We're building a better ClinicalTrials.gov. Check it out and tell us what you think!
ClinicalTrials.gov Menu

Growth Hormone Use in Cystic Fibrosis - a Multicenter Study

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT00005112
Recruitment Status : Completed
First Posted : April 17, 2000
Last Update Posted : June 24, 2005
Genentech, Inc.
Information provided by:
National Center for Research Resources (NCRR)

Brief Summary:
Cystic Fibrosis (CF) is the most common lethal genetic disorder in America. Previous studies by our group and others have shown that human recombinant growth hormone (GH) improves height velocity, weight velocity, lean body mass (LBM) and pulmonary function. These positive results have prompted us to ask further questions regarding GH use in CF including: a) Do patients with better baseline body weight and pulmonary function derive more benefit from treatment than those with worse weight and pulmonary function?, b) Does GH use improve the patient's quality of life?, c) Once GH is discontinued, are the positive effects sustained? We hypothesize that GH treatment in CF patients will improve their clinical status and their quality of life. We further hypothesize that these effects will be present regardless of baseline body weight or pulmonary function, and that positive outcome will be sustained for at least one year after GH treatment is discontinued. To test our hypothesis, we will recruit 40 prepubertal children from five CF centers across the United States (8 per center). Patients will be randomly assigned to receive treatment with GH (0.3mg/kg/wk) during either the first or the second year. All subjects will be seen every three months. We will evaluate the following parameters every three months: 1) height, height velocity and Z-score, 2) body weight and weight velocity. Every six months we will measure: 1) lean body mass utilizing DEXA, 2) pulmonary function, including measurement of respiratory muscle strength (peak inspiratory and peak expiratory pressure), 3) quality of life (QOL), quantitated from QOL forms specific for CF ("The Cystic Fibrosis Questionnaire"). After one year of study, subjects will "cross-over" to the other treatment arm. This 24 month study will allow us to statistically compare outcome measures in 20 treated and 20 nontreated subjects from multiple centers, and will allow us to assess sustained effect in the 20 subjects who receive GH during the first year, by comparing their results to results obtained during the year post treatment.

Condition or disease Intervention/treatment Phase
Cystic Fibrosis Drug: Human recombinant growth hormone Phase 3

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Allocation: Randomized
Primary Purpose: Treatment

Resource links provided by the National Library of Medicine

Information from the National Library of Medicine

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, Learn About Clinical Studies.

Layout table for eligibility information
Ages Eligible for Study:   5 Years to 12 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • CF patients ages 5-12 who are less than the 25th percentile for age and sex normal values for height and/or weight

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00005112

Layout table for location information
United States, Texas
University of Texas Medical School
Houston, Texas, United States, 77030
United States, Utah
University of Utah, Dept of Pediatric Endocrinology
Salt Lake City, Utah, United States, 84132
Sponsors and Collaborators
National Center for Research Resources (NCRR)
Genentech, Inc.
Layout table for investigator information
Principal Investigator: Dana Hardin, M.D.
Layout table for additonal information
ClinicalTrials.gov Identifier: NCT00005112    
Obsolete Identifiers: NCT00000109
Other Study ID Numbers: NCRR-M01RR02558-0111
M01RR002558 ( U.S. NIH Grant/Contract )
First Posted: April 17, 2000    Key Record Dates
Last Update Posted: June 24, 2005
Last Verified: December 2003
Additional relevant MeSH terms:
Layout table for MeSH terms
Cystic Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs