Idebenone as a novel, therapeutic approach for Duchenne muscular dystrophy: results from a 12 month, double-blind, randomized placebo-controlled trial

Neuromuscul Disord. 2011 Jun;21(6):396-405. doi: 10.1016/j.nmd.2011.02.016. Epub 2011 Mar 23.


Early mortality in Duchenne muscular dystrophy (DMD) is related to cardiac and respiratory complications. A phase IIa double-blind randomized placebo-controlled clinical trial was conducted to investigate the tolerability and efficacy of idebenone therapy in children with DMD. Twenty-one DMD patients (aged 8-16 years) were randomly assigned to daily treatment with 450 mg idebenone (Catena®) (n=13) or placebo (n=8) for 12 months. All subjects completed the study and idebenone was safe and well tolerated. Idebenone treatment resulted in a trend (p=0.067) to increase peak systolic radial strain in the left ventricular inferolateral wall, the region of the heart that is earliest and most severely affected in DMD. A significant respiratory treatment effect on peak expiratory flow was observed (p=0.039 for PEF and p=0.042 for PEF percent predicted). Limitations of this study were the small sample size, and a skewed age distribution between treatment groups. Data from this study provided the basis for the planning of a confirmatory study.

Publication types

  • Clinical Trial, Phase II
  • Randomized Controlled Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Antioxidants / adverse effects
  • Antioxidants / pharmacology
  • Antioxidants / therapeutic use*
  • Child
  • Disease Progression*
  • Dose-Response Relationship, Drug
  • Double-Blind Method
  • Forced Expiratory Volume / drug effects
  • Forced Expiratory Volume / physiology
  • Humans
  • Longitudinal Studies
  • Male
  • Muscle Strength / drug effects
  • Muscle Strength / physiology
  • Muscular Dystrophy, Duchenne / drug therapy*
  • Muscular Dystrophy, Duchenne / physiopathology
  • Natriuretic Peptide, Brain / blood
  • Treatment Outcome
  • Troponin I / blood
  • Ubiquinone / adverse effects
  • Ubiquinone / analogs & derivatives*
  • Ubiquinone / pharmacology
  • Ubiquinone / therapeutic use
  • Vital Capacity / drug effects
  • Vital Capacity / physiology


  • Antioxidants
  • Troponin I
  • Natriuretic Peptide, Brain
  • Ubiquinone
  • idebenone