## Protocol Section
### Identification Module
**NCT ID:** NCT01355679

**Brief Title:** Molecular Guided Therapy for Refractory or Recurrent Neuroblastoma

**Official Title:** A Feasibility Trial Using Molecular-Guided Therapy for the Treatment of Patients With Refractory or Recurrent Neuroblastoma

**NCT ID Aliases:**
- NCT01375517
#### Organization Study ID Info
**ID:** NMTRC 001
#### Organization
**Class:** OTHER
**Full Name:** Milton S. Hershey Medical Center
### Status Module
#### Completion Date
**Date:** 2015-01

**Type:** ACTUAL

#### Expanded Access Info
#### Last Update Post Date
**Date:** 2024-04-03

**Type:** ACTUAL

**Last Update Submit Date:** 2024-04-01

**Overall Status:** COMPLETED

#### Primary Completion Date
**Date:** 2013-09

**Type:** ACTUAL

#### Results First Post Date
**Date:** 2016-07-28

**Type:** ESTIMATED

**Results First Submit Date:** 2015-05-18

**Results First Submit QC Date:** 2016-06-16

#### Start Date
**Date:** 2011-05

**Status Verified Date:** 2024-04

#### Study First Post Date
**Date:** 2011-05-18
**Type:** ESTIMATED
**Study First Submit Date:** 2011-05-13
**Study First Submit QC Date:** 2011-05-17
### Sponsor Collaborators Module

#### Lead Sponsor

**Class:** OTHER

**Name:** Giselle Sholler

#### Responsible Party

**Investigator Affiliation:** Milton S. Hershey Medical Center

**Investigator Full Name:** Giselle Sholler

**Investigator Title:** Study Chair

**Type:** SPONSOR_INVESTIGATOR

### Oversight Module

**Oversight Has DMC:** True

### Description Module

**Brief Summary:** The purpose of this study is to test the feasibility (ability to be done) of an experimental test to help plan your cancer treatment. This study plan is not studying the effectiveness of the proposed combinations of therapy for your cancer that you may receive after the experimental testing.

This study will look at an experimental technology to determine a tumor's molecular makeup (gene expression profile). This technology (called "OncInsights") is being used to discover new ways to understand cancers and potentially predict the best treatments for patients with cancer in the future. The experimental technology has not been approved by the U.S. Food and Drug Administration.

### Conditions Module

**Conditions:**

- Neuroblastoma

### Design Module

#### Design Info

**Allocation:** NA

**Intervention Model:** SINGLE_GROUP

##### Masking Info

**Masking:** NONE

**Primary Purpose:** DIAGNOSTIC

#### Enrollment Info

**Count:** 16

**Type:** ACTUAL

**Phases:**

- NA

**Study Type:** INTERVENTIONAL

### Arms Interventions Module

#### Arm Group 1

**Description:** A total of 14 eligible neuroblastoma patients who are refractory or relapsed on conventional therapy will be treated. Guided therapy will allow the use of any therapeutic combination (up to 4 agents) provided it includes medications contained in the study report. All patients will be followed for survival, disease response, progression and safety. All patients will be treated according to the discretion of the treating oncologist and study committee (minimum 3 oncologists and one pharmacist). Extent of disease will be measured and assessed for changes throughout the course of the study and at 6-8 week intervals (every 2 cycles).

**Intervention Names:**

- Device: Guided Therapy

**Label:** Guided therapy

**Type:** EXPERIMENTAL

### Interventions

#### Intervention 1

**Arm Group Labels:**

- Guided therapy

**Description:** A total of 14 eligible neuroblastoma patients who are refractory or relapsed on conventional therapy will be treated. Guided therapy will allow the use of any therapeutic combination (up to 4 agents) provided it includes medications contained in the study report. All patients will be followed for disease response, progression and safety. All patients will be treated according to the discretion of the treating oncologist and study committee (minimum 3 oncologists and one pharmacist). Extent of disease will be measured and assessed for changes throughout the course of the study and at 6-8 week intervals (every 2 cycles).

**Name:** Guided Therapy

**Type:** DEVICE

### Outcomes Module

#### Primary Outcomes

**Description:** Feasibility parameter defined as: Enrollment onto study, quality mRNA obtained, gene chip completed, tumor board held, medical monitor review and approval, start of treatment by 21 days post biopsy/surgical resection date, and then completion of 1 cycle of therapy."

**Measure:** Percentage of Participants That Are Able to Meet Feasibility Parameters.

**Time Frame:** 1 year

#### Secondary Outcomes

**Description:** To determine the safety of allowing a molecular tumor board to determine individualized treatment plans

**Measure:** Number of Participants With Adverse Events as a Measure of Safety

**Time Frame:** 1 year

**Description:** Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0) for target lesions and assessed by MRI: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR.

**Measure:** Overall Response Rate (ORR) of Participants Using RECIST Criteria

**Time Frame:** 1 year

**Description:** Progression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0), as a 20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions

**Measure:** Activity of Treatments Chosen Based on Progression Free Survival (PFS)

**Time Frame:** 1 year

### Eligibility Module

**Eligibility Criteria:** Inclusion Criteria:

* 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
* No other significant organ toxicity defined as \> Grade 2 by National Cancer Institute Common Toxicity Criteria for Adverse Events NCI-CTCAE V4.0
* A negative serum pregnancy test is required for female participants of child bearing potential (≥ 13 years of age or after onset of menses)
* 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 include 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.
* Informed Consent: All patients and/or legal guardians must sign informed written consent. Assent, when appropriate, will be obtained according to institutional guidelines. Voluntary consent for optional biology studies will be included.

Exclusion Criteria:

* 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

**Minimum Age:** 1 Year

**Sex:** ALL

**Standard Ages:**

- CHILD

- ADULT

- OLDER_ADULT

### Contacts Locations Module

#### Locations

**Location 1:**

**City:** Hartford

**Country:** United States

**Facility:** Connecticut Children's Hospital

**State:** Connecticut

**Zip:** 06106

**Location 2:**

**City:** Orlando

**Country:** United States

**Facility:** Arnold Palmer Hospital for Children- MD Anderson

**State:** Florida

**Zip:** 32806

**Location 3:**

**City:** Bethesda

**Country:** United States

**Facility:** National Cancer Institute

**State:** Maryland

**Zip:** 20877

**Location 4:**

**City:** Grand Rapids

**Country:** United States

**Facility:** Helen DeVos Children's Hospital

**State:** Michigan

**Zip:** 49503

**Location 5:**

**City:** Kansas City

**Country:** United States

**Facility:** Children's Mercy Hospitals and Clinics

**State:** Missouri

**Zip:** 64108

**Location 6:**

**City:** Saint Louis

**Country:** United States

**Facility:** Cardinal Glennon Children's Medical Center

**State:** Missouri

**Zip:** 63104

**Location 7:**

**City:** Charlotte

**Country:** United States

**Facility:** Levine Children's Hospital

**State:** North Carolina

**Zip:** 28204

#### Overall Officials

**Official 1:**

**Affiliation:** Beat Childhood Cancer at Atrium Health

**Name:** Giselle Sholler, MD

**Role:** STUDY_CHAIR

### References Module

#### See Also Links

**Label:** Beat Childhood Cancer

**URL:** https://beatcc.org/

## Derived Section

### Condition Browse Module - Ancestors

- ID: D000018241

- Term: Neuroectodermal Tumors, Primitive, Peripheral

- ID: D000018242

- Term: Neuroectodermal Tumors, Primitive

- ID: D000018302

- Term: Neoplasms, Neuroepithelial

- ID: D000017599

- Term: Neuroectodermal Tumors

- ID: D000009373

- Term: Neoplasms, Germ Cell and Embryonal

- ID: D000009370

- Term: Neoplasms by Histologic Type

- ID: D000009369

- Term: Neoplasms

- ID: D000009375

- Term: Neoplasms, Glandular and Epithelial

- ID: D000009380

- Term: Neoplasms, Nerve Tissue

### Condition Browse Module - Browse Branches

- Abbrev: BC23

- Name: Symptoms and General Pathology

- Abbrev: All

- Name: All Conditions

- Abbrev: BC04

- Name: Neoplasms

- Abbrev: Rare

- Name: Rare Diseases

### Condition Browse Module - Browse Leaves

- ID: M14850

- Name: Recurrence

- Relevance: LOW

- As Found: Unknown

- ID: M12391

- Name: Neuroblastoma

- Relevance: HIGH

- As Found: Neuroblastoma

- ID: M20387

- Name: Neuroectodermal Tumors, Primitive, Peripheral

- Relevance: LOW

- As Found: Unknown

- ID: M19845

- Name: Neuroectodermal Tumors

- Relevance: LOW

- As Found: Unknown

- ID: M20388

- Name: Neuroectodermal Tumors, Primitive

- Relevance: LOW

- As Found: Unknown

- ID: M20446

- Name: Neoplasms, Neuroepithelial

- Relevance: LOW

- As Found: Unknown

- ID: M12318

- Name: Neoplasms, Germ Cell and Embryonal

- Relevance: LOW

- As Found: Unknown

- ID: M12315

- Name: Neoplasms by Histologic Type

- Relevance: LOW

- As Found: Unknown

- ID: M12320

- Name: Neoplasms, Glandular and Epithelial

- Relevance: LOW

- As Found: Unknown

- ID: M12325

- Name: Neoplasms, Nerve Tissue

- Relevance: LOW

- As Found: Unknown

- ID: T4085

- Name: Neuroblastoma

- Relevance: HIGH

- As Found: Neuroblastoma

- ID: T4092

- Name: Neuroepithelioma

- Relevance: LOW

- As Found: Unknown

### Condition Browse Module - Meshes

- ID: D000009447

- Term: Neuroblastoma

### Misc Info Module

- Version Holder: 2024-05-24

## Results Section

### Adverse Events Module

#### Event Groups

**Group ID:** EG000

**Title:** Guided Therapy

**Description:** A total of 14 eligible neuroblastoma patients who are refractory or relapsed on conventional therapy will be treated. Guided therapy will allow the use of any therapeutic combination (up to 4 agents) provided it includes medications contained in the study report. All patients will be followed for survival, disease response, progression and safety. All patients will be treated according to the discretion of the treating oncologist and study committee (minimum 3 oncologists and one pharmacist). Extent of disease will be measured and assessed for changes throughout the course of the study and at 6-8 week intervals (every 2 cycles).

**ID:** EG000

**Other Num Affected:** 12

**Other Num at Risk:** 14

**Serious Number At Risk:** 14

**Title:** Guided Therapy

**Frequency Threshold:** 0

#### Other Events

**Term:** Abdominal Pain

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

**Organ System:** Gastrointestinal disorders

**Source Vocabulary:** CTCAE (3.0)

**Term:** ALT elevation

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

**Organ System:** Hepatobiliary disorders

**Source Vocabulary:** CTCAE (3.0)

**Term:** AST Elevation

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

**Organ System:** Hepatobiliary disorders

**Source Vocabulary:** CTCAE (3.0)

**Term:** Anemia

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

**Organ System:** Blood and lymphatic system disorders

**Source Vocabulary:** CTCAE (3.0)

**Term:** Elevated Bilirubin

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

**Organ System:** Hepatobiliary disorders

**Source Vocabulary:** CTCAE (3.0)

**Term:** Constipation

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

**Organ System:** Gastrointestinal disorders

**Source Vocabulary:** CTCAE (3.0)

**Term:** Dehydration

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

**Organ System:** Gastrointestinal disorders

**Source Vocabulary:** CTCAE (3.0)

**Term:** Fatigue

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

**Organ System:** General disorders

**Source Vocabulary:** CTCAE (3.0)

**Term:** Fever

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

**Organ System:** Infections and infestations

**Source Vocabulary:** CTCAE (3.0)

**Term:** Hypoalbunemia

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

**Organ System:** Blood and lymphatic system disorders

**Source Vocabulary:** CTCAE (3.0)

**Term:** Hypocalcemia

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

**Organ System:** General disorders

**Source Vocabulary:** CTCAE (3.0)

**Term:** Hypophosphatemia

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

**Organ System:** General disorders

**Source Vocabulary:** CTCAE (3.0)

**Term:** Infection

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

**Organ System:** Infections and infestations

**Source Vocabulary:** CTCAE (3.0)

**Term:** Leukopenia

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

**Organ System:** Blood and lymphatic system disorders

**Source Vocabulary:** CTCAE (3.0)

**Term:** Lymphocytopenia

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

**Organ System:** Blood and lymphatic system disorders

**Source Vocabulary:** CTCAE (3.0)

**Term:** Mucositis

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

**Organ System:** Gastrointestinal disorders

**Source Vocabulary:** CTCAE (3.0)

**Term:** Myalgia

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

**Organ System:** General disorders

**Source Vocabulary:** CTCAE (3.0)

**Term:** Nausea

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

**Organ System:** Gastrointestinal disorders

**Source Vocabulary:** CTCAE (3.0)

**Term:** Neutropenia

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

**Organ System:** Blood and lymphatic system disorders

**Source Vocabulary:** CTCAE (3.0)

**Term:** Pain

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

**Organ System:** General disorders

**Source Vocabulary:** CTCAE (3.0)

**Term:** Rash

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

**Organ System:** Skin and subcutaneous tissue disorders

**Source Vocabulary:** CTCAE (3.0)

**Term:** Tachycardia

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

**Organ System:** Cardiac disorders

**Source Vocabulary:** CTCAE (3.0)

**Term:** Thrombocytopenia

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

**Organ System:** Blood and lymphatic system disorders

**Source Vocabulary:** CTCAE (3.0)

**Term:** Vomiting

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

**Organ System:** Gastrointestinal disorders

**Source Vocabulary:** CTCAE (3.0)

**Term:** Weight Loss

**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT

**Organ System:** General disorders

**Source Vocabulary:** CTCAE (3.0)

**Time Frame:** Time on therapy plus 30 days after last dose and until all related events resolved, an average of 1 year.

## Results Section - Baseline Characteristics Module

### Denomination Counts

**Group ID:** BG000

**Value:** 16

**Units:** Participants

### Group

**ID:** BG000

**Title:** Guided Therapy

**Description:** All subjects receive guided therapy in therapeutic combination (up to 4 agents) provided it includes medications contained in the study report.

### Measure

#### Measurement

**Group ID:** BG000

**Value:** 15

**Category Title:** <=18 years

#### Measurement

**Group ID:** BG000

**Value:** 1

**Category Title:** Between 18 and 65 years

#### Measurement

**Group ID:** BG000

**Value:** 0

**Category Title:** >=65 years

**Class Title:** 

### Measure

#### Measurement

**Group ID:** BG000

**Value:** 7

**Category Title:** Female

#### Measurement

**Group ID:** BG000

**Value:** 9

**Category Title:** Male

**Class Title:** 

### Measure

#### Measurement

**Group ID:** BG000

**Value:** 0

**Category Title:** American Indian or Alaska Native

#### Measurement

**Group ID:** BG000

**Value:** 0

**Category Title:** Asian

#### Measurement

**Group ID:** BG000

**Value:** 0

**Category Title:** Native Hawaiian or Other Pacific Islander

#### Measurement

**Group ID:** BG000

**Value:** 1

**Category Title:** Black or African American

#### Measurement

**Group ID:** BG000

**Value:** 11

**Category Title:** White

#### Measurement

**Group ID:** BG000

**Value:** 0

**Category Title:** More than one race

#### Measurement

**Group ID:** BG000

**Value:** 4

**Category Title:** Unknown or Not Reported

**Class Title:** 

### Measure

#### Measurement

**Group ID:** BG000

**Value:** 16

**Class Title:** United States

**Denomination Units Selected:** 

## Results Section - Baseline Characteristics Module

### Measure 1
**Parameter Type:** COUNT_OF_PARTICIPANTS
**Title:** Age, Categorical
**Unit of Measure:** Participants
### Measure 2
**Parameter Type:** COUNT_OF_PARTICIPANTS
**Title:** Sex: Female, Male
**Unit of Measure:** Participants
### Measure 3
**Parameter Type:** COUNT_OF_PARTICIPANTS
**Title:** Race (NIH/OMB)
**Unit of Measure:** Participants
### Measure 4
**Parameter Type:** NUMBER
**Title:** Region of Enrollment
**Unit of Measure:** participants
**Population Description:** Multiply relapsed or refractory neuroblastoma.
## Results Section - More Information Module
### Certain Agreement
### Point of Contact
**Email:** giselle.sholler@helendevoschildrens.org
**Organization:** NMTRC at Spectrum Health
**Phone:** 616-267-0334
**Title:** Giselle Sholler, MD
## Results Section - Outcome Measures Module
### Outcome Measure 1
### Outcome Measure 2
### Outcome Measure 3
### Outcome Measure 4
## Results Section
### Outcome Measures Module
#### Outcome Measure 1
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 
- **Upper Limit:** 
- **Value:** 100
**Title:** 
#### Outcome Measure 2
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 
- **Upper Limit:** 
- **Value:** 12
**Title:** 
#### Outcome Measure 3
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 
- **Spread:** 
- **Upper Limit:** 
- **Value:** 7
**Title:** 
#### Outcome Measure 4
**Class:**
##### Category
**Measurements:**
- **Comment:** 
- **Group ID:** OG000
- **Lower Limit:** 43
- **Spread:** 
- **Upper Limit:** 59
- **Value:** 59
**Title:** 
## Results Section
### Outcome Measures Module
#### Outcome Measure 1
**Description:** Feasibility parameter defined as: Enrollment onto study, quality mRNA obtained, gene chip completed, tumor board held, medical monitor review and approval, start of treatment by 21 days post biopsy/surgical resection date, and then completion of 1 cycle of therapy."
**Parameter Type:** NUMBER
**Population Description:** All subjects had soft tissue disease in which biopsy was possible. Two subjects were deemed ineligible due to benign tumor type after biopsy.
**Reporting Status:** POSTED
**Time Frame:** 1 year
**Title:** Percentage of Participants That Are Able to Meet Feasibility Parameters.
**Type:** PRIMARY
**Unit of Measure:** percentage of participants
##### Group
**Description:** All subjects receive guided therapy in therapeutic combination (up to 4 agents) provided it includes medications contained in the study report.
**ID:** OG000
**Title:** Guided Therapy
#### Outcome Measure 2
**Description:** To determine the safety of allowing a molecular tumor board to determine individualized treatment plans
**Parameter Type:** NUMBER
**Reporting Status:** POSTED
**Time Frame:** 1 year
**Title:** Number of Participants With Adverse Events as a Measure of Safety
**Type:** SECONDARY
**Unit of Measure:** participants
##### Group
**Description:** A total of 14 eligible neuroblastoma patients who are refractory or relapsed on conventional therapy will be treated. Guided therapy will allow the use of any therapeutic combination (up to 4 agents) provided it includes medications contained in the study report. All patients will be followed for survival, disease response, progression and safety. All patients will be treated according to the discretion of the treating oncologist and study committee (minimum 3 oncologists and one pharmacist). Extent of disease will be measured and assessed for changes throughout the course of the study and at 6-8 week intervals (every 2 cycles).
**ID:** OG000
**Title:** Guided Therapy
#### Outcome Measure 3
**Description:** Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0) for target lesions and assessed by MRI: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR.
**Parameter Type:** NUMBER
**Reporting Status:** POSTED
**Time Frame:** 1 year
**Title:** Overall Response Rate (ORR) of Participants Using RECIST Criteria
**Type:** SECONDARY
**Unit of Measure:** percentage of participants with PR or CR
##### Group
**Description:** A total of 14 eligible neuroblastoma patients who are refractory or relapsed on conventional therapy will be treated. Guided therapy will allow the use of any therapeutic combination (up to 4 agents) provided it includes medications contained in the study report. All patients will be followed for survival, disease response, progression and safety. All patients will be treated according to the discretion of the treating oncologist and study committee (minimum 3 oncologists and one pharmacist). Extent of disease will be measured and assessed for changes throughout the course of the study and at 6-8 week intervals (every 2 cycles).
**ID:** OG000
**Title:** Guided Therapy
#### Outcome Measure 4
**Description:** Progression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0), as a 20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions
**Dispersion Type:** 95% Confidence Interval
**Parameter Type:** MEAN
**Reporting Status:** POSTED
**Time Frame:** 1 year
**Title:** Activity of Treatments Chosen Based on Progression Free Survival (PFS)
**Type:** SECONDARY
**Unit of Measure:** Days
##### Group
**Description:** A total of 14 eligible neuroblastoma patients who are refractory or relapsed on conventional therapy will be treated. Guided therapy will allow the use of any therapeutic combination (up to 4 agents) provided it includes medications contained in the study report. All patients will be followed for survival, disease response, progression and safety. All patients will be treated according to the discretion of the treating oncologist and study committee (minimum 3 oncologists and one pharmacist). Extent of disease will be measured and assessed for changes throughout the course of the study and at 6-8 week intervals (every 2 cycles).
**ID:** OG000
**Title:** Guided Therapy
### Participant Flow Module
#### Group
**Description:** A total of 14 neuroblastoma patients who are refractory or relapsed on conventional therapy will be treated. Guided therapy will allow the use of any therapeutic combination (up to 4 agents) provided it includes medications contained in the study report. All patients will be followed for survival, disease response, progression and safety. All patients will be treated according to the discretion of the treating oncologist and study committee (minimum 3 oncologists and one pharmacist). Extent of disease will be measured and assessed for changes throughout the course of the study and at 6-8 week intervals (every 2 cycles).

Guided Therapy: A total of 14 neuroblastoma patients who are refractory or relapsed on conventional therapy will be treated. Guided therapy will allow the use of any therapeutic combination (up to 4 agents) provided it includes medications contained in the study report. All patients will be followed for disease response, progression and safety. All patients will be
**ID:** FG000
**Title:** Guided Therapy
#### Period
**Title:** Overall Study
##### Withdraw
**Type:** benign tumor types found at biopsy
###### Reason
**Group ID:** FG000
**Number of Subjects:** 2
##### Milestone
**Type:** STARTED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 16
##### Milestone
**Type:** COMPLETED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 14
##### Milestone
**Type:** NOT COMPLETED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 2
**Recruitment Details:** This was an open label, multi-center prospective feasibility study in patients with refractory or recurrent neuroblastoma that enrolled at NMTRC centers across the country between 8/11/2011 and 11/26/2012.
**Has Results:** True