Purpose:

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 daily prednisone, CoQ10 and a combination (prednisone and CoQ10) with an enhanced standard of care in wheelchair confined males age 10 to 18 years with an established DMD diagnosis.

Further Study Details:

Duchenne muscular dystrophy (DMD) is the most common form of muscular dystrophy affecting 1:3500 male births worldwide. Despite an increase in our understanding of the disorder since the discovery and characterization of the causative gene and its product dystrophin in 1987, current therapeutic management remains largely supportive. Improvement in the treatment of DMD will depend upon the development of better therapies. Affected boys become symptomatic at 3 to 5 years of age with proximal leg weakness that impairs mobility, ability to get up from a squat, and precludes a normal ability to run. By 8 years of age, some affected boys begin to lose the ability to walk and resort to a wheelchair for mobility. This shift from the ambulant to non-ambulant phase occurs in all boys with a diagnosis of DMD by age 12 years. In this study, participants will be randomized into groups after being screened to determine eligibility. Participants will then be followed for a 12-month investigation period.

Central Contact: Lauren Morgenroth
Phone: 412-383-7207
E-mail: lpm6@pitt.edu

Subject Inclusion Criteria:

• Age 10 to 18 years
• Non-ambulatory (primary mode of transportation is via wheelchair)
• 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 grip muscle score within 15% of the first assessment score
• Has not participated in other therapeutic research protocol for the 6 months prior to screening
• Ability to swallow tablets

Subject Exclusion Criteria:

• Failure to achieve one or more of the inclusion criteria cited above
• Symptomatic DMD carrier
• Spine curvature greater than 50% based on screening x-rays
• 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 chickenpox
• Baseline serum CoQ10 level of 5mg/L or greater