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
  • Phase

    Phase 2
  • Study Type

  • Intervention/Treatment

    idebenone ...
  • Study Participants

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.
Study Started
Oct 31
Primary Completion
Aug 31
Study Completion
Aug 31
Results Posted
Jul 29
Last Update
Aug 01

Drug idebenone

idebenone 450 mg/day (150 mg three times a day)

Drug placebo

1 Experimental

2 Placebo Comparator


Inclusion Criteria:

Patients 8 - 16 years of age at time of enrolment
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


Idebenone 450 mg/ Day


All Events

Event Type Organ System Event Term Idebenone 450 mg/ Day Placebo

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).

Assessing the peak systolic radial strain of the left ventricle inferolateral wall is used to characterize the cardiac involvement in the DMD patients. Color Doppler Myocardial Imaging technique is used to quantify regional myocardial function. The cardiac involvement in DMD is characterized by degeneration, atrophy and fibrosis of the myocardium, leading to dilated cardiomyopathy. The process begins in the posterolateral wall of the left ventricle, with septal involvement appearing at later stages.

Idebenone 450 mg/ Day

% change in peak systolic (Mean)
Standard Deviation: 92.0


% change in peak systolic (Mean)
Standard Deviation: 40.7

Respiratory Function: Forced Vital Capacity (FVC), Forced Expiratory Volume in 1 Second (FEV1), Maximal Inspiratory Pressure (MIP) and Peak Flow (PF)

Outcome Measure Data Not Reported

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)

Outcome Measure Data Not Reported

Safety and Tolerability, Assessed by Adverse Events, Blood and Urine Laboratory Measures, ECG.

Outcome Measure Data Not Reported



Age Continuous

years (Mean)
Standard Deviation: 2.0

Age, Categorical

Region of Enrollment

Sex: Female, Male

Overall Study

Idebenone 450 mg/ Day