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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:
ClinicalTrials.gov Identifier:
NCT00018109
First Posted: July 5, 2001
Last Update Posted: December 9, 2005
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Collaborator:
Children's Research Institute
Information provided by:
National Center for Research Resources (NCRR)
  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
Masking: Double
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

Information from the National Library of Medicine

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Ages Eligible for Study:   5 Years to 10 Years   (Child)
Sexes 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
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00018109


Locations
United States, Missouri
Washington University School of Medicine
St. Louis, Missouri, United States, 63110
Sponsors and Collaborators
National Center for Research Resources (NCRR)
Children's Research Institute
  More Information

ClinicalTrials.gov Identifier: NCT00018109     History of Changes
Other Study ID Numbers: NCRR-M01RR00036-5083
First Submitted: July 3, 2001
First Posted: July 5, 2001
Last Update Posted: December 9, 2005
Last Verified: December 2003

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