An Open-Label Pilot Study of Coenzyme Q10 in Steroid-Treated Duchenne Muscular Dystrophy - Tabular View

Tracking Information
First Received Date ^ICMJE	April 8, 2002
Last Updated Date	April 4, 2007
Start Date ^ICMJE	September 2001
Primary Completion Date
Current Primary Outcome Measures ^ICMJE
Original Primary Outcome Measures ^ICMJE
Change History	Complete list of historical versions of study NCT00033189 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures ^ICMJE
Original Secondary Outcome Measures ^ICMJE

Descriptive Information
Brief Title ^ICMJE	An Open-Label Pilot Study of Coenzyme Q10 in Steroid-Treated Duchenne Muscular Dystrophy
Official Title ^ICMJE
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.
Detailed Description
Study Phase	Phase II
Study Type ^ICMJE	Interventional
Study Design ^ICMJE	Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study
Condition ^ICMJE	Muscular Dystrophy, Duchenne
Intervention ^ICMJE	Drug: Coenzyme Q10
Study Arms / Comparison Groups
Publications *
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information
Recruitment Status ^ICMJE	Completed
Enrollment ^ICMJE	15
Completion Date	January 2005
Primary Completion Date
Eligibility Criteria ^ICMJE	Subject Inclusion Criteria Age: 5 - 11 years old Ambulant 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. 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. Evidence of muscle weakness by MRC score or clinical functional evaluation Ability to provide reproducible repeat QMT bicep score within 10% of first assessment score. Ability to swallow tablets Subject Exclusion Criteria Failure to achieve one or more of the diagnostic inclusion criteria cited above. Symptomatic DMD carrier Previous (6 months or less) or current use of Coenzyme Q10 (for DMD or any other disease) Use of carnitine, other amino acids, creatine, glutamine, or any herbal medicines within the last 3 months. History of significant concomitant illness or significant impairment of renal or hepatic function.
Gender	Male
Ages	5 Years to 11 Years
Accepts Healthy Volunteers	No
Contacts ^ICMJE	Contact information is only displayed when the study is recruiting subjects
Location Countries ^ICMJE	United States

Administrative Information
NCT ID ^ICMJE	NCT00033189
Responsible Party
Study ID Numbers ^ICMJE	CNMC0301
Study Sponsor ^ICMJE	Cooperative International Neuromuscular Research Group
Collaborators ^ICMJE
Investigators ^ICMJE
Information Provided By	Cooperative International Neuromuscular Research Group
Verification Date	April 2007
^ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP