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Effect of Oral Glutamine on Muscle Mass and Function in Duchenne Muscular Dystrophy (MDB-GLN)
This study has been completed.
Sponsored by: Assistance Publique - Hôpitaux de Paris
Information provided by: Assistance Publique - Hôpitaux de Paris
ClinicalTrials.gov Identifier: NCT00296621
  Purpose

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).


Condition Intervention Phase
Muscular Dystrophy, Duchenne
Drug: L-Glutamine
Drug: placebo
Phase II

Genetics Home Reference related topics: Duchenne and Becker muscular dystrophy L1 syndrome
MedlinePlus related topics: Muscular Dystrophy
Drug Information available for: Glutamine
U.S. FDA Resources
Study Type: Interventional
Study Design: Treatment, Randomized, Double Blind (Subject, Caregiver, Investigator), Placebo Control, Crossover Assignment, Efficacy Study
Official Title: Efficacy Study of Oral Glutamine Supplementation in Duchenne Muscular Dystrophy

Further study details as provided by Assistance Publique - Hôpitaux de Paris:

Primary Outcome Measures:
  • walking speed at 0,2,4,5,7,9 months [ Time Frame: at 0,2,4,5,7,9 months ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • work (kcal) at 0,2,4,5,7,9 months [ Time Frame: at 0,2,4,5,7,9 months ] [ Designated as safety issue: Yes ]
  • power (kcal/s) at 0,2,4,5,7,9 months [ Time Frame: at 0,2,4,5,7,9 months ] [ Designated as safety issue: Yes ]
  • 2-minute walk test at 0,2,4,5,7,9 months [ Time Frame: at 0,2,4,5,7,9 months ] [ Designated as safety issue: Yes ]
  • body composition (bioelectrical impedance analysis) at 0,2,4,5,7,9 months [ Time Frame: at 0,2,4,5,7,9 months ] [ Designated as safety issue: Yes ]
  • body composition (BIPHOTONIC absorptiometry) at 4,9 months [ Time Frame: at 4,9 months ] [ Designated as safety issue: Yes ]
  • 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 ] [ Designated as safety issue: Yes ]
  • 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 ] [ Designated as safety issue: Yes ]
  • 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 ] [ Designated as safety issue: Yes ]

Enrollment: 30
Study Start Date: February 2006
Study Completion Date: November 2007
Estimated Primary Completion Date: February 2008 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
1: Experimental Drug: L-Glutamine
L-Glutamine
2: Placebo Comparator Drug: placebo
placebo

Detailed Description:

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).

  Eligibility

Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Clinical diagnosis of Duchenne muscular dystrophy
  • Able to walk >170 m
  • Absence of hepatic insufficiency
  • Absence of renal insufficiency

Exclusion Criteria:

  • Dependent upon wheelchair
  • Body weight >60kg
  • Liver failure
  • Kidney failure
  • Surgery scheduled during the year following the first visit
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00296621

Locations
France
Pédiatrie Multidisciplinaire et Nutrition de l'Enfant, Centre Hospitalier Universitaire de Poitiers
Poitiers, France, 86000
Service de Neuropédiatrie, Hôpital Roger Salengro, CHR&U de Lille
Lille, France, 59037
Service d'Hépato Gastro Entérologie, Hôpital Jeanne de Flandre, CHR&U de Lille
Lille, France, 59037
Centre d'Investigation Clinique, Hôpital Cardiologique, CHR&U de Lille
Lille, France, 59037
Centre d'Investigation Clinique (CIC9202), Hôpital Robert Debré, Assistance Publique-Hôpitaux de Paris
Paris, France, 75935
Sponsors and Collaborators
Assistance Publique - Hôpitaux de Paris
Investigators
Principal Investigator: Régis Hankard, MD, PhD Centre Hospitalier Universitaire (CHU) de Poitiers
  More Information

Related Info  This link exits the ClinicalTrials.gov site

Publications:
Responsible Party: CHU de Poitiers ( Régis Hankard, MD PhD )
Study ID Numbers: P030420, AOM 03 121
Study First Received: February 23, 2006
Last Updated: December 20, 2007
ClinicalTrials.gov Identifier: NCT00296621  
Health Authority: France: Ministry of Health

Keywords provided by Assistance Publique - Hôpitaux de Paris:
glutamine
nutrition
children
pediatrics
randomized controlled clinical trial
therapy
supplement
oral administration
handicap

Study placed in the following topic categories:
Muscular dystrophy, Duchenne and Becker type
Muscular Dystrophies
Muscular Diseases
Becker's muscular dystrophy
Muscular Disorders, Atrophic
Musculoskeletal Diseases
Neuromuscular Diseases
Genetic Diseases, Inborn
Muscular Dystrophy, Duchenne
Genetic Diseases, X-Linked
Duchenne muscular dystrophy
Atrophy
Muscular dystrophy

Additional relevant MeSH terms:
Nervous System Diseases

ClinicalTrials.gov processed this record on January 15, 2009