NCT00308113

CoQ10 and Prednisone in Non-Ambulatory DMD

PITT0503: Clinical Trial of Coenzyme Q10 and Prednisone in Duchenne Muscular Dystrophy

• Status: Terminated
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 3 (actual)
• Sponsor: Cooperative International Neuromuscular Research Group · Network
• Sex: Male
• Age: 10 Years – 18 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Duchenne Muscular Dystrophy

Interventions

Type	Name	Description
DRUG	Prednisone	Prednisone 0/75 mg/kg/day.
DIETARY_SUPPLEMENT	Coenzyme Q10	serum levels of greater or equal to 2.5 micrograms/mL.

Timeline

• Start date: 2007-04-01
• Primary completion: 2010-11-01
• Completion: 2010-11-01
• First posted: 2006-03-29
• Last updated: 2013-11-08
• Results posted: 2013-10-11

Locations

1 site across 1 country: United States