Phase 2
N=21
Efficacy and Tolerability of Idebenone in Boys With Cardiac Dysfunction Associated With Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy (DMD)
Bottom Line
View on ClinicalTrials.gov: NCT00654784 ↗Enrolled (actual)
21
Serious AEs
9.5%
Results posted
Jul 2011
Primary outcome: Primary: The Relative Change in Peak Systolic Radial Strain of the Left Ventricle (LV) Inferolateral Wall From Baseline (at Screening) to Week 52, Assessed by Color Doppler Myocardial Imaging (CDMI). — 104.4; 28.9 % change in peak systolic
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- idebenone (Drug); placebo (Drug)
- Age
- Pediatric · 8+ yrs
- Sex
- Male
- Sponsor
- Santhera Pharmaceuticals
- Primary completion
- Aug 2007
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY The Relative Change in Peak Systolic Radial Strain of the Left Ventricle (LV) Inferolateral Wall From Baseline (at Screening) to Week 52, Assessed by Color Doppler Myocardial Imaging (CDMI). |
104.4; 28.9 | — |
| SECONDARY Respiratory Function: Forced Vital Capacity (FVC), Forced Expiratory Volume in 1 Second (FEV1), Maximal Inspiratory Pressure (MIP) and Peak Flow (PF) |
— | — |
| SECONDARY Skeletal Muscle Strength (Upper Limb, Right and Left): Hand Grip, Elbow Flexors and Elbow Extensors (Upper Limb Score) Timed Walking Test (10 Metres) (Ambulant Patients Only) |
— | — |
| SECONDARY Safety and Tolerability, Assessed by Adverse Events, Blood and Urine Laboratory Measures, ECG. |
— | — |
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
Data sourced from ClinicalTrials.gov (NCT00654784). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.