CoQ10 and Prednisone in Non-Ambulatory DMD

Phase 3

Terminated

Brief 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.

Detailed Description: 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.

Recruitment & Eligibility

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

Arm && Interventions

Group	Intervention	Description
3	Prednisone	CoenzymeQ10 and prednisone each taken once a day in the morning by mouth.
1	Coenzyme Q10	CoenzymeQ10 taken once a day each morning by mouth.
3	Coenzyme Q10	CoenzymeQ10 and prednisone each taken once a day in the morning by mouth.
2	Prednisone	Prednisone taken once a day each morning by mouth

Primary Outcome Measures

Name	Time	Method
One Year Change of Left Ventricular Mean Systolic Wall Stress/Rate-corrected Velocity of Fiber Shortening Relation.	12 months	Comparing change from baseline of mean systolic wall stress and rate-corrected mean velocity of circumferential shortening in the three treatment groups relative to the enhanced standard of care group and relative to each other at one year. The values are obtained via an echocardiogram read locally at each site.
One Year Change in Pulmonary Function (Forced Expiratory Volume, FEV1 and Forced Vital Capacity, FVC)	12 months	Comparing change from baseline levels in pulmonary function (FEV1 and FVC) in the three treatment groups relative to the enhanced standard of care group and relative to each other at one year.

Secondary Outcome Measures

Name	Time	Method
Compare Side Effect Profiles of the Three Study Groups	12 months	To compare side effect profiles of the three regimens to the enhanced standard of care group, to include height, weight, weight/height ratio, body mass index, cataract formation, blood glucose, blood pressure, and behavioral changes.