Growth Hormone Therapy for Wasting in Cystic Fibrosis

This study has been terminated.
(Poor enrollment, patients were lost to follow up)
Sponsor:
Information provided by (Responsible Party):
Michael Stalvey, University of Massachusetts, Worcester
ClinicalTrials.gov Identifier:
NCT00803179
First received: December 4, 2008
Last updated: December 10, 2012
Last verified: December 2012
  Purpose

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.


Condition Intervention Phase
Cystic Fibrosis
Drug: Nutropin AQ
Phase 1

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Growth Hormone Therapy for Wasting in Cystic Fibrosis

Resource links provided by NLM:


Further study details as provided by University of Massachusetts, Worcester:

Primary Outcome Measures:
  • Measure Change in Weight in Adults With Cystic Fibrosis (CF) Related Wasting Following Growth Hormone (GH) Therapy [ Time Frame: 14 months ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Evaluate Overall Quality of Life (QOL) in Adults With CF Related Wasting Treated With GH Therapy [ Time Frame: 14 months ] [ Designated as safety issue: No ]

Enrollment: 5
Study Start Date: November 2008
Study Completion Date: March 2012
Primary Completion Date: March 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Growth Hormone Therapy

Nutropin Aqueous (AQ):

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

Drug: Nutropin AQ
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.
Other Name: Growth Hormone (GH)

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.

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00803179

Locations
United States, Massachusetts
Umms/Ummhc
Worcester, Massachusetts, United States, 01655
Sponsors and Collaborators
University of Massachusetts, Worcester
Investigators
Principal Investigator: Michael Stalvey, MD Unversity of Massachusetts Medical School
  More Information

No publications provided

Responsible Party: Michael Stalvey, Study Principle Investigator, University of Massachusetts, Worcester
ClinicalTrials.gov Identifier: NCT00803179     History of Changes
Other Study ID Numbers: 900005
Study First Received: December 4, 2008
Results First Received: November 13, 2012
Last Updated: December 10, 2012
Health Authority: United States: Institutional Review Board

Keywords provided by University of Massachusetts, Worcester:
CF

Additional relevant MeSH terms:
Cystic Fibrosis
Fibrosis
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Pathologic Processes
Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs
Pharmacologic Actions

ClinicalTrials.gov processed this record on August 26, 2014