Ignite Creation Date:
2024-05-05 @ 12:09 PM
Last Modification Date:
2024-10-26 @ 9:21 AM
Study NCT ID:
NCT00256555
Status:
WITHDRAWN
Last Update Posted:
2019-01-10
First Post:
2005-11-17
Brief Title:
Growth Hormone Treatment Study in Children With Cystic Fibrosis
Sponsor:
University of Texas Southwestern Medical Center
Organization:
University of Texas Southwestern Medical Center
Study Overview
Official Title:
GROWTH HORMONE USE IN CYSTIC FIBROSIS - A MULTICENTER STUDY
Status:
WITHDRAWN
Status Verified Date:
2019-01
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
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 03 mgkgwk 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 andor 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
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 andor 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 Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None