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Phase 3 randomized double-blind placebo-controlled study of ataluren in nonsense mutation Duchenne muscular dystrophy with open-label extension
jRCT2080224563Phase 3Completed
- Sponsor
- PTC Therapeutics (Medpace Japan K.K.. ; In-Country Caretaker for Clinical trials).
- Start
- Feb 2019
- Modality
- Small Molecules
01
Overview
Phase 3, randomised, double-blind trial in Duchenne muscular dystrophy (DMD) comparing Ataluren arm with Placebo arm (2 arms). Primary endpoint: Efficacy (preservation of ambulation and physical function). Started Feb 2019; status: completed.
02
Drugs
ataluren
03
Conditions
Duchenne muscular dystrophy (DMD)
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