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

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT00033189
• Date of registration: 08/04/2002
• Prospective Registration: No
• Primary sponsor: Cooperative International Neuromuscular Research Group
• Public title: An Open-label Pilot Study of Coenzyme Q10 in Steroid-Treated Duchenne Muscular Dystrophy
• Date of first enrolment: September 2001
• Target sample size: 15
• Recruitment status: Completed
• URL: http://clinicaltrials.gov/show/NCT00033189
• Study type: Interventional
• Study design: Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
• Phase: Phase 2

Countries of recruitment

United States

Key inclusion & exclusion 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.



Age minimum: 5 Years
Age maximum: 11 Years
Gender: Male

Health Condition(s) or Problem(s) studied

Muscular Dystrophy, Duchenne

Intervention(s)

Drug: Coenzyme Q10

Secondary ID(s)

CNMC0301

Source(s) of Monetary Support

Please refer to primary and secondary sponsors