Muscular Dystrophy, Duchenne Clinical Trial
Official title:
Efficacy Study of Oral Glutamine Supplementation in Duchenne Muscular Dystrophy
The purpose of this study is to determine whether long-term oral glutamine supplementation is effective in improving muscle mass and function in children with Duchenne muscular dystrophy (DMD).
Glutamine inhibits whole body protein degradation in children with Duchenne Muscular
Dystrophy (DMD). The effect is observed after 5 h oral glutamine administration and is also
found when glutamine is given over a 10-day period. This multi-site national study aims to
evaluate the functional benefit of long-term oral glutamine administration in 30 DMD
children using a randomized double-blind placebo-controlled cross-over design. The study
includes two 4-month periods: 1) a treatment period in which the subject receives oral
glutamine (0.5 g/kg/d) and 2) a control period in which the subject receives a placebo. The
order of treatment allocation is randomized. The two 4-month periods are separated by a 1
month wash-out period. The children are monitored every 2 months during period 1 (M0, M2,
M4) and period 2 (M5, M7, M9) in the clinical investigation centres of Hospital Robert Debré
in Paris and the CHR&U de Lille, as well as the clinical research centre of the CHU de
Poitiers. Evidence of a functional benefit would involve evaluating the administration of
glutamine over longer periods (as early as possible following diagnosis) among severely
handicapped children and in other chronic pathologies associated with increased muscle
protein catabolism. In DMD, such evidence would enable children to undergo gene therapy
under improved physical condition.
Comparisons: Glutamine administration compared to placebo on the following outcome measures:
walking speed on a standard course, work (kcal) and power (kcal/s) in relation to effort,
body composition (bioelectrical impedance analysis and BIPHOTONIC absorptiometry), muscle
mass (24-h urinary creatinine excretion), indices of protein degradation (CPK and 3-methyl
histidine excretion) and biochemical parameters (electrolytes, fasting glucose,
transaminases, insulin, IgfI, Igf-BPI).
;
Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Crossover Assignment, Masking: Double Blind (Subject, Caregiver, Investigator), Primary Purpose: Treatment
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