An Open-label Pilot Study of Coenzyme Q10 in Steroid-Treated Duchenne Muscular Dystrophy

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. Identifier: NCT00033189
Recruitment Status : Completed
First Posted : April 9, 2002
Last Update Posted : November 16, 2010
Information provided by:
Cooperative International Neuromuscular Research Group

Brief Summary:
This study will help to determine the safety and efficacy of the nutritional supplement Coenzyme Q10 when added to steroids as a treatment for Duchenne muscular dystrophy (DMD). Boys with DMD who are enrolled in this study will should be on a stable dose of steroids for at least six months, and will remain on their usual dose throughout the study. They will complete two screening visits within a one-week period, and if enrolled will then have their strength tested monthly for three months before beginning therapy with Coenzyme Q10. Once Coenzyme Q10 therapy is started, participants will have their strength tested monthly for six months. Following the six month treatment period, participants will be given the option to remain on Coenzyme Q10 until the study is completed.

Condition or disease Intervention/treatment Phase
Muscular Dystrophy, Duchenne Drug: Coenzyme Q10 Phase 2

Study Type : Interventional  (Clinical Trial)
Enrollment : 15 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Study Start Date : September 2001
Study Completion Date : January 2005

Information from the National Library of Medicine

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Ages Eligible for Study:   5 Years to 11 Years   (Child)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No

Subject Inclusion Criteria

  1. Age: 5 - 11 years old
  2. Ambulant
  3. Diagnosis of DMD confirmed by at least one the following:

    • Positive X-linked family history for typical Duchenne muscular dystrophy in older male relatives (onset by age 5 yr., wheelchair-bound by age 12 yr.) OR
    • Dystrophin immunofluorescence and/or immunoblot showing complete dystrophin deficiency, and clinical picture consistent with typical Duchenne dystrophy OR
    • Gene deletion test positive (missing one or more exons) in the central rod domain (exons 25-60) of dystrophin, where reading frame can be predicted as `out-of-frame', and clinical picture consistent with typical Duchenne dystrophy.
  4. On Glucocorticosteroids: Children must be on a steady dose of prednisone or deflazacort, on any schedule (Daily, alternate days, 10 days on, 10 days off or twice a week), for the last 6 months before starting the clinical trial. Dose of steroid or schedule cannot be altered during the study.
  5. Evidence of muscle weakness by MRC score or clinical functional evaluation
  6. Ability to provide reproducible repeat QMT bicep score within 10% of first assessment score.
  7. Ability to swallow tablets

Subject Exclusion Criteria

  1. Failure to achieve one or more of the diagnostic inclusion criteria cited above.
  2. Symptomatic DMD carrier
  3. Previous (6 months or less) or current use of Coenzyme Q10 (for DMD or any other disease)
  4. Use of carnitine, other amino acids, creatine, glutamine, or any herbal medicines within the last 3 months.
  5. History of significant concomitant illness or significant impairment of renal or hepatic function.

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 identifier (NCT number): NCT00033189

United States, District of Columbia
Children's National Medical Center
Washington, District of Columbia, United States, 20010
United States, Missouri
Washington University-St. Louis
St. Louis, Missouri, United States, 63110
Sponsors and Collaborators
Cooperative International Neuromuscular Research Group Identifier: NCT00033189     History of Changes
Other Study ID Numbers: CNMC0301
First Posted: April 9, 2002    Key Record Dates
Last Update Posted: November 16, 2010
Last Verified: November 2010

Keywords provided by Cooperative International Neuromuscular Research Group:
clinical trial

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
Coenzyme Q10
Growth Substances
Physiological Effects of Drugs