NCT03642145

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

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

• Status: Withdrawn
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 0 (actual)
• Sponsor: PTC Therapeutics · Industry
• Sex: Male
• Age: 2 Years – 4 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Duchenne Muscular Dystrophy

Interventions

Type	Name	Description
DRUG	Deflazacort	Deflazacort tablets will be administered as per schedule and dose specified in respective arms.

Timeline

• Start date: 2018-10-31
• Primary completion: 2021-07-31
• Completion: 2021-07-31
• First posted: 2018-08-22
• Last updated: 2019-06-21

Regulatory

• FDA-regulated drug study