title: 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: Child Neurology, University Hospitals Leuven, Leuven, Belgium. gunnar.buyse@uzleuven.be
authors: Gunnar M
authors: GM
authors: Buyse
authors: Nathalie
authors: N
authors: Goemans
authors: Marleen
authors: M
authors: van den Hauwe
authors: Daisy
authors: D
authors: Thijs
authors: Imelda J M
authors: IJ
authors: de Groot
authors: Ulrike
authors: U
authors: Schara
authors: Berten
authors: B
authors: Ceulemans
authors: Thomas
authors: T
authors: Meier
authors: Luc
authors: L
authors: Mertens
mesh_terms: D000293:Adolescent; D000975:Antioxidants / Q000009:adverse effects / Q000494:pharmacology / Q000627:therapeutic use*; D002648:Child; D018450:Disease Progression; D004305:Dose-Response Relationship, Drug; D004311:Double-Blind Method; D005541:Forced Expiratory Volume / Q000187:drug effects / Q000502:physiology; D006801:Humans; D008137:Longitudinal Studies; D008297:Male; D053580:Muscle Strength / Q000187:drug effects / Q000502:physiology; D020388:Muscular Dystrophy, Duchenne / Q000188:drug therapy* / Q000503:physiopathology; D020097:Natriuretic Peptide, Brain / Q000097:blood; D016896:Treatment Outcome; D019210:Troponin I / Q000097:blood; D014451:Ubiquinone / Q000009:adverse effects / Q000031:analogs & derivatives* / Q000494:pharmacology / Q000627:therapeutic use; D014797:Vital Capacity / Q000187:drug effects / Q000502:physiology
chemical_list: D000975:Antioxidants; D019210:Troponin I; D020097:Natriuretic Peptide, Brain; D014451:Ubiquinone; C036619:idebenone