Status:
COMPLETED
Efficacy and Tolerability of Idebenone in Boys With Cardiac Dysfunction Associated With Duchenne Muscular Dystrophy
Lead Sponsor:
Santhera Pharmaceuticals
Conditions:
Duchenne Muscular Dystrophy (DMD)
Eligibility:
MALE
8-16 years
Phase:
PHASE2
Brief 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 ox...
Eligibility Criteria
Inclusion
- Patients 8 - 16 years of age at time of enrolment
- Male
- Presence of cardiac involvement/dysfunction, defined by abnormal peak systolic strain in left ventricle (LV) inferolateral wall
- Confirmed diagnosis of DMD (out of frame dystrophin gene deletion OR absent/\<5% dystrophin protein on muscle biopsy; clinical picture consistent of typical DMD)
- If on chronic glucocorticosteroids treatment (deflazacort, prednisone) for DMD (or any other disease) (i.e. concomitant medication): dosage must be stable (unchanged) 6 months prior to inclusion
- If on chronic medication for DMD associated cardiomyopathy (β-blocker, diuretics): dosage must be stable (unchanged) 3 months prior to inclusion
- Ability to provide reproducible repeat quantitative muscle testing (QMT) upper limb score within 15% of first assessment score (at Visit1/Day 1 versus Screening Visit
Exclusion
- Symptomatic cardiomyopathy or heart failure
- Asymptomatic but severe cardiac dysfunction on baseline (Screening) evaluation: Fractional shortening (FS) \< 20% and/or Ejection fraction (EF) \< 40%
- Use of ACE-inhibitors
- Previous history of ventricular arrhythmias (other than isolated ventricular extrasystole); ventricular arrhythmias presented at Screening
- Previous (6 months or less) participation in any other therapeutic trial for DMD
- Use of coenzymeQ10, idebenone, creatine, glutamine, oxatomide, or any herbal medicines within the last 6 months
- History of significant concomitant illness or significant impairment of renal or hepatic function
- Known individual hypersensitivity to idebenone
Key Trial Info
Start Date :
October 1 2005
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
August 1 2007
Estimated Enrollment :
21 Patients enrolled
Trial Details
Trial ID
NCT00654784
Start Date
October 1 2005
End Date
August 1 2007
Last Update
August 1 2011
Active Locations (1)
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1
Children's Hospital, University Hospital
Leuven, Belgium