Cystic FibrosisPhase 2/3NCT00256555
Growth Hormone Treatment Study in Children With Cystic Fibrosis
A Phase 2/3 study testing Nutropin AQ in people with Cystic Fibrosis, run by University of Texas Southwestern Medical Center.
Trial 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… Read more about this study
- Who is it for?
- Ages 5 to 12 years
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- Nutropin AQ
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- Feb 2000to 12 Dec 2018
- Phase
- Phase 2/3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 5 to 12 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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.
Who cannot join
- previous diagnosis with diabetes
- previous insulin requirement
- inability to perform pulmonary function testing
- colonization with burkholderia cepacia
About this study
Official title: GROWTH HORMONE USE IN CYSTIC FIBROSIS - A MULTICENTER STUDY
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.
Read the 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:
- 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.
- 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.
- 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.
- 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.
- Conditions
- Cystic Fibrosis
- Being tested
- Nutropin AQ
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Nutropin AQDrug
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Nov 2005 (estimated)
- Study startFeb 2000
- Primary completion12 Dec 2018
- Study completion12 Dec 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (6)Papers the sponsor has linked to this study.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- More trials run by University of Texas Southwestern Medical CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2/3
- Primary purpose
- Treatment
- Intervention model
- Crossover
- Model description
- 00
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Texas Southwestern Medical Center
- Sponsor type
- University, hospital or other
- Collaborators
- Genentech, Inc.
- Responsible party
- Sponsor
- Investigators
- Dana S. Hardin, M.D., Principal investigator, University of Texas Southwestern Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00256555
- Sponsor's protocol ID
- 999-M02
- Status last verified
- Jan 2019
- First submitted
- 17 Nov 2005
- Last updated
- 10 Jan 2019
- Results posted
- No
Publications
Hardin DS. GH improves growth and clinical status in children with cystic fibrosis -- a review of published studies. Eur J Endocrinol. 2004 Aug;151 Suppl 1:S81-5. doi: 10.1530/eje.0.151s081.
PubMed 15339250Hardin DS. Growth hormone treatment in children with cystic fibrosis. Minerva Pediatr. 2002 Oct;54(5):365-71.
PubMed 12244275Hardin DS. Growth problems and growth hormone treatment in children with cystic fibrosis. J Pediatr Endocrinol Metab. 2002 May;15 Suppl 2:731-5.
PubMed 12092687Hardin DS, Ellis KJ, Dyson M, Rice J, McConnell R, Seilheimer DK. Growth hormone improves clinical status in prepubertal children with cystic fibrosis: results of a randomized controlled trial. J Pediatr. 2001 Nov;139(5):636-42. doi: 10.1067/mpd.2001.117578.
PubMed 11713439Hardin DS, Ellis KJ, Dyson M, Rice J, McConnell R, Seilheimer DK. Growth hormone decreases protein catabolism in children with cystic fibrosis. J Clin Endocrinol Metab. 2001 Sep;86(9):4424-8. doi: 10.1210/jcem.86.9.7822.
PubMed 11549686Hardin DS, Rice J, Ahn C, Ferkol T, Howenstine M, Spears S, Prestidge C, Seilheimer DK, Shepherd R. Growth hormone treatment enhances nutrition and growth in children with cystic fibrosis receiving enteral nutrition. J Pediatr. 2005 Mar;146(3):324-8. doi: 10.1016/j.jpeds.2004.10.037.
PubMed 15756212
Study at a glance
From the registry record, last updated by the sponsor on 10 Jan 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- GROWTH HORMONE USE IN CYSTIC FIBROSIS - A MULTICENTER STUDY
- ClinicalTrials.gov ID
- NCT00256555
- Acronym / sponsor ID
- 999-M02
- Conditions
- Cystic Fibrosis
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Withdrawn
- Enrolment
- 0 participants (actual)
- Lead sponsor
- University of Texas Southwestern Medical CenterUniversity, hospital or other
- Collaborators
- Genentech, Inc.
- Study start
- Feb 2000 (actual)
- Primary completion
- 12 Dec 2018 (actual)
- Study completion
- 12 Dec 2018 (actual)
- First posted
- 21 Nov 2005 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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:
- 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.
- 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.
- 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.
- 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 design
- Primary purpose
- Treatment
- Intervention model
- Crossover
- Masking
- None (open label)
- Model description
- 00
Who may qualify
- Ages
- 5 to 12 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- 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.
Key exclusion criteria (4 listed)
- previous diagnosis with diabetes
- previous insulin requirement
- inability to perform pulmonary function testing
- colonization with burkholderia cepacia
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Nutropin AQ | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Jan 2019.
- Background: PubMed 15339250Hardin DS. GH improves growth and clinical status in children with cystic fibrosis -- a review of published studies. Eur J Endocrinol. 2004 Aug;151 Suppl 1:S81-5. doi: 10.1530/eje.0.151s081.
- Background: PubMed 12244275Hardin DS. Growth hormone treatment in children with cystic fibrosis. Minerva Pediatr. 2002 Oct;54(5):365-71.
- Background: PubMed 12092687Hardin DS. Growth problems and growth hormone treatment in children with cystic fibrosis. J Pediatr Endocrinol Metab. 2002 May;15 Suppl 2:731-5.
- Background: PubMed 11713439Hardin DS, Ellis KJ, Dyson M, Rice J, McConnell R, Seilheimer DK. Growth hormone improves clinical status in prepubertal children with cystic fibrosis: results of a randomized controlled trial. J Pediatr. 2001 Nov;139(5):636-42. doi: 10.1067/mpd.2001.117578.
- Background: PubMed 11549686Hardin DS, Ellis KJ, Dyson M, Rice J, McConnell R, Seilheimer DK. Growth hormone decreases protein catabolism in children with cystic fibrosis. J Clin Endocrinol Metab. 2001 Sep;86(9):4424-8. doi: 10.1210/jcem.86.9.7822.
- Results publication: PubMed 15756212Hardin DS, Rice J, Ahn C, Ferkol T, Howenstine M, Spears S, Prestidge C, Seilheimer DK, Shepherd R. Growth hormone treatment enhances nutrition and growth in children with cystic fibrosis receiving enteral nutrition. J Pediatr. 2005 Mar;146(3):324-8. doi: 10.1016/j.jpeds.2004.10.037.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Dana S. Hardin, M.D. principal investigator
University of Texas Southwestern Medical Center
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Nutropin AQ in people with Cystic Fibrosis, run by University of Texas Southwestern Medical Center.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 10 Jan 2019. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00256555, which also lists 6 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Jan 2019.