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Growth Hormone Use in Cystic Fibrosis - a Multicenter Study

This study has been completed.

Sponsors and Collaborators: National Center for Research Resources (NCRR)
Genentech
Information provided by: National Center for Research Resources (NCRR)
ClinicalTrials.gov Identifier: NCT00005112
  Purpose

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 Intervention Phase
Cystic Fibrosis
Drug: Human recombinant growth hormone
Phase III

Genetics Home Reference related topics:   cystic fibrosis   

MedlinePlus related topics:   Cystic Fibrosis   

Drug Information available for:   Somatotropin    Somatropin   

U.S. FDA Resources

Study Type:   Interventional
Study Design:   Treatment, Randomized
  Eligibility
Ages Eligible for Study:   5 Years to 12 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

Criteria

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
  Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00005112

Locations
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

Investigators
Principal Investigator:     Dana Hardin, M.D.    
  More Information


Study ID Numbers:   NCRR-M01RR02558-0111, M01RR02558
First Received:   April 15, 2000
Last Updated:   June 23, 2005
ClinicalTrials.gov Identifier:   NCT00005112
Health Authority:   United States: Federal Government

Study placed in the following topic categories:
Digestive System Diseases
Genetic Diseases, Inborn
Respiratory Tract Diseases
Cystic Fibrosis
Fibrosis
Lung Diseases
Infant, Newborn, Diseases
Pancreatic Diseases
Cystic fibrosis

Additional relevant MeSH terms:
Pathologic Processes

ClinicalTrials.gov processed this record on December 03, 2008




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