Title
Idebenone as a novel, therapeutic approach for Duchenne muscular dystrophy : results from a 12 month, double-blind, randomized placebo-controlled trialIdebenone as a novel, therapeutic approach for Duchenne muscular dystrophy : results from a 12 month, double-blind, randomized placebo-controlled trial
Author
Faculty/Department
Faculty of Medicine and Health Sciences
Research group
Primary and interdisciplinary care Antwerp (ELIZA)
Publication type
article
Publication
Subject
Human medicine
Source (journal)
Neuromuscular disorders
Volume/pages
21(2011):6, p. 396-405
ISSN
0960-8966
ISI
000291917700003
Carrier
E
Target language
English (eng)
Full text (Publishers DOI)
Affiliation
University of Antwerp
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 816 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.
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