Chronic DiseasePhase 3NCT00813189
Efficacy Study of Recombinant Growth Hormone on Muscle Function in Children Long-term Treated With Glucocorticoid
A Phase 3 study testing GH treatment in people with Chronic Disease and Glucocorticoid Therapy, Response to, run by Association REMEDE at 1 site in France.
Trial summary
Children suffering from chronic disease and receiving long-term glucocorticoid therapy suffer over years from severe growth retardation and profoundly altered body composition. They consist in a marked increase in fat mass and a decrease in lean body mass. Read more about this study
- Who is it for?
- Ages 6 years and older
- Conditions the study is for
- Chronic Disease, Glucocorticoid Therapy, Response to, Growth Retardation
- What is being tested?
- GH treatment
- Type of treatment
- Injection or infusion, Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 1 siteFrance
- Study dates
- Apr 2005to May 2012
- Phase
- Phase 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 6 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Measured Height below -2 SD
- Bone age below 13 years for a boy and below 11 years for a girl
- Glucocorticosteroid treatment for 12 months at least
- Glucocorticosteroid dose above or equal to 0.2 mg/kg/day of prednisone or equivalent dose over the last 12 months
- Glucocorticosteroid treatment is anticipated to be sustained for 1 more year at least
- The child benefits of the French social security cover
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- Child who has never been treated by GH.
- Evidence of a personally signed and dated informed consent document indicating that the patient's parents/guardians and from the patient himself/herself if he/she is able to receive and understand the information have been informed of all pertinent aspects of the study.
- No glucose intolerance or diabetes mellitus on an Oral Glucose Tolerance Test dated less than 3 months
Who cannot join
- Severe acute or chronic medical or psychiatric condition or laboratory abnormality that may increase the risk associated with trial participation or investigational product administration or may interfere with the interpretation of trial results and, in the judgment of the investigator, would make the subject inappropriate for entry into this trial.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Effects of Growth Hormone on Corticoid Myopathy in Children With Chronic Disease: Effects on Muscle Mass and Strength
Children suffering from chronic disease and receiving long-term glucocorticoid therapy suffer over years from severe growth retardation and profoundly altered body composition. They consist in a marked increase in fat mass and a decrease in lean body mass. Published studies have shown that Growth Hormone (GH) treatment in children with Juvenile Idiopathic Arthritis can improve body composition by increasing lean mass and by preventing increase in fat mass. The aim of the present protocol is to evaluate whether the increase in lean body mass observed during GH treatment is associated with changes in muscle strength and mass.
In order to be able to evaluate the effect of GH on the muscle a comparative group is needed. Therefore it will be proposed to delay in a group of patients the start of Growth Hormone(GH) treatment by 6 months. As most publications have shown a maximum effect of GH within the first year of treatment, six months should be enough to evaluate short-term effect of GH on the muscle. Therefore, this study will be a randomized trial: immediate start of Growth Hormone (GH) treatment versus start of Growth Hormone treatment 6 months later. After 6 months all children will be treated with GH. Therefore, the follow-up will be one year after baseline.
Read the detailed description
The aim of the present protocol is to evaluate the effects of GH treatment in long-term steroid treated children, on muscle mass, and muscle strength. It will be an open, randomized, controlled, 2-parallel group study. The follow-up in this study will be one year. The objective of this study will be to evaluate short-term effects of GH treatment on muscle mass, muscle strength and body composition in children suffering from various diseases requiring steroid therapy. Expected pathologies are juvenile idiopathic arthritis, nephrotic syndrome, uveitis, systemic diseases and organ transplantation. Changes in muscle mass will be assessed by measuring the muscle and sub-cutaneous fat on the cross sectional area of the thigh by MRI. MRI offer the advantage of non-invasive technique, allowing serial and accurate measurements.Muscle strength will be performed by serial muscular testing of different muscular groups. Body composition will be assessed by DEXA. DEXA allows rapid, accurate and highly reproductible determination not only of bone mass but also of lean and fat mass of the whole body, with very low radiation exposure.It appears to be the most sensitive method for assessment of muscle wasting as well as of fat repartition that contribute to Cushing's appearance in steroid treated patients.
The present study will be performed in children with growth retardation related to long-term glucocorticoid treatment These patients had never been treated with GH. In order to be able to evaluate the effect of GH on the muscle a comparative group is needed. Therefore it will be proposed in the study to delay in a group of patient the start of GH treatment by 6 months. Six months should be enough to evaluate short-term effect of GH on the muscle and most publication have shown a maximum effect of GH within the first year of treatment. Therefore, this study will be a randomized trial: immediate start of GH treatment versus start of GH treatment 6 months later. After 6 months all children will be treated with GH. The dose administered in the present trial will be a GH dose already tested in glucocorticoid treated children (0.46 mg/kg/week)
- Being tested
- GH treatment
- Keywords
- glucocorticoid therapy, muscle function, growth hormone treatment
- Medical subject headings
- Chronic Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GH treatmentDevice
GH treatment will be administered at a weekly dose of 0.46 mg/kg/ week, divided into seven daily subcutaneous injections. Subcutaneous injections should be given slowly, in the thigh. In order to prevent lipoatrophy, the injection site should be varied. The injection should be given at bedtime.
| Study group | Role | What they receive |
|---|---|---|
| 1 : early start (GH treatment) group | Experimental | in the early start group, patients were treated with growth hormone for one year immediately after randomisation Receives: Device: GH treatment |
| 2 :delayed start (GH treatment) group | No intervention | in the delayed start group patients took GH treatment for 1 year , 6 months after randomisation |
What is being measured
The primary outcome is the main question the study is designed to answer.
The main criteria of efficacy will be - the mean strength changes assessed as a composite score (mean of the relative changes of the ten muscle functions tested): ∆ strength as % of baseline values :6 months-baseline.
Measured: 6 months
Secondary outcomes (1)
Strength changes assessed as a composite score (mean of the relative changes of the ten muscle functions tested) as % of baseline values : 12 months-baseline and 12 months- 6 months, 18 months- 6 months for group B patients
Measured: 18 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Dec 2008 (estimated)
- Study startApr 2005
- Primary completionJun 2010
- Study completionMay 2012
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Chronic DiseaseStudies of the same condition, open and under way.
- 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 30 participants (actual)
Sponsor and investigators
- Lead sponsor
- Association REMEDE
- Sponsor type
- University, hospital or other
- Collaborators
- Pfizer
- Responsible party
- Principal investigator, Dominique SIMON, Association REMEDE
- Investigators
- Dominique SIMON, PHD, Principal investigator, Hopital Robert -Debré, Assistance Publique, Hopitaux de Paris
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00813189
- Sponsor's protocol ID
- 2004/64
- Status last verified
- May 2015
- First submitted
- 19 Dec 2008
- Last updated
- 5 May 2015
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 May 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Effects of Growth Hormone on Corticoid Myopathy in Children With Chronic Disease: Effects on Muscle Mass and Strength
- ClinicalTrials.gov ID
- NCT00813189
- Acronym / sponsor ID
- 2004/64
- Keywords
- glucocorticoid therapy, muscle function, growth hormone treatment
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 30 participants (actual)
- Lead sponsor
- Association REMEDEUniversity, hospital or other
- Collaborators
- Pfizer
- Study start
- Apr 2005
- Primary completion
- Jun 2010 (actual)
- Study completion
- May 2012 (actual)
- First posted
- 22 Dec 2008 (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.
Children suffering from chronic disease and receiving long-term glucocorticoid therapy suffer over years from severe growth retardation and profoundly altered body composition. They consist in a marked increase in fat mass and a decrease in lean body mass. Published studies have shown that Growth Hormone (GH) treatment in children with Juvenile Idiopathic Arthritis can improve body composition by increasing lean mass and by preventing increase in fat mass. The aim of the present protocol is to evaluate whether the increase in lean body mass observed during GH treatment is associated with changes in muscle strength and mass.
In order to be able to evaluate the effect of GH on the muscle a comparative group is needed. Therefore it will be proposed to delay in a group of patients the start of Growth Hormone(GH) treatment by 6 months. As most publications have shown a maximum effect of GH within the first year of treatment, six months should be enough to evaluate short-term effect of GH on the muscle. Therefore, this study will be a randomized trial: immediate start of Growth Hormone (GH) treatment versus start of Growth Hormone treatment 6 months later. After 6 months all children will be treated with GH. Therefore, the follow-up will be one year after baseline.
Detailed description
The aim of the present protocol is to evaluate the effects of GH treatment in long-term steroid treated children, on muscle mass, and muscle strength. It will be an open, randomized, controlled, 2-parallel group study. The follow-up in this study will be one year. The objective of this study will be to evaluate short-term effects of GH treatment on muscle mass, muscle strength and body composition in children suffering from various diseases requiring steroid therapy. Expected pathologies are juvenile idiopathic arthritis, nephrotic syndrome, uveitis, systemic diseases and organ transplantation. Changes in muscle mass will be assessed by measuring the muscle and sub-cutaneous fat on the cross sectional area of the thigh by MRI. MRI offer the advantage of non-invasive technique, allowing serial and accurate measurements.Muscle strength will be performed by serial muscular testing of different muscular groups. Body composition will be assessed by DEXA. DEXA allows rapid, accurate and highly reproductible determination not only of bone mass but also of lean and fat mass of the whole body, with very low radiation exposure.It appears to be the most sensitive method for assessment of muscle wasting as well as of fat repartition that contribute to Cushing's appearance in steroid treated patients.
The present study will be performed in children with growth retardation related to long-term glucocorticoid treatment These patients had never been treated with GH. In order to be able to evaluate the effect of GH on the muscle a comparative group is needed. Therefore it will be proposed in the study to delay in a group of patient the start of GH treatment by 6 months. Six months should be enough to evaluate short-term effect of GH on the muscle and most publication have shown a maximum effect of GH within the first year of treatment. Therefore, this study will be a randomized trial: immediate start of GH treatment versus start of GH treatment 6 months later. After 6 months all children will be treated with GH. The dose administered in the present trial will be a GH dose already tested in glucocorticoid treated children (0.46 mg/kg/week)
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 1 : early start (GH treatment) group | Experimental | Device: GH treatmentin the early start group, patients were treated with growth hormone for one year immediately after randomisation |
| 2 :delayed start (GH treatment) group | No intervention | –in the delayed start group patients took GH treatment for 1 year , 6 months after randomisation |
Who may qualify
- Ages
- 6 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (9 listed)
- Measured Height below -2 SD
- Bone age below 13 years for a boy and below 11 years for a girl
- Glucocorticosteroid treatment for 12 months at least
- Glucocorticosteroid dose above or equal to 0.2 mg/kg/day of prednisone or equivalent dose over the last 12 months
- Glucocorticosteroid treatment is anticipated to be sustained for 1 more year at least
Key exclusion criteria (1 listed)
- Severe acute or chronic medical or psychiatric condition or laboratory abnormality that may increase the risk associated with trial participation or investigational product administration or may interfere with the interpretation of trial results and, in the judgment of the investigator, would make the subject inappropriate for entry into this trial.
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 |
|---|---|---|
| GH treatmentGH treatment will be administered at a weekly dose of 0.46 mg/kg/ week, divided into seven daily subcutaneous injections. Subcutaneous injections should be given slowly, in the thigh. In order to prevent lipoatrophy, the injection site should be varied. The injection should be given at bedtime. | Device | 1 : early start (GH treatment) group |
Primary outcome
The main criteria of efficacy will be - the mean strength changes assessed as a composite score (mean of the relative changes of the ten muscle functions tested): ∆ strength as % of baseline values :6 months-baseline.
Time frame: 6 months
1 secondary outcome
Strength changes assessed as a composite score (mean of the relative changes of the ten muscle functions tested) as % of baseline values : 12 months-baseline and 12 months- 6 months, 18 months- 6 months for group B patients
Time frame: 18 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 May 2015.
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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.
Dominique SIMON, PHD principal investigator
Hopital Robert -Debré, Assistance Publique, Hopitaux de Paris
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing GH treatment in people with Chronic Disease and Glucocorticoid Therapy, Response to, run by Association REMEDE at 1 site in France.
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 9 inclusion and 1 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: finished, as of the sponsor's last update on 5 May 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.
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 phase mean?
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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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.
What does the primary outcome measure?
The main criteria of efficacy will be - the mean strength changes assessed as a composite score (mean of the relative changes of the ten muscle functions tested): ∆ strength as % of baseline values :6 months-baseline. (time frame: 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 5 May 2015.