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EPI-743 in Children With Leigh Syndrome
NCT01721733Phase 2Completed
- Sponsor
- PTC Therapeutics, Inc.
- Start
- Oct 2012
- Primary completion
- Feb 2015
- Modality
- Small Molecules
- Countries
- 1
01
Overview
Phase 2, randomised, double-blind trial of vatiquinone in Leigh syndrome (3 arms), sponsored by PTC Therapeutics, Inc. Primary endpoint: Newcastle Pediatric Mitochondrial Disease Scale Sections 1-3 at 6 months. Started Oct 2012; status: completed.
02
Drugs
vatiquinone
03
Conditions
Leigh syndrome
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