed consent | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 2.0-80.0, Brain Disorders Birth Defects Craniofacial Differences Subject is 2-80 years, any gender, race or ethnic group, inclusive. 2. Subject has a diagnosis of congenital facial palsy, isolated or combined with other congenital anomalies, based on MPIs review of prior medical records and interview with patient and/or patient physicians. 3. Subject is a family member of a patient with a diagnosis of congenital facial palsy, isolated or combined with other congenital anomalies. 4. Subject has the ability to travel to the NIH Clinical Center for admissions. 5. Subject or subject s legal guardian is able to provide written informed consent Subject has severe respiratory difficulties (i.e., requiring a tracheostomy or other assistive device to maintain respiration) or other disease manifestation that would interfere with the ability to comply with the requirements of this protocol and/or pose a severe anesthesia risk. 2. Subject has a psychiatric illness or neurological disease that would interfere with the ability to comply with the requirements of this protocol. This includes, but is not limited to, uncontrolled/untreated psychotic depression, bipolar disorder, schizophrenia, substance abuse or dependence, antisocial personality disorder, or panic disorder. 3. Subject shows evidence of clinically significant cardiovascular, pulmonary, hepatic, renal, hematological, metabolic, or gastrointestinal disease, or has a condition that requires immediate surgical intervention. 4. Subject is pregnant during the study. 5. Subject or subject s legal guardian is unable or unwilling to provide consent or assent. 6. The principal investigator may decline to enroll a patient for other reasons | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 18.0-999.0, Metastatic Malignant Neoplasm in the Brain Recurrent Non-Small Cell Lung Carcinoma Stage IIA Non-Small Cell Lung Carcinoma Stage IIB Non-Small Cell Lung Carcinoma Stage IIIA Non-Small Cell Lung Cancer Stage IIIB Non-Small Cell Lung Cancer Stage IV Non-Small Cell Lung Cancer Pathologically proven (either histologic or cytologic) diagnosis of NSCLC with any of the following stages (according to the American Joint Committee on Cancer [AJCC] Staging Manual, 7th edition) Stage IIIA or IIIB Stage II NSCLC with medical contraindication to curative surgical resection Stage IV disease with solitary brain metastasis that has been treated radically (eg: with surgical resection or stereotactic radiosurgery) and thoracic disease that would be classified as stage II-III Appropriate diagnostic/staging workup, including Complete history and physical examination Whole body PET/computed tomography (CT) scan within 42 days prior to study entry demonstrating hypermetabolic pulmonary lesion(s) and/or thoracic lymph node(s), with a maximum standardized uptake volume (SUV) > 6 for at least one lesion; if PET/CT was obtained more than 42 days prior to study entry and is not repeated, CT scan of the chest within 28 days prior to study entry demonstrating stable disease is required Magnetic resonance imaging (MRI) of the brain or CT scan of the head with contrast within 42 days prior to study entry Biopsy confirmation of suspected metastatic disease identified by PET/CT is recommended Pulmonary function tests (PFTs) within 6 weeks of study entry are highly recommended but not required Pleural or pericardial effusion A patient with pleural effusion may be enrolled the effusion is sampled by thoracentesis and cytology is negative or the effusion is seen on axial imaging but not on chest x-ray and deemed too small to tap under CT or ultrasound guidance Prisoners or subjects who are compulsorily detained (involuntarily incarcerated) for treatment of either a psychiatric or physical (e.g., infectious) illness Women who Are unwilling or unable to use an acceptable method to avoid pregnancy for the entire study period and for at least 4 weeks after cessation of study therapy Have a positive pregnancy test at baseline Are pregnant or breastfeeding Poorly controlled diabetes (defined as fasting glucose level > 200 mg/dL) despite attempts to improve glucose control by fasting duration and adjustment of medications; patients with diabetes will preferably be scheduled for PET/CT imaging in the morning, and instructions for fasting and use of medications will be provided in consultation with the patients' primary physicians | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 16.0-999.0, Thermography Laser Doppler Blood Flow Spectrophotometric Intracutaneous Analysis adults burn injury within 72 hours of presentation able to undergo all three imaging modalities children patients not consenting | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 18.0-999.0, Relapsed or Refractory Acute Myeloid Leukemia (AML) Untreated AML Other IDH1-mutated Positive Hematologic Malignancies Myelodysplastic Syndromes Key Subject must be ≥18 years of age Subjects must have documented IDH1 R132 gene-mutated advanced hematologic malignancy based on local or central evaluation Subjects must be amenable to serial bone marrow biopsies, peripheral blood sampling, and urine sampling during the study Subjects must have ECOG PS of 0 to 2 Platelet count ≥20,000/µL (Transfusions to achieve this level are allowed) Subjects must have adequate hepatic function as evidenced by: Aspartate aminotransferase (AST), alanine aminotransferase (ALT), and alkaline phosphatase (ALP) ≤3.0 × ULN, unless considered due to leukemic disease and serum total bilirubin ≤1.5 x upper limit of normal (ULN), unless considered due to Gilbert's disease or leukemic disease Subjects must have adequate renal function as evidenced by a serum creatinine ≤2.0 × ULN or creatinine clearance >40mL/min based on Cockroft-Gault glomerular filtration rate (GFR) Subjects must be recovered from any clinically relevant toxic effects of any prior surgery, radiotherapy, or other therapy intended for the treatment of cancer Female subjects with reproductive potential must have a negative serum pregnancy test within 7 days prior to the start of therapy and on the first day of study drug administration. Key Subjects who have undergone hematopoietic stem cell transplant (HSCT) within 60 days of the first dose of AG-120, or subjects on immunosuppressive therapy post HSCT at the time of screening, or with clinically significant graft-versus-host disease (GVHD). (The use of a stable dose of oral steroids post HSCT and/or topical for ongoing skin GVHD is permitted.) Subjects who received systemic anticancer therapy or radiotherapy <14 days prior to their first day of study drug administration. (Hydroxyurea is allowed prior to enrollment and after the start of AG-120) Subjects who received an investigational agent <14 days prior to their first day of study drug administration Subjects who are pregnant or breastfeeding Subjects with an active severe infection or with an unexplained fever >38.5°C during screening visits or on their first day of study drug administration (at the discretion of the Investigator, subjects with tumor fever may be enrolled) Subjects with New York Heart Association (NYHA) Class III or IV congestive heart failure or LVEF <40% by echocardiogram (ECHO) or multi-gated acquisition (MUGA) scan within approximately 28 days of C1D1 Subjects with a history of myocardial infarction within the last 6 months of screening Subjects with a known unstable or uncontrolled angina pectoris Subjects with a known history of severe and/or uncontrolled ventricular arrhythmias Subjects with known unstable or uncontrolled angina pectoris | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 0.0-999.0, Recurrent Neuroblastoma Neuroblastoma diagnosis of neuroblastoma either by histologic verification and/or demonstration of tumor cells in the bone marrow with increased urinary catecholamines must have high risk neuroblastoma with one of the following: 1) recurrent/progressive disease at any time, 2) refractory disease, 3) persistent disease after at least a partial response to frontline therapy, or 4) Second or greater complete remission after definitive disease progression must have at least one of the following sites of disease: 1) measurable tumor on MRI, CT scan, or X-Ray; 2)MIBG scan with positive uptake in at least one site; 3) bone marrow with tumor cells seen on routine morphology must have an ECOG performance status of 0, 1, or 2 must have a life expectancy of greater than or equal to 8 weeks must have fully recovered from the acute toxic effects of all prior chemotherapy, immunotherapy, or radiotherapy hemoglobin greater than or equal to 8.0 (may transfuse to achieve this level) ANC greater than or equal to 500 (must be at least 7 days after last dose of growth factor) platelet count greater than or equal to 50,000 (must be transfusion independent, defined as at least 1 week since last platelet transfusion) age-adjusted serum creatinine less than or equal to 1.5 times normal for age Pregnancy or breast feeding. Due to the potential teratogenic effects of retinoids, pregnant women are NOT eligible. Breast milk feeding by study patient is NOT allowed Patients with history of organ and allogeneic stem cell transplantation Patients with a known history of allergy to soy products Patients with a known history of a severe allergy or sensitivity of wheat gluten Patients requiring anti-arrhythmia cardiac medications are NOT eligible Prior therapy with fenretinide, or fenretinide + ketoconazole, if DLT's were experienced A known history of intolerance of ketoconazole Patients on other essential medications for which an interaction with ketoconazole can be expected and for which dose reductions to other essential medications cannot be made in a manner adequate to ensure patient safety Patients who, in the opinion of the investigator, may not be able to comply with safety monitoring requirements of the study Active hepatitis | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 18.0-999.0, Advanced Solid Tumors Amenable to Anthracycline Therapy Sarcoma, Breast Cancer, Lung Carcinomas, and Gynecological Cancer Amenable to Anthracycline Therapy Patients with histologically or cytologically confirmed advanced solid cancer deemed suitable for combination therapy of L19TNFα and doxorubicin Patients aged ≥18 years old with advanced or metastatic solid tumor in whom standard anticancer therapies have been exhausted and who are amenable for a doxorubicin monotherapy according to the discretion of the Principal Investigators and previously treated with a cumulative dose of anthracyclines (≤ 300 mg/m2) Eastern cooperative oncology group (ECOG) performance status ≤ 2 Patients may have received previous chemotherapy (>4 weeks prior therapy) or radiation therapy (>6 weeks prior therapy), but they must be amenable for doxorubicin treatment according to the discretion of the Principal Investigator Patients must have at least one unidimensionally measurable lesion by computed tomography as defined by version 1.1. This lesion must not have been irradiated during previous treatments Previous anthracycline therapy, including liposomal doxorubicin, for metastatic and/or adjuvant disease is allowed. However, patients must not have received a cumulative anthracycline dose of more than 300 mg/m2 of doxorubicin, nor more than 500 mg/m2 of epirubicin, nor more than 600 mg/m2 of pegylated or non-pegylated liposomal doxorubicin prior to study entry, in order to avoid anthracycline-associated cardiotoxicity Life expectancy more than 3 months Absolute neutrophil count (ANC) ≥ 1.5 x 109/L, platelets ≥ 100 x 109/L and haemoglobin (Hb) ≥ 9.5 g/dl All acute adverse effects (excluding alopecia) of any prior therapy (including surgery, radiation therapy, chemotherapy) must have resolved to ≤ Grade 1, except elevated liver transaminases judged to be associated with tumor infiltration (see below) (graded according to the National Cancer Institute CTCAE v.4.02 dated September 15, 2009) Alkaline phosphatase (AP), alanine aminotransferase (ALT) and/or aspartate aminotransferase (AST) ≤ 2.5 x upper limit of normal (ULN), and total bilirubin ≤ 2.0 mg/dL, unless liver involvement by the tumor, in which case the transaminase levels up to 5 x ULN are allowed Pregnancy or breast-feeding. Patients must agree to use effective contraception, or be surgically sterile or postmenopausal. The definition of effective contraception will be based on the guidelines ICH M3 rev2 Presence of active infections (e.g. requiring antimicrobial therapy) or other severe concurrent disease, which, in the opinion of the investigator, would place the subject at undue risk or interfere with the study Presence of known brain metastases. If patient is symptomatic, negative CT scan within two months before study beginning is required. However, presence of controlled brain metastases (i.e., evaluated as SD of PR after radiotherapy) is allowed Known cancer of other primary origin within the prior 5 years History of chronic hepatitis B or C, or chronic active hepatitis or active autoimmune diseases Cardiac disease as manifested by any of the following > Grade II heart failure, graded per New York Heart Association (NYHA) criteria History within the last year of acute or subacute coronary syndromes including myocardial infarction, unstable or severe stable angina pectoris Irreversible cardiac arrhythmias requiring permanent medication Ejection fraction less than the institutional lower limit of normal as assessed by MUGA scan or echocardiogram | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 0.0-30.0, Relapsed Pediatric Solid Tumors Refractory Pediatric Solid Tumors Rhabdomyosarcoma Ewing Sarcoma Osteosarcoma Neuroblastoma Wilms Tumor Hepatic Tumor Germ Cell Tumor Desmoid Tumor AGE: ≤ 30 years of age. Histologically confirmed malignant solid tumors, which may but are not limited to rhabdomyosarcoma and other soft tissue sarcomas, Ewing's sarcoma family of tumors, osteosarcoma, neuroblastoma, Wilms' tumor, hepatic tumors, germ cell tumors, and desmoid tumors. TUMOR Target lesion(s) must be located in bone or soft tissue in close proximity to bone. Target lesions must be reachable within the normal safety margins of HIFU as specified in the instructions for use. Radiographically evaluable or measurable solid tumor target lesion(s). Malignant Tumor: The patient's cancer must have relapsed after or failed to respond to frontline curative therapy and there must not be other potentially curative treatment options available. Curative therapy may surgery, radiation therapy, chemotherapy, or any combination of these modalities. PRIOR Patients must have fully recovered from the acute toxic effects of all prior chemotherapy, immunotherapy, or radiotherapy prior to entering on this study. No limitation on the number of prior chemotherapy regimens that the patient may have received prior to study entry. Myelosuppressive chemotherapy: The last dose of all myelosuppressive anticancer drugs must be at least 3 weeks prior to study entry. Immunotherapy: The last dose of immunotherapy (monoclonal antibody or vaccine) must be at least 4 weeks prior to study entry. Biologic (anti-cancer agent): The last dose of all biologic agents for the treatment of the patient's cancer (such as retinoids or tyrosine kinase inhibitors) must be at least 7 days prior to study entry. Radiation therapy: The last dose of radiation to more than 25 % of marrow containing bones (pelvis, spine, skull) must be at least 4 weeks prior to study entry. The last dose of all other local palliative (limited port) radiation must be at least 2 weeks prior to study entry. Stem Cell Transplantation. At least 2 months post-autologous stem cell transplant or at least 3 months post-allogeneic transplant and recovered from toxicities without evidence of graft versus host disease and on stable doses of immunosuppressive medications if required. Growth Factors. The last dose of colony stimulating factors, such as filgrastim, sargramostim, and erythropoietin, must be at least 1 week prior to study entry, the last dose of long-acting colony stimulating factors, such as pegfilgrastim, must be at least 2 weeks prior to study entry. No other anti-cancer therapy (chemotherapy, biological therapy, radiation therapy) is permitted during HIFU treatment and post treatment follow up for tolerability (see section 3.3). Patients > 16 years old must have a Karnofsky performance level ≥ 50%, and children ≤ 16 years old must have a Lansky performance level ≥ 50% (See Appendix I). Patients who are unable to walk because of paralysis or motor weakness, but who are up in a wheelchair will be considered ambulatory for the purpose of calculating the performance score Peripheral absolute neutrophil count (ANC) of ≥750/µL 2. Platelet count ≥75,000/µL (may receive transfusions) RENAL Age-adjusted normal serum creatinine OR a creatinine clearance ≥60 mL/min/1.73 m2 Defined as no dyspnea at rest, and a pulse oximetry >94% on room air if there is clinical indication for determination Normal PT, PTT and INR < 1.5 x ULN (including patients on prophylactic anticoagulation) Clinically significant unrelated systemic illness, such as serious infections, hepatic, renal or other organ dysfunction, which in the judgment of the Principal or Associate Investigator would compromise the patient's ability to tolerate the general anesthetic required for the procedure. Implant or prosthesis or scar tissue within the path of the HIFU beam. Target <1 cm from nerve plexus, spinal canal, bladder, bowel Target in contact with hollow viscera Lesion in the skull Inability to undergo MRI and/or contraindication for MRI Inability to tolerate stationary position during HIFU Patients currently receiving other anticancer agents. Patients currently receiving other investigational agents | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 18.0-999.0, Carcinoma, Non-small Cell Lung For all Parts: The participant must have stage IV non-small cell lung cancer (NSCLC) For Part A (abemaciclib + pemetrexed): Non-squamous subtypes only. The participant must have received at least one but no more than three prior therapies for advanced/metastatic NSCLC For Part B (abemaciclib + gemcitabine): Any subtype. The participant must have received at least one but not more than three prior therapies for advanced/metastatic NSCLC For Part C (abemaciclib + ramucirumab): Any subtype. The participant must have received at least two but not more than three prior therapies for advanced/metastatic NSCLC For Part D (abemaciclib + LY3023414): Any subtype. The participant must have received at least two, but not more than three prior therapies for advanced/metastatic NSCLC. The participant must not have received prior treatment with any phosphoinositide 3-kinase (PI3K) or mammalian target of rapamycin (mTOR) inhibitor For Part E (abemaciclib + pembrolizumab): Any subtype. The participant must have received at least one but no more than three prior therapies for advanced/metastatic NSCLC Have either measureable or nonmeasurable disease as defined by the Response Evaluation in Solid Tumors (RECIST v1.1) Have adequate organ function including Hematologic: Absolute neutrophil count (ANC) 1.5 x 109/liter (L), platelets 100 x 109/L, and hemoglobin 8 gram/deciliter (g/dL) Hepatic: Bilirubin 1.5 times upper limits of normal (ULN), alanine aminotransferase (ALT) and aspartate transaminase (AST) 3.0 times ULN. For participants with tumor involvement of the liver, AST and ALT equaling ≤5.0 times ULN are acceptable. Alkaline phosphatase ≤5.0 times ULN for participants with tumor involvement of the bone is acceptable Have a personal history of any of the following conditions: presyncope or syncope of either unexplained or cardiovascular etiology, ventricular arrhythmia (including but not limited to ventricular tachycardia and ventricular fibrillation), or sudden cardiac arrest. Exception: Participants with controlled atrial fibrillation for >30 days prior to study treatment are eligible Parts A, B, D and E: Have central nervous system (CNS) metastasis with development of associated neurological changes 14 days prior to receiving study drug Have a history of any other cancer (except non-melanoma skin cancer or carcinoma in-situ of the cervix or breast), unless in complete remission with no therapy for a minimum of 3 years Is pregnant or breastfeeding, or expecting to conceive or father children within the projected duration of the trial, starting with the pre-screening or screening visit through 3 to 4 months after the last dose of trial treatment (as appropriate) Have active bacterial, fungal, and/or known viral infection (for example, human immunodeficiency virus [HIV] antibodies, hepatitis B surface antigen [HBSAg], or hepatitis C antibodies). Screening is not required for enrollment Parts A, B, C, and E: Have QTc interval of > 470 millisecond (msec) on screening electrocardiogram (ECG). Part D participants have QTc interval of >450msec on screening ECG. Additional For Part C History or evidence of cardiovascular risk including any of the following History of acute coronary syndromes (including myocardial infarction and angina), coronary angioplasty, or stenting within 6 months prior to enrollment History or evidence of current ≥Class II congestive heart failure as defined by New York Heart Association | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 21.0-90.0, Myasthenia Gravis Subjects 21 to 90 years old 2. Subjects must have generalized MG, defined as MGFA clinical classification grades 2 (mild), 3 (moderate), or 4 (severe, but not intubated) at the time of screening/randomization. 3. Elevated AChR antibody titer 4. Subject's signs and symptoms should not be better explained by another disease process. 5. Subjects must be on a stable standard immunosuppressive regimen: 1. Prednisone only: Prednisone dose must be at least 15mg/day (or the equivalent on alternate days), and the dose of prednisone must have been stable for at least 4 weeks (28 days) prior to the baseline visit. 2. Prednisone plus another immunosuppressive therapy (IST). Immunosuppressive therapies other than prednisone, specifically azathioprine, mycophenolate mofetil, cyclosporine, tacrolimus or methotrexate, are permitted, but the dose must have been stable for at least 6 months prior to the baseline visit. (Note: The prednisone dose must be stable as defined in the prednisone only group. The IST dose must remain stable throughout the course of the study). 6. Subjects must be willing to complete the study and return for follow-up visits. 7. No history of thymoma, tumor, infection, or interstitial lung disease on chest CT, MRI, or chest x-ray. Note: Chest x-ray will be completed at screening to look of interstitial lung disease. A chest CT or MRI to evaluate for thymoma must be completed as part of prescreening. 8. Able and willing to give written informed consent and comply with the requirements of the study protocol. 9. Subjects must be able to give written informed consent before participating in this study. A copy of the signed consent must be kept in the subject's medical record. 10. Men and women of reproductive potential must agree to use an acceptable method of birth control during treatment and for twelve months (1 year) after completion of treatment A history of chronic degenerative, psychiatric, or neurologic disorder other than MG that can produce weakness or fatigue. 2. Other major chronic or debilitating illnesses within six months prior to study entry. 3. Female subjects who are premenopausal and are: 1. pregnant on the basis of a serum pregnancy test, 2. breast-feeding, or 3. not using an effective method of double barrier (1 hormonal plus 1 barrier method or 2 simultaneous barrier methods) or birth control (birth control pills, male condom, female condom, intrauterine device, Norplant, tubal ligation, or other sterilization procedures). 4. Altered levels of consciousness, dementia, or abnormal mental status. 5. Thymectomy in the previous six months. 6. Subjects who have been medicated with immunosuppressive drugs not listed in #5 within the last 8 weeks (56 days) prior to the baseline visit 7. Subjects who have been medicated with an immunosuppressive agent such as azathioprine, mycophenolate mofetil, cyclosporine, tacrolimus or methotrexate, that is withdrawn within 8 weeks (56 days) of the Baseline Visit. 8. Subjects who have received IVIg or PLEX treatment within the last 4 weeks (28 days) prior to the baseline visit. 9. Unstable dose or a stable dose of > 480 mg/day of pyridostigmine in 2 weeks prior to screening visit. 10. Daily use of non-steroidal anti-inflammatory drugs (NSAIDs). 11. History of renal or hepatic insufficiency or elevated liver enzymes (AST or ALT >2.5 x Upper Limit of Normal). 12. History of bone marrow hypoplasia, leucopenia, thrombocytopenia, significant anemia, clinical or laboratory evidence of immunodeficiency syndromes. 13. Forced Vital Capacity (FVC) <50% of percent predicted. General Safety & Laboratory ANC < 1.5 x 103 cells/microliter 15. Hemoglobin: < 8.0 gm/dL 16. Platelets: < 100,000/mm 17. Positive Hepatitis B or C serology (Hep B surface antigen and Hep C antibody) 18. History of positive HIV (HIV conducted during screening if applicable) 19. Treatment with any investigational agent within 4 weeks of screening or 5 half-lives of the investigational drug (whichever is longer) 20. Receipt of a live vaccine within 4 weeks prior to randomization 21. Previous treatment with rituximab (MabThera® / Rituxan®) 22. Previous treatment with natalizumab (Tysabri®) 23. History of severe allergic or anaphylactic reactions to humanized or murine monoclonal antibodies 24. History of recurrent significant infection or history of recurrent bacterial infections 25. Known active bacterial, viral fungal mycobacterial, or other infection (including tuberculosis or atypical mycobacterial disease, but excluding fungal infections of nail beds) or any major episode of infection requiring hospitalization or treatment with IV antibiotics within 4 weeks of screening or oral antibiotics within 2 weeks prior to screening 26. Unstable steroid dose in the past 4 weeks (28 days) 27. Lack of peripheral venous access 28. History of drug, alcohol, or chemical abuse within 6 months prior to screening 29. Concomitant malignancies or previous malignancies, with the exception of adequately treated basal or squamous cell carcinoma of the skin or carcinoma in situ of the cervix or prostate. 30. History of psychiatric disorder that would interfere with normal participation in this protocol 31. Significant cardiac or pulmonary disease (including obstructive pulmonary disease) 32. Any other disease, metabolic dysfunction, physical examination finding, or clinical laboratory finding giving reasonable suspicion of a disease or condition that contraindicates the use of an investigational drug or that may affect the interpretation of the results or render the subject at high risk from treatment complications 33. Subjects that do not record daily prednisone doses for at least 28 days before the Baseline Visit, or subjects whose prednisone dose varies by ≥6mg/day on average. 34. Prednisone dose of more than 100 mg/day (or 200 mg over a two day period) | 1 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 0.5-25.0, Neuroblastoma Ganglioneuroblastoma Participants must have histologically confirmed neuroblastoma or ganglioneuroblastoma or elevated urinary catecholamine metabolites. If tumor tissue was obtained, pathological review of surgical specimen at the Massachusetts General Hospital or other DF/HCC institution is required, but preliminary report only required prior to enrollment. If no tumor tissue was obtained, urinary catecholamine metabolites are required Participants do not need to have measurable disease at the time of radiation. Age ≥ 3 and ≤ 25 years at the time of diagnosis because this study evaluates this disease entity in the pediatric population which may differ from the adult population Life expectancy of greater than 12 months ECOG performance status ≤2 (Karnofsky ≥60%, Lansky ≥60%, see Appendix A) Diagnostic Imaging MRI and/or CT of the area to be treated within 8 weeks of any treatment. Baseline bone marrow biopsy and bone scan (with 99mTc-diphosphonate or MIBG scan (131I-MIBG or 123I-MIBG) from time of original diagnosis is required Because radiation is known to be teratogenic, women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation. Should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician immediately Ability of the patient or the patient's legal guardian to understand and the willingness to sign a written informed consent document Participants or their legal guardian must sign consent prior to the initiation of radiation therapy Patients will be ineligible if any prior therapeutic radiation therapy > 500 cGy has been delivered Patients will be ineligible if chemotherapy was completed ≥ 1 year from the planned start date of radiation therapy or if the patient is referred for radiation therapy after a relapse following a regimen with chemotherapy alone Individuals with a history of a different malignancy are ineligible except for the following circumstances. Individuals with a history of other malignancies are eligible if they have been disease-free for at least 5 years and are deemed by the investigator to be at low risk for recurrence of that malignancy. Individuals with the following cancers are eligible if diagnosed and treated within the past 5 years: cervical cancer in situ, and basal cell or squamous cell carcinoma of the skin Pregnant females are excluded. Women of childbearing age/menstruating must have a negative pregnancy test prior to initiation of radiation therapy | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 18.0-110.0, Critically Ill Patients With a Predicted Prolonged ICU Stay All adult ICU patients with a predicted prolonged ICU stay Preexisting neuromuscular disease, polyneuropathy or myopathy (e.g. Guillain-Barré, myasthenia gravis, Amyotrophic Lateral Sclerosis, paraplegia, multiple sclerosis, …) Receiving neuromuscular blocking agents Musculoskeletal conditions of the pelvis and/or lower limb (e.g. fractures, skin disease ) Focal neurological conditions of the pelvis and/or lower limb Skin disease (e.g. burns) Presence of a pace-maker or defibrillator Hemodynamic or Respiratory Instability, active cardiac ischemia High fever (>39°) Pregnancy Brain death | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 2.0-21.0, Clostridium Difficile Recipient Documented laboratory-confirmed clostridium difficile infection Documentation of ongoing diarrhea at time of recruitment Children ≥2 years old, <18 years old; young adults >18 years old, <21 years old Undergoing clinically-indicated colonoscopy Recurrent c-diff infection (three or more occurrences) Donor First-degree relative recommended, but not compulsory ≥ 18 years old In good health No antibiotic use within the last 90 days Recipient Severe comorbid condition (at discretion of the principal investigator) On immunosuppressive medications (high dose steroids 30 mg/kg of methylprednisolone) Severe or fulminant C. difficile colitis Toxic appearance Signs of hemodynamic instability Peritoneal signs on physical exam Anemia on complete blood count electrolyte imbalances on basic metabolic panel Considerations for Increased Risk of Adverse Events Should Be Given to patients with decompensated liver cirrhosis, advanced HIV/acquired immune deficiency syndrome, recent bone marrow transplant, or other cause of severe immunodeficiency | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 17.0-100.0, Organophosphate Poisoning Male or female Age over 16 Clinical diagnosis of OP insecticide poisoning Admission to Intensive Care Unit for Ventilation Informed consent from family Train of four measurement > 50% Age 16 or under Pregnant Consent not obtained from patient or patient's family | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 2.0-17.0, Epilepsy The following were applied in selecting patients for participation in the trial. Patient was eligible for entry into the baseline phase if he/she fulfilled the following at Visit 1 Written informed consent given by the parent(s)/guardian(s), and by the patient when appropriate Male or female patient aged between 2 and 17 years Body weight within the 10th and 90th percentiles, by age and sex A documented diagnosis of partial-onset seizures (simple or complex seizures with or without secondary generalisation), classified according to the International Classification of Epileptic Seizures Currently treated with 1 to 3 AEDs (any except OXC or CBZ), in a stable dosage regimen during at least 1 month prior to screening Good general health (apart from epilepsy) based on medical history and physical examination In case of a female patient, she was premenarchal, surgically sterile or presented a urine pregnancy test consistent with a non-gravid state and practiced an effective non-hormonal contraception method. At Visit 2, patient was eligible for entry into the Eslicarbazepine acetate treatment phase if he/she fulfilled the following At least 4 partial-onset seizures during the last 4 weeks of the baseline phase Brain CT scan or MRI that excluded rapidly progressive neurological diseases Patient was not allowed for entry into the screening phase if he/she fulfilled the following at Visit 1 Primarily generalised epilepsy Clinically relevant medical condition, other than epilepsy History of status epilepticus in the last 3 months History of suicide attempt History of alcohol or drug abuse History of hypersensitivity or intolerance to OXC or CBZ Use of any investigational drug or participated in any clinical trial within the previous 2 months Patient and/or his/her caregiver(s) unlikely to co-operate with the requirements of the study If female, she was sexually active and of child-bearing potential and she did not use reliable contraception | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 0.0-55.0, Sickle Cell Disease Transfusion Dependent Alpha- or Beta- Thalassemia Diamond Blackfan Anemia Paroxysmal Nocturnal Hemoglobinuria Glanzmann Thrombasthenia Severe Congenital Neutropenia Shwachman-Diamond Syndrome Non-Malignant Hematologic Disorders Diagnosis of Sickle Cell Disease, Thalassemia, Diamond Blackfan Anemia or other non-malignant hematologic disorders for which a stem cell transplant is indicated Acceptable stem cell source identified Performance status of ≥ 70% (Karnofsky),or ≥ 70 (Lansky play score) Creatinine <2.0 mg/dl for adults or glomerular filtration rate > 50 ml/min for children Bilirubin, Aspartate Aminotransferase, Alkaline phosphatase <5 times the upper limit of institutional normal Absence of decompensated congestive heart failure, or uncontrolled arrhythmia and left ventricular ejection fraction > 40% active, uncontrolled infection pregnant or breastfeeding HIV positive | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 0.0-999.0, Ocular Hypertension Open-angle Glaucoma Subject 1. 0-2 years of age and 18 years or greater. 2. Diagnosis of open angle glaucoma or ocular hypertension 3. Unmedicated (post-washout) IOP (Intraocular Pressure) >20 mm Hg and < 27 mm Hg in the study eye at 2 qualification visits. 4. Corrected visual acuity in each eye equivalent to 20/200. 5. Able and willing to give signed informed consent (parent or guardian consent for pediatric patient) and follow study instructions. Subject Ophthalmic: 1. Glaucoma: pseudoexfoliation or pigment dispersion component, history of angle closure, or narrow angles. Note: Previous laser peripheral iridotomy is NOT acceptable. 2. Intraocular pressure ≥27 mm Hg (unmedicated) in both eyes (individuals who are excluded for this criterion are not allowed to attempt requalification), or use of more than two ocular hypotensive medications within 30 days of screening. Note: fixed dose combinations count as two medications. 3. Known hypersensitivity to any component of the formulations to be used (benzalkonium chloride, etc.), to topical anesthetics or beta adrenoceptor antagonists. 4. Previous glaucoma intraocular surgery or glaucoma laser procedures in either eye 5. Refractive surgery in either eye. 6. Ocular trauma in either eye within the six months prior to screening, or ocular surgery or non-refractive laser treatment within the three months prior to screening. 7. Recent or current evidence of ocular infection or inflammation in either eye. Current evidence of clinically significant blepharitis, conjunctivitis, or a history of herpes simplex or zoster keratitis at screening in either eye. 8. Ocular medication in either eye of any kind within 30 days of screening. 9. Clinically significant ocular disease in either eye (e.g., corneal edema, uveitis, severe keratoconjunctivitis sicca) which might interfere with the study, including glaucomatous damage so severe that washout of ocular hypotensive medications for one month is not judged safe. 10. Central corneal thickness in either eye greater than 600 µm at screening. 11. Any abnormality in either eye preventing reliable applanation tonometry of either eye. Systemic: 12. Clinically relevant abnormalities (as determined by the investigator) in laboratory tests at screening which may impact the study. 13. Known hypersensitivity or contraindication to beta-adrenoceptor antagonists (e.g., chronic obstructive pulmonary disease or bronchial asthma; abnormally low blood pressure or heart rate; second or third degree heart block or congestive heart failure; severe diabetes). 14. Clinically significant systemic disease (e.g., uncontrolled diabetes, myasthenia gravis, hepatic, renal, endocrine or cardiovascular disorders) which might interfere with the study. 15. Participation in any investigational study within 30 days prior to screening. 16. Changes of systemic medication that could have an effect on intraocular pressure within 30 days prior to screening, or anticipated during the study. 17. Women of childbearing potential who are pregnant, nursing, planning a pregnancy, or not using a medically acceptable form of birth control. An adult woman is considered to be of childbearing potential unless she is one year post-menopausal or three months post-surgical sterilization. All females of childbearing potential must have a negative urine pregnancy test result at the screening examination and must not intend to become pregnant during the study | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 0.0-999.0, Osteoarthritis BMI ≥ 40 Revision of total knee replacement Rheumatoid arthritis or secondary arthritis Patients on Warfarin Patients on steroids Patients on long term antibiotics Previous deep joint infection Allergy to silver Simultaneous bilateral total knee replacement patients Patients who do not wish to participate Patients who are unable to give informed consent Patients who are unable to attend Golden Jubilee National Hospital for follow up | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 6.0-999.0, Spinal Muscular Atrophy Type 3 Spinal muscular atrophy type 3, genetically confirmed Age higher than 6 years old Ambulatory patient Informed consent signed More than 100 meters of walking at 6-minute walk test at screening Value at screening and baseline in a range of 20% of the highest value at 6-minute walk test Patient who had surgical intervention or suffer from a recent traumatism (less than 6 months) Associated pathology such as endocrinopathy, infectious disease, allergy, myopathy, chronic or acute inflammatory pathology, during 3 weeks preceding the inclusion Other therapeutics than food supplements or those frequently prescribed in spinal muscular atrophy or its complications Non tolerance of electromyography Limited collaboration due to trouble in information comprehension Pathology inducing contra-indication for pyridostigmine treatment (allergy at molecule, asthma, Parkinson disease, mechanic obstruction of urinary or digestive tracts) | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 0.083-999.0, Pulmonary Arterial Hypertension of Congenital Heart Disease The patient has a congenital heart disease other than patent foramen ovale The diagnosis of pulmonary hypertension was confirmed by cardiac catheterization. Only patients with Eisenmenger syndrome can be included without catheterization The catheterization was done after 1 January 2009 A mean pulmonary artery pressure > 25 mm Hg Pulmonary vascular resistances > 3 piece Wood m2 Pulmonary capillary pressure available Consent for in the study must be signed by parents or legal guardians for minors, by the patient for adults The patient he had a surgical procedure or interventional catheterization cardiac catheterization between his diagnosis and in the observatory? If yes, it can only be included if a new catheterization confirmed the persistence of HTAP at least 6 months after the procedure Patient follow-up (at least once a year) in the center for its HTAP associated with congenital heart disease its | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 0.0-18.0, Autoimmune Disease Juvenile Idiopathic Arthritis Inflammatory Bowel Disease Evidence of Liver Transplantation Kidney Transplant Infection Bone Marrow Transplant Infection Immunosuppressed patients with following diseases; Bone marrow transplant recipients, liver transplant patients, renal transplant, Children with inflammatory bowel disease, juvenile Idiopathic arthritis and autoimmune conditions A platelet count of <50 Immunoglobulin therapy within 3 months Yeast allergy Any other known allergies to one of the vaccine component | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 0.0-25.0, Diffuse Intrinsic Pontine Glioma (DIPG) for Newly Diagnosed Patients with diffuse intrinsic pontine glioma (DIPG) Diagnosis: Patients with newly diagnosed DIPG, defined as tumors with a pontine epicenter and diffuse involvement of the pons who undergo a biopsy are eligible. Patients with disseminated disease are not eligible, and MRI of the spine must be performed if disseminated disease is suspected by the treating physician Enrollment within 28 days of the date of radiographic diagnosis Age ≤ 25 years Karnofsky score ≥ 50 for patients ≥ 16 years of age and Lansky score ≥ 50 for patients ≤15 years of age. Patients who are unable to walk because of paralysis but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score Organ Function Requirements Adequate Bone Marrow Function Defined as Peripheral absolute neutrophil count (ANC) ≥ 1000/mm 3 Platelet count ≥ 100,000/mm 3 (transfusion independent, defined as not receiving platelet transfusions for at least 7 days prior to enrollment) Hemoglobin ≥ 8 g/dl (can be transfusion dependent) for Newly Diagnosed Patients with DIPG Patients who are currently taking any anti-cancer directed therapy. Steroids are not considered anti-cancer therapy Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements Female patients of childbearing potential must not be pregnant or breast-feeding. Female patients of childbearing potential must have a negative serum or urine pregnancy test prior to the start of therapy Patients with inability to return for follow-up visits or obtain follow-up studies required to assess toxicity to therapy | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 2.0-17.0, Down Syndrome • Patients with DS and those without DS receiving BIS monitoring Children between the ages of 2 and 17 years old (inclusive) Patients scheduled for unilateral or bilateral tympanostomy (ear tube placement) at Penn State Hershey Medical Center Patients with American Society of Anesthesiologists (ASA) physical status I, II and III Eligible for standard general anesthesia technique protocol as determined by anesthesia provider(s) Patients with congenital diseases/anomalies except Down's Syndrome Patients with myotonic dystrophies or other neurodegenerative diseases Patients with cerebrovascular accidents (strokes) Patients not receiving BIS monitoring Pregnant patients Patients with allergic skin reactions to electrode patches Patients 18 years of age and greater | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 0.0-999.0, Primary and Secondary Immunodeficiency and Other Conditions Requiring Regular Administration of Octagam 5% or 10% IVIG Any patient who needs to be treated with a product of this class because of his/her medical condition and whom the treating physician decides to prescribe Octagam or panzyga regularly, may be included None | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 16.0-999.0, Cystic Fibrosis Pass the inhalation test Chronic sputum production, at least ≥ 10 ml /24h Cystic Fibrosis (CF) diagnosed ( established by genotype or sweat sodium>70 mmol/l or sweat chloride of>60 mmol/l) Clinically stable at the time of recruitment (defined as no requirement for antibiotics or change in respiratory medication in the preceding 4 weeks) Trained in the use of autogenic drainage technique (at least 6 months) Inhaling hypertonic saline since at least 6 months To be able to provide written, informed consent and perform the protocol and the evaluations Active massive hemoptysis during the previous 2 months Patient in transplantation or retransplantation list Patient already participating in another study at the same time | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 0.0-45.0, Infants of Diabetic Mothers (understood to only abstemious women) 1. . Pregnant women expected to deliver between 37 and 41 weeks gestation (controls), and their babies 2. . Pregnant women expected to deliver between 37 and 41 weeks gestation who have class A1 diabetes mellitus, and their babies 3. . Pregnant women expected to deliver between 37 and 41 weeks gestation who have class A2 diabetes mellitus, and their babies 4. . Pregnant women expected to deliver between 37 and 41 weeks gestation who were diagnosed with diabetes mellitus prior to their pregnancy, and their babies . Mothers who self-reported any alcohol or any illicit drug use during their pregnancy (and their babies) 2. . Mothers who had a positive drug screen at any point during their pregnancy (and their babies) 3. . Babies whose mothers suffered a placental abruption during their pregnancy. 4. . Babies whose mothers had inadequate prenatal care (defined as <3 prenatal clinic visits prior to admission for delivery) 5. . Non-English-speaking mothers 6. . Babies who pass meconium in utero. 7. . Babies born with multiple congenital anomalies or abdominal wall defects | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 0.0-999.0, Hip Fracture Patients scheduled for surgical fixation of hip fracture Patients with isolated inter-trochantric fracture American Society of Anesthesiologists (ASA) physical status 1 to 3 Patients who are able to give their own consent Patients unable to give consent or inability to communicate/ cooperate Patients with regular consumption of strong opioids (morphine, oxycodone) or steroids Patients with allergy to local anaesthetics or any drugs included in the study Patients with contraindications to spinal anaesthesia such as coagulation disorders/ thrombocytopenia and local infection at site of injection | 0 |
The patient is a 16-year-old girl recently diagnosed with myasthenia gravis, class IIa. She complains of diplopia and weakness affecting in her upper extremities. She had a positive anti-AChR antibody test, and her single fiber electromyography (SFEMG) was positive. She is on acetylcholinesterase inhibitor treatment combined with immunosuppressants. But she still has some symptoms. She does not smoke or use illicit drugs. She is not sexually active, and her menses are regular. Her physical exam and lab studies are not remarkable for any other abnormalities. BP: 110/75 Hgb: 11 g/dl WBC: 8000 /mm3 Plt: 300000 /ml Creatinine: 0.5 mg/dl BUN: 10 mg/dl Beta hcg: negative for pregnancy | eligible ages (years): 35.0-999.0, Graves Ophthalmopathy Active TED: Onset less than 6 months AND: Progressive with historic or measured worsening in one or more of VISA parameters in past 2 months: (increasing soft tissue inflammatory changes, development of intermittent or constant diplopia or increased prominence of either eye or lid retraction) 2. Moderately severe TED (all of the following must be met): V: No optic neuropathy I: Inflammatory score >/= 4/10 S: Intermittent or constant diplopia in any direction except primary gaze AND/OR restriction in ductions to < 30 degrees in any cardinal direction on clinical examination - Age < 35 yrs 2. Diabetes mellitus 3. Previous orbital surgery or radiotherapy for TED 4. Corticosteroid or immunotherapy within previous 2 months for TED 5. Unable or unwilling to provide informed consent- | 0 |
Subsets and Splits
No community queries yet
The top public SQL queries from the community will appear here once available.