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Phase 3 randomized placebo-controlled trial of Emflaza (deflazacort) in LGMD2I
2018-004740-36Phase 3Terminated
- Sponsor
- PTC Therapeutics, Inc.
- Start
- Apr 2019
- Modality
- Orthosteric Ligands → Classical Binding Small Molecules → Small Molecules
- Countries
- 1
01
Overview
Phase 3, randomised, double-blind trial in limb-girdle muscular dystrophy type 2I (LGMD2I) and muscular dystrophy (MD) comparing Deflazacort (Emflaza) with Placebo (2 arms). Primary endpoint: Change from baseline in time to climb 4 stairs at Week 26. Started Apr 2019; status: terminated.
02
Drugs
deflazacort
03
Conditions
limb-girdle muscular dystrophy type 2I (LGMD2I)muscular dystrophy (MD)
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