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

NCT00813189 | Completed Phase 3

• 1 other identifier: 2004/64
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 1

Brief 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. 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.

Conditions

Chronic Disease; Glucocorticoid Therapy, Response to; Growth Retardation

Keywords

glucocorticoid therapy; muscle function; growth hormone treatment

Outcome Measures

Primary Outcomes (1)

• 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.

  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

  18 months

Study Arms (2)

1 : early start (GH treatment) group

EXPERIMENTAL

in the early start group, patients were treated with growth hormone for one year immediately after randomisation

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

Interventions

GH treatment DEVICE

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.

1 : early start (GH treatment) group

Eligibility Criteria

• Age: 6 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Association REMEDE: lead
• Pfizer: collaborator

Study Sites (1)

Hôpital Robert Debré

Paris, 75019, France

Location

MeSH Terms

Conditions

Chronic Disease

Condition Hierarchy (Ancestors)

Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Dominique SIMON, PHD

  Hopital Robert -Debré, Assistance Publique, Hopitaux de Paris

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: dominique Simon MD

Study Record Dates

• First Submitted: December 19, 2008
• First Posted: December 22, 2008
• Study Start: April 1, 2005
• Primary Completion: June 1, 2010
• Study Completion: May 1, 2012
• Last Updated: May 5, 2015

Record last verified: 2015-05

Locations

FR (1)