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Long-Term Vatiquinone (EPI-743) in Leigh Syndrome
NCT02352896Phase 2Completed
- Sponsor
- PTC Therapeutics, Inc.
- Start
- Jan 2014
- Primary completion
- Oct 2023
- Modality
- Small Molecules
- Countries
- 1
01
Overview
Phase 2, open-label, single-arm trial of vatiquinone in Leigh syndrome, sponsored by PTC Therapeutics, Inc. Primary endpoints: Newcastle Pediatric Mitochondrial Disease Scale Sections 1-3 at 36 months and Serious Adverse Events at 36 months. Started Jan 2014; status: completed.
02
Drugs
vatiquinone
03
Conditions
Leigh syndrome
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