A Phase III Double-Blind, Randomised, Placebo-Controlled Study of the Efficacy, Safety and Tolerability of Idebenone in 10 [?] 18 Year Old Patients with Duchenne Muscular Dystrophy
The study will be a double-blind, randomised, placebo-controlled, parallel-group, multicenter study in pts. with DMD - randomised to receive either idebenone or placebo. a minimum of 216 pts. are required to achieve the required statistical power and these will be randomised in a 1:1 ratio to blinded administration of idebenone or placebo.
an 8 week screening period is permissable for patients washing out from systemic glucocorticoid burst therapy for [Quote]non-glucocorticoid users[Quote]. Pts. will undergo regular assessments in the clinic throughout the study period until Week 52 when the study will be completed and medication discontinued.
For patients not continuing into an open-label extension study/compassionate use program, a F/u visit will take place 4 weeks after discontinuation of study medication.
Patients 10 x 18 years of age at Baseline.
2. Signed and dated informed consent.
3. Documented diagnosis of DMD or severe dystrophinopathy and
clinical features consistent of typical DMD at diagnosis (i.e.
documented delayed motor skills and muscle weakness by age 5
years). DMD should be confirmed by mutation analysis in the
dystrophin gene or by substantially reduced levels of dystrophin
protein (i.e. absent or <5% of normal) on Western blot or
4. Ability to provide reliable and reproducible repeat PEF within 15% of
the first assessment (i.e. Baseline vs. Screening).
5. Patients assessed by the investigator as willing and able to comply
with the requirements of the study, possess the required cognitive
abilities and are able to swallow study medication.