Efficacy and Tolerability of Idebenone in Boys With Cardiac Dysfunction Associated With Duchenne Muscular Dystrophy
Condition: Duchenne Muscular Dystrophy (DMD)
MeSH terms: Muscular Dystrophies; Muscular Dystrophy, Duchenne
Intervention: idebenone; placebo
Intervention description: idebenone 450 mg/day (150 mg three times a day)
Intervention MeSH terms: Idebenone
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Enrollment: 21.0
Sex: Male
Minimum age: 8 Years
Maximum age: 16 Years
Healthy volunteers: No
Keywords: Duchenne Muscular Dystrophy; DMD; Duchenne

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.
    

Eligibility criteria:

        Inclusion Criteria:

          -  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 Criteria:

          -  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