Growth Hormone Use in Cystic Fibrosis - a Multicenter Study

This study has been completed.
Sponsor:
Collaborator:
Genentech, Inc.
Information provided by:
National Center for Research Resources (NCRR)
ClinicalTrials.gov Identifier:
NCT00005112
First received: April 15, 2000
Last updated: June 23, 2005
Last verified: December 2003
  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 3

Study Type: Interventional
Study Design: Allocation: Randomized
Primary Purpose: Treatment

Resource links provided by NLM:


Further study details as provided by National Center for Research Resources (NCRR):

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

No publications provided

ClinicalTrials.gov Identifier: NCT00005112     History of Changes
Obsolete Identifiers: NCT00000109
Other Study ID Numbers: NCRR-M01RR02558-0111, M01RR02558
Study First Received: April 15, 2000
Last Updated: June 23, 2005
Health Authority: United States: Federal Government

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

ClinicalTrials.gov processed this record on October 19, 2014