Vatiquinone
Sponsors
PTC Therapeutics Inc., PTC Therapeutics, Medical University of South Carolina
Conditions
Alpers DiseaseAlpers SyndromeDrug Resistant EpilepsyFriedreich AtaxiaFriedreich ataxiaFriedreich's AtaxiaInherited Mitochondrial DiseaseLeigh Disease
Phase 2
A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy
TerminatedNCT04378075
Start: 2020-09-28End: 2023-12-27Updated: 2026-03-31
A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich Ataxia
CompletedNCT04577352
Start: 2020-12-17End: 2023-10-02Updated: 2026-04-13
A Study of Vatiquinone for the Treatment of Participants With Friedreich Ataxia
CompletedNCT05485987
Start: 2022-10-12End: 2024-08-29Updated: 2025-08-22
Phase 3
A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease
CompletedNCT05218655
Start: 2022-06-22End: 2025-04-15Updated: 2025-12-22
A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia
Active, not recruitingNCT05515536
Start: 2022-12-08End: 2027-12-31Updated: 2025-10-30
Long-Term Open-Label Study to Assess the Safety and Efficacy of Vatiquinone in Patients With Friedreich Ataxia
RecruitingCTIS2024-516505-23-00
Start: 2023-03-22Target: 34Updated: 2025-07-21
Long-Term Efficacy Study of Vatiquinone for the Treatment of Friedreich's Ataxia (FA)
Not yet recruitingNCT07681713
Start: 2026-08-15End: 2029-03-15Target: 120Updated: 2026-08-14