## Protocol Section
### Identification Module
**NCT ID:** NCT00803179

**Brief Title:** Growth Hormone Therapy for Wasting in Cystic Fibrosis

**Official Title:** Growth Hormone Therapy for Wasting in Cystic Fibrosis

#### Organization Study ID Info
**ID:** 900005
#### Organization
**Class:** OTHER
**Full Name:** University of Massachusetts, Worcester
### Status Module
#### Completion Date
**Date:** 2012-03

**Type:** ACTUAL

#### Expanded Access Info
#### Last Update Post Date
**Date:** 2013-01-15

**Type:** ESTIMATED

**Last Update Submit Date:** 2012-12-10

**Overall Status:** TERMINATED

#### Primary Completion Date
**Date:** 2012-03

**Type:** ACTUAL

#### Results First Post Date
**Date:** 2013-01-15

**Type:** ESTIMATED

**Results First Submit Date:** 2012-11-13

**Results First Submit QC Date:** 2012-12-10

#### Start Date
**Date:** 2008-11

**Status Verified Date:** 2012-12

#### Study First Post Date
**Date:** 2008-12-05
**Type:** ESTIMATED
**Study First Submit Date:** 2008-12-04
**Study First Submit QC Date:** 2008-12-04
**Why Stopped:** Poor enrollment, patients were lost to follow up
### Sponsor Collaborators Module

#### Lead Sponsor

**Class:** OTHER

**Name:** University of Massachusetts, Worcester

#### Responsible Party

**Investigator Affiliation:** University of Massachusetts, Worcester

**Investigator Full Name:** Michael Stalvey

**Investigator Title:** Study Principle Investigator

**Type:** PRINCIPAL_INVESTIGATOR

### Oversight Module

**Oversight Has DMC:** False

### Description Module

**Brief Summary:** Our hypothesis is that Growth Hormone (GH) will not only target the peripheral tissue to stimulate weight and muscle growth which will maximize nutritional potential and improve overall quality of life. We theorize that this will occur through a multitude of factors: increased appetite, more constructive utilization of caloric intake and decreased catabolic signaling. The first aim will address changes in weight and lean body mass following the institution of GH therapy in adults with Cystic Fibrosis (CF) related wasting. The second aim will measure impact on quality of life of these individuals. Additionally, the third aim will monitor effects of GH therapy on diabetes and insulin sensitivity. Finally, the fourth aim will observe changes in the subjects underlying diagnosis of CF, specifically lung function, muscle strength and inflammatory state.

**Detailed Description:** The following is a more detailed description of the aims listed above:

Specific Aim 1: Measure change in weight in adults with CF related wasting following GH therapy.

1.1) Monitor weight gained or loss from baseline. 1.2) Assess changes in fat free mass from baseline by bioelectrical impedence analysis.

Specific Aim 2: Evaluate overall quality of life (QOL) in adults with CF related wasting treated with GH therapy.

2.1) Perform CF disease-specific and general QOL analysis via CF QOL questionnaires.

2.2) Monitor compliance with therapy via subject report.

Specific Aim 3: Monitor impact of GH therapy in relation to CF related diabetes onset or control.

3.1) Measure impact on insulin sensitivity in non-diabetes subjects 3.2) Observe change in exogenous insulin requirements and glycemic control in subjects with diabetes.

Specific Aim 4: Quantify impact of anabolic therapy on manifestations of underlying diagnosis associated with CF.

4.1) Observe changes in lung function from baseline during GH therapy. 4.2) Determine changes in overall muscle strength via hand grip and six minute walk.

4.3) Evaluate changes in serum markers.

### Conditions Module

**Conditions:**

- Cystic Fibrosis

**Keywords:**

- CF

### Design Module

#### Design Info

**Allocation:** NA

**Intervention Model:** SINGLE_GROUP

##### Masking Info

**Masking:** NONE

**Primary Purpose:** TREATMENT

#### Enrollment Info

**Count:** 5

**Type:** ACTUAL

**Phases:**

- PHASE1

**Study Type:** INTERVENTIONAL

### Arms Interventions Module

#### Arm Group 1

**Description:** Nutropin Aqueous (AQ):

Initiation treatment for adult males is 0.2mg/d and for women 0.4mg/d

**Intervention Names:**

- Drug: Nutropin AQ

**Label:** Growth Hormone Therapy

**Type:** EXPERIMENTAL

### Interventions

#### Intervention 1

**Arm Group Labels:**

- Growth Hormone Therapy

**Description:** Based on recommendations from the 2007 GH Deficiency Consensus Workshop on adult GH deficiency, the recommended initiation of treatment for adult males is 0.2mg/d and for women 0.4mg/d, with a titration upwards based on insulin-like growth factor (IGF-1) (product of GH stimulation at target tissues) levels and patient response. IGF-1 will be monitored at the 3,4,5 and 11 month intervals. For subjects under the age of 25 with an open epiphysis of the hand and/or wrist we will treat with the dose of 0.3mg/kg/week. Subjects will be on growth hormone for 8 months with a baseline visit prior to initiation of therapy and a 3 month follow-up visit after stopping therapy.

**Name:** Nutropin AQ

**Other Names:**

- Growth Hormone (GH)

**Type:** DRUG

### Outcomes Module

#### Primary Outcomes

**Measure:** Measure Change in Weight in Adults With Cystic Fibrosis (CF) Related Wasting Following Growth Hormone (GH) Therapy

**Time Frame:** 14 months

#### Secondary Outcomes

**Measure:** Evaluate Overall Quality of Life (QOL) in Adults With CF Related Wasting Treated With GH Therapy

**Time Frame:** 14 months

### Eligibility Module

**Eligibility Criteria:** Inclusion Criteria:

* Ability to provide written informed consent and comply with study assessments for the full duration of the study.
* Age \> 18 years
* Cystic fibrosis, diagnosed by either sweat chloride or genetic testing
* Less than 92% ideal body weight based on body mass index (BMI) of 22 for women and 23 for men
* Moderate or better pulmonary function (Forced Expiratory Volume (FEV1) \>40% of predicted).
* Agree to use an effective method of birth control to prevent pregnancy during the research study.

Women should not nurse (breast feed) a baby while on this study because Nutropin AQ may enter breast milk and possibly harm the child.

Exclusion Criteria:

* Pregnancy (positive pregnancy test) prior enrollment in the study
* Any other condition that the investigator believes would pose a significant hazard to the subject if the investigational therapy were initiated
* Participation in another simultaneous medical investigation or trial
* Pediatric patients
* Active neoplasm
* History of organ transplantation
* Prader Willi Syndrome who are severely obese or have severe respiratory impairment
* Patients with hepatic impairment resulting in abnormal coagulation studies (\>1.5 times normal reference range)
* Poorly controlled diabetes as determined by a Hemoglobin A1c greater than or equal to 9.0%.
* Individuals with electrocardiogram abnormality or cardiac pacing.

**Minimum Age:** 18 Years

**Sex:** ALL

**Standard Ages:**

- ADULT

- OLDER_ADULT

### Contacts Locations Module

#### Locations

**Location 1:**

**City:** Worcester

**Country:** United States

**Facility:** Umms/Ummhc

**State:** Massachusetts

**Zip:** 01655

#### Overall Officials

**Official 1:**

**Affiliation:** Unversity of Massachusetts Medical School

**Name:** Michael Stalvey, MD

**Role:** PRINCIPAL_INVESTIGATOR

### References Module

#### References

**Citation:** Thaker V, Carter B, Putman M. Recombinant growth hormone therapy for cystic fibrosis in children and young adults. Cochrane Database Syst Rev. 2021 Aug 23;8(8):CD008901. doi: 10.1002/14651858.CD008901.pub5.

**PMID:** 34424546

## Derived Section

### Condition Browse Module - Ancestors

- ID: D000010335

- Term: Pathologic Processes

- ID: D000010182

- Term: Pancreatic Diseases

- ID: D000004066

- Term: Digestive System Diseases

- ID: D000008171

- Term: Lung Diseases

- ID: D000012140

- Term: Respiratory Tract Diseases

- ID: D000030342

- Term: Genetic Diseases, Inborn

- ID: D000007232

- Term: Infant, Newborn, Diseases

### Condition Browse Module - Browse Branches

- Abbrev: BC06

- Name: Digestive System Diseases

- Abbrev: BC08

- Name: Respiratory Tract (Lung and Bronchial) Diseases

- Abbrev: BC16

- Name: Diseases and Abnormalities at or Before Birth

- Abbrev: All

- Name: All Conditions

- Abbrev: BC23

- Name: Symptoms and General Pathology

- Abbrev: BC18

- Name: Nutritional and Metabolic Diseases

- Abbrev: Rare

- Name: Rare Diseases

### Condition Browse Module - Browse Leaves

- ID: M6755

- Name: Cystic Fibrosis

- Relevance: HIGH

- As Found: Cystic Fibrosis

- ID: M8485

- Name: Fibrosis

- Relevance: HIGH

- As Found: Fibrosis

- ID: M21265

- Name: Wasting Syndrome

- Relevance: LOW

- As Found: Unknown

- ID: M5363

- Name: Cachexia

- Relevance: LOW

- As Found: Unknown

- ID: M13102

- Name: Pancreatic Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M7255

- Name: Digestive System Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M8883

- Name: Gastrointestinal Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M11168

- Name: Lung Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M14977

- Name: Respiratory Tract Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M23686

- Name: Genetic Diseases, Inborn

- Relevance: LOW

- As Found: Unknown

- ID: M10276

- Name: Infant, Newborn, Diseases

- Relevance: LOW

- As Found: Unknown

- ID: T1710

- Name: Cystic Fibrosis

- Relevance: HIGH

- As Found: Cystic Fibrosis

### Condition Browse Module - Meshes

- ID: D000003550

- Term: Cystic Fibrosis

- ID: D000005355

- Term: Fibrosis

### Intervention Browse Module - Ancestors

- ID: D000006730

- Term: Hormones, Hormone Substitutes, and Hormone Antagonists

- ID: D000045505

- Term: Physiological Effects of Drugs

### Intervention Browse Module - Browse Branches

- Abbrev: Hypo

- Name: Hypoglycemic Agents

- Abbrev: All

- Name: All Drugs and Chemicals

### Intervention Browse Module - Browse Leaves

- ID: M10365

- Name: Insulin

- Relevance: LOW

- As Found: Unknown

- ID: M16759

- Name: Tin Fluorides

- Relevance: LOW

- As Found: Unknown

- ID: M9789

- Name: Hormones

- Relevance: HIGH

- As Found: Loss

- ID: M173166

- Name: Insulin, Globin Zinc

- Relevance: LOW

- As Found: Unknown

- ID: M11900

- Name: Mitogens

- Relevance: LOW

- As Found: Unknown

- ID: M9788

- Name: Hormone Antagonists

- Relevance: LOW

- As Found: Unknown

### Intervention Browse Module - Meshes

- ID: D000006728

- Term: Hormones

### Misc Info Module

- Version Holder: 2024-05-24

## Results Section

### Adverse Events Module

#### Event Groups

**Group ID:** EG000

**Title:** Growth Hormone Therapy

**Description:** Nutropin AQ:

Initiation treatment for adult males is 0.2mg/d and for women 0.4mg/d

**ID:** EG000

**Other Num at Risk:** 5

**Serious Number At Risk:** 5

**Title:** Growth Hormone Therapy

**Frequency Threshold:** 0

## Results Section - Baseline Characteristics Module

### Denomination Counts

**Group ID:** BG000

**Value:** 5

**Units:** Participants

### Group

**ID:** BG000

**Title:** Growth Hormone Therapy

**Description:** Nutropin AQ:

Initiation treatment for adult males is 0.2mg/d and for women 0.4mg/d

### Measure

#### Measurement

**Group ID:** BG000

**Value:** 0

**Category Title:** <=18 years

#### Measurement

**Group ID:** BG000

**Value:** 5

**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:** 0

**Category Title:** Female

#### Measurement

**Group ID:** BG000

**Value:** 5

**Category Title:** Male

**Class Title:** 

### Measure

#### Measurement

**Group ID:** BG000

**Value:** 5

**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:** NUMBER
**Title:** Region of Enrollment
**Unit of Measure:** participants
## Results Section - More Information Module
### Certain Agreement
**PI Sponsor Employee:** True
### Point of Contact
**Email:** mstalvey@peds.uab.edu
**Organization:** UMass Medical School
**Phone:** 508-856-4280
**Title:** Michael Stalvey, MD, Principal Investigator
## Results Section - Outcome Measures Module
### Outcome Measure 1
### Outcome Measure 2
## Results Section
### Outcome Measures Module
#### Outcome Measure 1
#### Outcome Measure 2
## Results Section
### Outcome Measures Module
#### Outcome Measure 1
**Reporting Status:** POSTED
**Time Frame:** 14 months
**Title:** Measure Change in Weight in Adults With Cystic Fibrosis (CF) Related Wasting Following Growth Hormone (GH) Therapy
**Type:** PRIMARY
##### Group
**Description:** Nutropin Aqueous (AQ):

Initiation treatment for adult males is 0.2mg/d and for women 0.4mg/d
**ID:** OG000
**Title:** Growth Hormone Therapy
#### Outcome Measure 2
**Reporting Status:** NOT_POSTED
**Time Frame:** 14 months
**Title:** Evaluate Overall Quality of Life (QOL) in Adults With CF Related Wasting Treated With GH Therapy
**Type:** SECONDARY
### Participant Flow Module
#### Group
**Description:** Nutropin AQ:

Initiation treatment for adult males is 0.2mg/d and for women 0.4mg/d
**ID:** FG000
**Title:** Growth Hormone Therapy
#### Period
**Title:** Overall Study
##### Withdraw
**Type:** Lost to Follow-up
###### Reason
**Group ID:** FG000
**Number of Subjects:** 3
##### Milestone
**Type:** STARTED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 5
##### Milestone
**Type:** COMPLETED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 2
##### Milestone
**Type:** NOT COMPLETED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 3
**Has Results:** True