Effect of Oral Glutamine on Muscle Mass and Function in Duchenne Muscular Dystrophy (MDB-GLN)

This study has been completed.
Sponsor:
Information provided by:
Assistance Publique - Hôpitaux de Paris
ClinicalTrials.gov Identifier:
NCT00296621
First received: February 23, 2006
Last updated: December 20, 2007
Last verified: December 2007
  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 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Crossover Assignment
Masking: Double Blind (Subject, Caregiver, Investigator)
Primary Purpose: Treatment
Official Title: Efficacy Study of Oral Glutamine Supplementation in Duchenne Muscular Dystrophy

Resource links provided by NLM:


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
Experimental: 1 Drug: L-Glutamine
L-Glutamine
Other Name: L-Glutamine
Placebo Comparator: 2 Drug: placebo
placebo
Other Name: 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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00296621

Locations
France
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
Pédiatrie Multidisciplinaire et Nutrition de l'Enfant, Centre Hospitalier Universitaire de Poitiers
Poitiers, France, 86000
Sponsors and Collaborators
Assistance Publique - Hôpitaux de Paris
Investigators
Principal Investigator: Régis Hankard, MD, PhD Centre Hospitalier Universitaire (CHU) de Poitiers
  More Information

Additional Information:
Publications:
Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Régis Hankard, MD PhD, CHU de Poitiers
ClinicalTrials.gov Identifier: NCT00296621     History of Changes
Other Study ID Numbers: P030420, AOM 03 121
Study First Received: February 23, 2006
Last Updated: December 20, 2007
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

Additional relevant MeSH terms:
Muscular Dystrophy, Duchenne
Muscular Dystrophies
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn

ClinicalTrials.gov processed this record on July 29, 2014