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Growth Hormone Treatment Study in Children With Cystic Fibrosis

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ClinicalTrials.gov Identifier: NCT00256555
Recruitment Status : Withdrawn
First Posted : November 21, 2005
Last Update Posted : January 10, 2019
Sponsor:
Collaborator:
Genentech, Inc.
Information provided by (Responsible Party):
University of Texas Southwestern Medical Center

Tracking Information
First Submitted Date  ICMJE November 17, 2005
First Posted Date  ICMJE November 21, 2005
Last Update Posted Date January 10, 2019
Actual Study Start Date  ICMJE February 2000
Actual Primary Completion Date December 12, 2018   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE Not Provided
Original Primary Outcome Measures  ICMJE Not Provided
Change History Complete list of historical versions of study NCT00256555 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Growth Hormone Treatment Study in Children With Cystic Fibrosis
Official Title  ICMJE GROWTH HORMONE USE IN CYSTIC FIBROSIS - A MULTICENTER STUDY
Brief Summary 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.
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.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Phase 3
Study Design  ICMJE Intervention Model: Crossover Assignment
Intervention Model Description:
00
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Cystic Fibrosis
Intervention  ICMJE Drug: Nutropin AQ
Study Arms  ICMJE Not Provided
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Withdrawn
Actual Enrollment  ICMJE
 (submitted: January 8, 2019)
0
Original Enrollment  ICMJE
 (submitted: November 17, 2005)
60
Actual Study Completion Date  ICMJE December 12, 2018
Actual Primary Completion Date December 12, 2018   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 5 Years to 12 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Not Provided
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT00256555
Other Study ID Numbers  ICMJE 999-M02
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party University of Texas Southwestern Medical Center
Study Sponsor  ICMJE University of Texas Southwestern Medical Center
Collaborators  ICMJE Genentech, Inc.
Investigators  ICMJE
Principal Investigator: Dana S. Hardin, M.D. University of Texas Southwestern Medical Center
PRS Account University of Texas Southwestern Medical Center
Verification Date January 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP