Efficacy Study of Recombinant Growth Hormone on Muscle Function in Children Long-term Treated With Glucocorticoid

The recruitment status of this study is unknown because the information has not been verified recently.
Verified December 2009 by Association REMEDE.
Recruitment status was  Active, not recruiting
Sponsor:
Collaborator:
Pfizer
Information provided by:
Association REMEDE
ClinicalTrials.gov Identifier:
NCT00813189
First received: December 19, 2008
Last updated: December 1, 2009
Last verified: December 2009
  Purpose

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.


Condition Intervention Phase
Chronic Disease
Long Term Glucocorticoid Treatment
Growth Retardation
Device: recombinant somatotropin
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Effects of Growth Hormone on Corticoid Myopathy in Children With Chronic Disease: Effects on Muscle Mass and Strength

Resource links provided by NLM:


Further study details as provided by Association REMEDE:

Primary Outcome Measures:
  • 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 ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • 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 ] [ Designated as safety issue: No ]

Enrollment: 30
Study Start Date: April 2005
Estimated Study Completion Date: April 2010
Primary Completion Date: September 2009 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: 1
active arm treated with growth hormone
Device: recombinant somatotropin
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.
Other Name: Biosynthetic growth hormone
No Intervention: 2

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)

  Eligibility

Ages Eligible for Study:   6 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • 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
  • 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

Exclusion Criteria:

  • 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.
  Contacts and Locations
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Please refer to this study by its ClinicalTrials.gov identifier: NCT00813189

Locations
France
Hôpital Robert Debré
Paris, France, 75019
Sponsors and Collaborators
Association REMEDE
Pfizer
Investigators
Principal Investigator: Dominique SIMON, PHD Hopital Robert -Debré, Assistance Publique, Hopitaux de Paris
  More Information

No publications provided

Responsible Party: Dr Dominique SIMON, Assistance Publique
ClinicalTrials.gov Identifier: NCT00813189     History of Changes
Other Study ID Numbers: 2004/64
Study First Received: December 19, 2008
Last Updated: December 1, 2009
Health Authority: France: Ministry of Health

Keywords provided by Association REMEDE:
glucocorticoid therapy
muscle function
growth hormone treatment

Additional relevant MeSH terms:
Chronic Disease
Disease Attributes
Pathologic Processes
Glucocorticoids
Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs
Pharmacologic Actions

ClinicalTrials.gov processed this record on September 14, 2014