NCT00654784

Efficacy and Tolerability of Idebenone in Boys With Cardiac Dysfunction Associated With Duchenne Muscular Dystrophy

A Phase IIa Double Blind, Randomised, Placebo Controlled, Single Centre Study at the University of Leuven to Assess the Efficacy and Tolerability of Idebenone in 8 - 16 Year Old Males With Cardiac Dysfunction Associated With Duchenne Muscular Dystrophy

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 21 (actual)
• Sponsor: Santhera Pharmaceuticals · Industry
• Sex: Male
• Age: 8 Years – 16 Years
• Healthy volunteers: Not accepted

Summary

Idebenone is a synthetic analogue of coenzyme Q10 and is a powerful antioxidant and essential constituent of the process of energy production on the cellular level. It can protect mitochondria from oxidative damage and boost their impaired function. It is thought that this mechanism will slow decline in heart function that is part of the disease process of Duchenne Muscular Dystrophy (DMD). It is possible that patients may benefit in terms of muscle strength and respiratory function. This pilot trial is designed to investigate this.

Conditions

• Duchenne Muscular Dystrophy (DMD)

Interventions

Type	Name	Description
DRUG	idebenone	idebenone 450 mg/day (150 mg three times a day)
DRUG	placebo

Timeline

• Start date: 2005-10-01
• Primary completion: 2007-08-01
• Completion: 2007-08-01
• First posted: 2008-04-09
• Last updated: 2011-08-01
• Results posted: 2011-07-29

Locations

1 site across 1 country: Belgium