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Growth Hormone Therapy for Wasting in Cystic Fibrosis

Primary Purpose

Cystic Fibrosis

Status

Terminated

Phase

Phase 1

Locations

United States

Study Type

Interventional

Intervention

Nutropin AQ

About this trial

This is an interventional treatment trial for Cystic Fibrosis focused on measuring CF

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

Umms/Ummhc

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Growth Hormone Therapy

Arm Description

Nutropin Aqueous (AQ): Initiation treatment for adult males is 0.2mg/d and for women 0.4mg/d

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

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

Full Information

NCT ID

NCT00803179

First Posted

December 4, 2008

Last Updated

December 10, 2012

Sponsor

University of Massachusetts, Worcester

1. Study Identification

Unique Protocol Identification Number

NCT00803179

Brief Title

Growth Hormone Therapy for Wasting in Cystic Fibrosis

Official Title

Growth Hormone Therapy for Wasting in Cystic Fibrosis

Study Type

Interventional

2. Study Status

Record Verification Date

December 2012

Overall Recruitment Status

Terminated

Why Stopped

Poor enrollment, patients were lost to follow up

Study Start Date

November 2008 (undefined)

Primary Completion Date

March 2012 (Actual)

Study Completion Date

March 2012 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Principal Investigator

Name of the Sponsor

University of Massachusetts, Worcester

4. Oversight

Data Monitoring Committee

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Cystic Fibrosis

Keywords

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

5 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Growth Hormone Therapy

Arm Type

Experimental

Arm Description

Nutropin Aqueous (AQ): Initiation treatment for adult males is 0.2mg/d and for women 0.4mg/d

Intervention Type

Drug

Intervention Name(s)

Nutropin AQ

Other Intervention Name(s)

Growth Hormone (GH)

Intervention 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.

Primary Outcome Measure Information:

Title

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

Time Frame

14 months

Secondary Outcome Measure Information:

Title

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

Time Frame

14 months

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

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.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Michael Stalvey, MD

Organizational Affiliation

Unversity of Massachusetts Medical School

Official's Role

Principal Investigator

Facility Information:

Facility Name

Umms/Ummhc

City

Worcester

State/Province

Massachusetts

ZIP/Postal Code

01655

Country

United States

12. IPD Sharing Statement

Citations:

PubMed Identifier

34424546

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.

Results Reference

derived

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Growth Hormone Therapy for Wasting in Cystic Fibrosis

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