Clinical Trial: Effect of Oral Glutamine on Muscle Mass and Function in Duchenne Muscular Dystrophy

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: Efficacy Study of Oral Glutamine Supplementation in Duchenne Muscular Dystrophy

Brief Summary: 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).

Detailed Summary:

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).


Sponsor: Assistance Publique - Hôpitaux de Paris

Current Primary Outcome: walking speed at 0,2,4,5,7,9 months [ Time Frame: at 0,2,4,5,7,9 months ]

Original Primary Outcome: walking speed at 0,2,4,5,7,9 months

Current Secondary Outcome:

  • work (kcal) at 0,2,4,5,7,9 months [ Time Frame: at 0,2,4,5,7,9 months ]
  • power (kcal/s) at 0,2,4,5,7,9 months [ Time Frame: at 0,2,4,5,7,9 months ]
  • 2-minute walk test at 0,2,4,5,7,9 months [ Time Frame: at 0,2,4,5,7,9 months ]
  • body composition (bioelectrical impedance analysis) at 0,2,4,5,7,9 months [ Time Frame: at 0,2,4,5,7,9 months ]
  • body composition (BIPHOTONIC absorptiometry) at 4,9 months [ Time Frame: at 4,9 months ]
  • muscle mass (24-h urinary creatinine excretion) at 0,2,4,5,7,9 months [ Time Frame: at 0,2,4,5,7,9 months ]
  • indices of protein degradation (CPK and 3-methyl histidine excretion) at 0,2,4,5,7,9 months [ Time Frame: at 0,2,4,5,7,9 months ]
  • biochemical parameters (electrolytes, fasting glucose, transaminases, insulin, IgfI, Igf-BP3) at 0,2,4,5,7,9 months [ Time Frame: at 0,2,4,5,7,9 months ]


Original Secondary Outcome:

  • work (kcal) at 0,2,4,5,7,9 months
  • power (kcal/s) at 0,2,4,5,7,9 months
  • 2-minute walk test at 0,2,4,5,7,9 months
  • body composition (bioelectrical impedance analysis) at 0,2,4,5,7,9 months
  • body composition (BIPHOTONIC absorptiometry) at 4,9 months
  • muscle mass (24-h urinary creatinine excretion) at 0,2,4,5,7,9 months
  • indices of protein degradation (CPK and 3-methyl histidine excretion) at 0,2,4,5,7,9 months
  • biochemical parameters (electrolytes, fasting glucose, transaminases, insulin, IgfI, Igf-BPI) at 0,2,4,5,7,9 months


Information By: Assistance Publique - Hôpitaux de Paris

Dates:
Date Received: February 23, 2006
Date Started: February 2006
Date Completion:
Last Updated: December 20, 2007
Last Verified: December 2007