Vatiquinone Expanded Access Protocol
- Status
- NO_LONGER_AVAILABLE
- Phase
- N/A
Clinical Trials Related to PTC Therapeutics
Related studies linked through entity extraction aliases.
A Study of Sepiapterin in Participants With Phenylketonuria (PKU)
- Status
- RECRUITING
- Phase
- PHASE3
A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia
- Status
- ACTIVE_NOT_RECRUITING
- Phase
- PHASE3
A Study of Vatiquinone for the Treatment of Participants With Friedreich Ataxia
- Status
- COMPLETED
- Phase
- PHASE2
A Study to Evaluate the Safety and Efficacy of PTC518 in Participants With Huntington's Disease (HD)
- Status
- COMPLETED
- Phase
- PHASE2
Study to Assess the Effects of PTC857 Treatment in Participants With Amyotrophic Lateral Sclerosis ALS
- Status
- TERMINATED
- Phase
- PHASE2
A Study of Unesbulin in Participants With Advanced Leiomyosarcoma (LMS)
- Status
- TERMINATED
- Phase
- PHASE2/PHASE3
A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease
- Status
- COMPLETED
- Phase
- PHASE3
A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria
- Status
- ACTIVE_NOT_RECRUITING
- Phase
- PHASE3
A Study of PTC923 in Participants With Phenylketonuria
- Status
- COMPLETED
- Phase
- PHASE3
A Study of SmartFlow Magnetic Resonance (MR) Compatible Ventricular Cannula for Administering Eladocagene Exuparvovec to Pediatric Participants
- Status
- ACTIVE_NOT_RECRUITING
- Phase
- PHASE2
A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich Ataxia
- Status
- COMPLETED
- Phase
- PHASE2/PHASE3
A Study to Evaluate Efficacy and Safety of PTC299 (Emvododstat) in Hospitalized Participants With Coronavirus (COVID-19)
- Status
- TERMINATED
- Phase
- PHASE2/PHASE3
A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy
- Status
- TERMINATED
- Phase
- PHASE2/PHASE3
A Study to Evaluate the Safety and Pharmacokinetics of Ataluren in Participants From ≥6 Months to <2 Years of Age With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
- Status
- COMPLETED
- Phase
- PHASE2
A Phase 2 Open Label Extension Study in Participants With Nonsense Mutation Aniridia
- Status
- WITHDRAWN
- Phase
- PHASE2
Pembrolizumab With Ataluren in Patients With Metastatic pMMR and dMMR Colorectal Carcinoma or Metastatic dMMR Endometrial Carcinoma: the ATAPEMBRO Study
- Status
- UNKNOWN
- Phase
- PHASE1/PHASE2
A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Who Have Been Treated With Ataluren
- Status
- COMPLETED
- Phase
- PHASE2
A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I)
- Status
- TERMINATED
- Phase
- PHASE3
A Study of Unesbulin (PTC596) in Combination With Dacarbazine in Participants With Advanced Leiomyosarcoma (LMS)
- Status
- COMPLETED
- Phase
- PHASE1