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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

15 participants

Study Classification

INTERVENTIONAL

Study Start Date

2001-09-30

Study Completion Date

2005-01-31

Brief Summary

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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.

Detailed Description

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Conditions

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Muscular Dystrophy, Duchenne

Keywords

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duchenne dystrophy DMD clinical trial CINRG

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Interventions

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Coenzyme Q10

Intervention Type DRUG

Eligibility Criteria

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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

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.

Minimum Eligible Age

5 Years

Maximum Eligible Age

11 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No

Locations

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Children's National Medical Center

Washington D.C., District of Columbia, United States

Site Status

Washington University-St. Louis

St Louis, Missouri, United States

Site Status

Countries

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United States

Other Identifiers

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CNMC0301

Identifier Type: -

Identifier Source: org_study_id