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

Abstract

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.

• Authors: Gunnar M Buyse, Nathalie Goemans, Marleen van den Hauwe, Daisy Thijs, Imelda J M de Groot, Ulrike Schara, Berten Ceulemans, Thomas Meier, Luc Mertens
• Journal: Neuromuscular disorders : NMD (Neuromuscul Disord) Vol. 21 Issue 6 Pg. 396-405 (Jun 2011) ISSN: 1873-2364 [Electronic] England
• PMID: 21435876 (Publication Type: Clinical Trial, Phase II, Journal Article, Randomized Controlled Trial, Research Support, Non-U.S. Gov't)
• Copyright: Copyright © 2011 Elsevier B.V. All rights reserved.

Chemical References

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

Topics

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