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


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Coenzyme Q-10

Co Q 10; Mitoquinone; Q 10; Ubidecarenone; Ubiquinone; Vitamin Q 




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

This study is no longer recruiting patients.

Sponsored by: Cooperative International Neuromuscular Research Group
Information provided by: Cooperative International Neuromuscular Research Group

Purpose

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 Treatment or Intervention Phase
Muscular Dystrophy, Duchenne
 Drug: Coenzyme Q10
Phase II

MedlinePlus related topics:  Muscular Dystrophy
Genetics Home Reference related topics:  muscular dystrophy, Duchenne and Becker types

Study Type: Interventional
Study Design: Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study

Further Study Details: 

Expected Total Enrollment:  15

Study start: September 2001

Eligibility

Ages Eligible for Study:  5 Years   -   11 Years,  Genders Eligible for Study:  Male

Criteria

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.


Location Information


District of Columbia
      Children's National Medical Center, Washington,  District of Columbia,  20010,  United States

Missouri
      Washington University-St. Louis, St. Louis,  Missouri,  63110,  United States

More Information

The Cooperative International Neuromuscular Research Group

Study ID Numbers:  CNMC0301
Record last reviewed:  January 2005
Last Updated:  January 26, 2005
Record first received:  April 8, 2002
ClinicalTrials.gov Identifier:  NCT00033189
Health Authority: United States: Food and Drug Administration
ClinicalTrials.gov processed this record on 2005-04-08


Source: ClinicalTrials.gov
Cache Date: April 9, 2005

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Page Updated: June 1, 2005
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