CoQ10 and Prednisone in Non-Ambulatory DMD

• Status: Terminated
• Trial end date: 2010-11-01
• Target enrollment: 0
• Participant gender: Male

Summary

This study will help determine if CoQ10 and prednisone, alone and as a combination decrease the decline in cardiopulmonary and skeletal muscle function that occurs in the wheelchair confined phase of DMD. Participants who are enrolled in this study should not have taken any corticosteroids within the last six months. This is a 13-month, prospective, randomized study comparing a daily prednisone arm (0.75mg/kg/day), a CoQ10 arm (serum of greater than 2.5 ug/mL) and a combination arm (prednisone and CoQ10) with an enhanced standard of care arm in wheelchair confined males age 10 to 18 years with an established DMD diagnosis.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Cooperative International Neuromuscular Research Group
• Collaborator: United States Department of Defense
• Treatments: Coenzyme Q10; Prednisone; Ubiquinone

Criteria

Inclusion Criteria:

• Age 10-18 years

• Non-ambulatory (primary mode of transportation is via wheelchair for 3 years or less)

• Confirmed DMD diagnosis

• Steroid-naive for the 6 months prior to screening

• Stable dose of b-blocker or ACE inhibitor medication for the 6 months prior to screening, if taking either of these medications

• Ability to provide reproducible repeat QMT grip score within 15% of first assessment score

• Has not participated in other therapeutic research protocol within the last 6 months prior to screening

• Ability to swallow tablets

Exclusion Criteria:

• Failure to achieve one or more of the diagnostic inclusion criteria cited above

• Symptomatic DMD carrier

• Use of carnitine, other amino acids, creatine, glutamine, CoQ10 or any herbal medicines (this would not include herbal teas unless they are consumed daily with intended medicinal effect) within the last 3 months

• History of significant concomitant illness or significant impairment of renal or hepatic function, or other contraindication to steroid therapy

• Positive PPD

• No prior exposure to chickenpox and no immunization against chicken pox

• Baseline serum CoQ10 level of 5.0mg/ml or greater