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drugset / Trial / NCT03642145

A Study of Deflazacort (Emflaza®) in Participants With Duchenne Muscular Dystrophy (DMD)

NCT03642145 ↗

Phase 3 Withdrawn PTC Therapeutics
RandomizedParallel-groupOpen-labelTreatment

Summary

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.

Timeline

Start
2018-10-31
Primary completion
2021-07-31
Completion
2021-07-31

Outcome

Outcome not reported

Stopped: “Study is no longer necessary given the safety and efficacy of emflaza in this age range has already been established after review of already available data.”

Drugs

EvaluationDrugModalityDoseRoute
Subject Deflazacort Other / unclassified 0.45 mg/kg Oral
Subject Deflazacort Other / unclassified 0.9 mg/kg Oral

Indications

Duchenne muscular dystrophy