Growth Hormone Treatment Study in Children With Cystic Fibrosis

The recruitment status of this study is unknown because the information has not been verified recently.
Verified November 2005 by University of Texas Southwestern Medical Center.
Recruitment status was  Active, not recruiting
Sponsor:
Collaborator:
Genentech, Inc.
Information provided by:
University of Texas Southwestern Medical Center
ClinicalTrials.gov Identifier:
NCT00256555
First received: November 17, 2005
Last updated: NA
Last verified: November 2005
History: No changes posted
  Purpose

Cystic fibrosis (CF) is a catabolic condition, and children affected with this disease frequently have problems with growth, despite adequate nutrition. We hypothesize that the anabolic effects of growth hormone (GH) will improve the height and weight of CF patients and thereby improve their clinical status and their quality of life. We further hypothesize that these effects will be sustained for at least one year after GH treatment is complete.


Condition Intervention Phase
Cystic Fibrosis
Drug: Nutropin AQ
Phase 2
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Crossover Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: GROWTH HORMONE USE IN CYSTIC FIBROSIS - A MULTICENTER STUDY

Resource links provided by NLM:


Further study details as provided by University of Texas Southwestern Medical Center:

Estimated Enrollment: 60
Study Start Date: February 2000
Detailed Description:

We will test our hypotheses by recruiting 40 CF children from five CF centers (8 per center) across the country. Patients will be randomly assigned to receive treatment with GH (0.3 mg/kg/wk) during the first 12 months, or during the second 12 months. All subjects will be followed every three months for the entire 24 months. Growth data and PFT data from the year prior to study will be obtained from the medical record for each subject. Our specific aims include:

  1. To determine the effect of GH on height, height velocity, body weight and lean body mass. We will measure height and weight using a standardized stadiometer and scale, respectively, every three months during the study. From these measurements we will calculate height and weight velocity and height and weight Z score. Lean body mass (LBM) will be measured by DEXA every six months. Baseline height and weight will be analyzed as covariates to determine whether children whose height and/or weight are at the lowest percentiles achieve greater improvement in height and weight velocity and lean body mass. This specific aim tests the hypothesis that GH significantly improves height, height velocity, weight, weight velocity and lean body mass in CF children irrespective of growth prior to starting the drug.
  2. To determine the effect of GH on pulmonary function. Pulmonary function tests, including FEV1, FVC and PImax and PEmax (for estimation of respiratory muscle strength), will be measured at baseline and every 6 months in all subjects. We will document inpatient admissions and outpatient antibiotic therapy during the study. Baseline pulmonary function will be analyzed as a covariate to determine whether children with good pulmonary function achieve greater benefit than children with poor pulmonary function. This specific aim tests the hypothesis that GH use improves pulmonary function in CF children regardless of level of pulmonary functions prior to using the drug.
  3. To determine if GH use in CF patients positively impacts quality of life. At baseline and every six months, we will administer a 15 minute questionnaire to both the parent and patient to assess quality of life. These questionnaires entitled "The Cystic Fibrosis Questionnaire" have been recently developed and tested for quality. They are specific for CF patients and have been approved by the National CF Foundation. This specific aim will test the hypothesis that GH use improves quality of life in CF patients.
  4. To determine if the clinical improvement obtained from GH use is sustained. Those patients treated with GH during the first 12 months will be followed for 12 months after treatment to determine if the outcome variables remain better than baseline after growth hormone therapy is discontinued. This specific aim tests the hypothesis that GH use results in sustained improvement in height velocity, weight velocity, lean body mass, pulmonary function and quality of life.
  Eligibility

Ages Eligible for Study:   5 Years to 12 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • prepubertal CF patients (ages 5-12)
  • capable of performing pulmonary function tests
  • less than the 25th percentile for age and sex normal values for height and/or weight.

Exclusion Criteria:

  • previous diagnosis with diabetes
  • previous insulin requirement
  • inability to perform pulmonary function testing
  • colonization with burkholderia cepacia
  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: NCT00256555

Sponsors and Collaborators
University of Texas Southwestern Medical Center
Genentech, Inc.
Investigators
Principal Investigator: Dana S. Hardin, M.D. University of Texas Southwestern Medical Center
  More Information

Publications:
ClinicalTrials.gov Identifier: NCT00256555     History of Changes
Other Study ID Numbers: 999-M02
Study First Received: November 17, 2005
Last Updated: November 17, 2005
Health Authority: United States: Food and Drug Administration

Additional relevant MeSH terms:
Cystic Fibrosis
Fibrosis
Digestive System Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Lung Diseases
Pancreatic Diseases
Pathologic Processes
Respiratory Tract Diseases

ClinicalTrials.gov processed this record on October 23, 2014