A Multicenter Randomized Placebo-Controlled Double-Blind Study to Assess Efficacy and Safety of Glutamine and Creatine Monohydrate in Duchenne Muscular Dystrophy (DMD)

This study has been completed.
Sponsor:
Collaborator:
Children's Research Institute
Information provided by:
National Center for Research Resources (NCRR)
ClinicalTrials.gov Identifier:
NCT00018109
First received: July 3, 2001
Last updated: June 23, 2005
Last verified: December 2003
  Purpose

To establish a collaborative group of clinical trial centers, with standardized equipment and protocols, able to conduct both drug and gene therapy trials in DMD. To evaluate the therapeutic effect of glutamine and creatine monohydrate on muscle strength in children with DMD. To validate the use of QMT (quantitative muscle strength testing) and gait analysis in children with DMD as reliable tools to quantify muscle strength, monitor disease progression and assess therapeutic response.


Condition Intervention Phase
Muscular Dystrophy, Duchenne
Drug: glutamine
Drug: creatine monohydrate
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Masking: Double-Blind
Primary Purpose: Treatment
Official Title: A Multicenter Randomized Placebo-Controlled Double-Blind Study to Assess Efficacy and Safety of Glutamine and Creatine Monohydrate in Duchenne Muscular Dystrophy (DMD)

Resource links provided by NLM:


Further study details as provided by National Center for Research Resources (NCRR):

Detailed Description:

Duchenne muscular dystrophy (DMD) is the most common lethal inherited disorder worldwide. Despite the exponential increase in our understanding of the disorder since the discovery and characterization of the causative gene and its product dystrophin in 1987, current therapeutic management remains largely supportive. Awaiting a final genetic cure to be available in the future, further investments in developing better drug therapies for DMD remain important. Not only because the uniform use of prednisone (the only drug proven to be beneficial) is hampered by potential adverse effects, but also because it may very well be the case that ultimately a combination of both gene and drug therapy will be needed to cure Duchenne children. Here, we test two compounds that have shown promise in a 45-drug screen in the mdx mouse model of Duchenne dystrophy.

The effect of glutamine (0.6/kg/day) and creatine monohydrate (5g/day) on muscle strength will be evaluated in a multi-center randomized double-blind placebo-controlled 3-arm study. Ambulant children aged 5-10 years with an established DMD diagnosis will be studied. Patients will undergo 2 screening evaluations within 2 weeks. Patients will be randomized into treatment groups on the second screening visits, followed by a 6-month treatment period. During the treatment period, patients will be evaluated at monthly intervals. The primary endpoints are percentage change in average muscle strength score and QMT performance for specific muscle groups. Secondary endpoints include timed function tests, functional grades for arms and legs, and pulmonary function tests.

  Eligibility

Ages Eligible for Study:   5 Years to 10 Years
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Ambulant children age 5-10 years with an established diagnosis of Duchenne Muscular Dystrophy

  Contacts and Locations
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Please refer to this study by its ClinicalTrials.gov identifier: NCT00018109

Locations
United States, Missouri
Washington University School of Medicine
St. Louis, Missouri, United States, 63110
Sponsors and Collaborators
Children's Research Institute
  More Information

No publications provided

ClinicalTrials.gov Identifier: NCT00018109     History of Changes
Other Study ID Numbers: NCRR-M01RR00036-5083
Study First Received: July 3, 2001
Last Updated: June 23, 2005
Health Authority: United States: Federal Government

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 August 18, 2014