Duchenne Muscular Dystrophy Clinical Trial
Official title:
A 52-Week Phase 3B Randomized Open-Label Study Evaluating the Safety and Pharmacokinetics of Emflaza® (Deflazacort) Compared to a Comparable Natural History Control Group in Males Aged ≥2 to <5 Years With Duchenne Muscular Dystrophy (DMD) Followed by a 52-Week Extension Period
The primary objective of this study is to evaluate the safety of a 0.9 milligrams per
kilogram (mg/kg) and 0.45 mg/kg daily dose of deflazacort with a comparable natural history
control group after 52 weeks of treatment in males with DMD aged greater than or equal to
(>=) 2 to lesser than (<) 5 years.
The study will comprise of 2 periods (Period 1: 52-week safety and pharmacokinetics [PK], and
Period 2: 52-week extension). Participants will be randomized in a 1:1 ratio to one of 2
treatment arms: 0.9 mg/kg deflazacort, and 0.45 mg/kg of deflazacort. A historic control
group (which should match the study population as closely as possible) will be used as a
comparator to characterize the safety and tolerability of deflazacort.
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