Growth Hormone Therapy for Wasting in Cystic Fibrosis - Full Text View

Sponsored by:	University of Massachusetts
Information provided by:	University of Massachusetts
ClinicalTrials.gov Identifier:	NCT00803179

Purpose

Our hypothesis is that 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 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 I

Genetics Home Reference related topics: cystic fibrosis

MedlinePlus related topics: Cystic Fibrosis

Drug Information available for: Somatropin Somatotropin

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment, Non-Randomized, Open Label, Single Group Assignment, Safety/Efficacy Study
Official Title:	Growth Hormone Therapy for Wasting in Cystic Fibrosis

Further study details as provided by University of Massachusetts:

Primary Outcome Measures:

Measure change in weight in adults with CF related wasting following 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 ]

Estimated Enrollment:	30
Study Start Date:	November 2008
Estimated Study Completion Date:	November 2011
Estimated Primary Completion Date:	November 2010 (Final data collection date for primary outcome measure)

Intervention Details:

Based on recommendations from the 2007 GH Deficiency Consensus Worskshop 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 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.

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 BMI of 22 for women and 23 for men
Moderate or better pulmonary function (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 (>1.5X 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

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

Locations

United States, Massachusetts
UMMS/UMMHC	Recruiting
Worcester, Massachusetts, United States, 01655
Contact: Dawn Baker, MSN 508-334-2339 dawn.baker@umassmed.edu
Contact: Michael Stalvey, MD 508-856-4280 michael.stalvey@umassmed.edu
Principal Investigator: Michael Stalvey, MD

Sponsors and Collaborators

University of Massachusetts

Investigators

Principal Investigator:

Michael Stalvey, MD

Unversity of Massachusetts Medical School

More Information

No publications provided

Responsible Party:	University of Massachusetts Medical School ( Michael Stalvey, MD )
Study ID Numbers:	900005
Study First Received:	December 4, 2008
Last Updated:	December 4, 2008
ClinicalTrials.gov Identifier:	NCT00803179 History of Changes
Health Authority:	United States: Institutional Review Board

Keywords provided by University of Massachusetts:

CF

Study placed in the following topic categories:

Digestive System Diseases
Genetic Diseases, Inborn
Respiratory Tract Diseases
Cystic Fibrosis
Fibrosis
Hormone Antagonists

Lung Diseases
Hormones, Hormone Substitutes, and Hormone Antagonists
Infant, Newborn, Diseases
Pancreatic Diseases
Hormones

Additional relevant MeSH terms:

Fibrosis
Physiological Effects of Drugs
Hormones, Hormone Substitutes, and Hormone Antagonists
Hormones
Pharmacologic Actions
Digestive System Diseases
Pathologic Processes

Cystic Fibrosis
Respiratory Tract Diseases
Genetic Diseases, Inborn
Lung Diseases
Pancreatic Diseases
Infant, Newborn, Diseases

ClinicalTrials.gov processed this record on May 07, 2009