Organization
PTC Therapeutics
21 clinical trials
Clinical trial
A Phase 1B Study of Unesbulin (PTC596) in Combination With Dacarbazine in Patients With Locally Recurrent, Unresectable or Metastatic Relapsed/Refractory LeiomyosarcomaStatus: Completed, Estimated PCD: 2024-02-05
Clinical trial
A Phase 3, Randomized, Double-blind, Placebo-controlled Efficacy and Safety Study of Ataluren in Patients With Nonsense Mutation Duchenne Muscular Dystrophy and Open-Label ExtensionStatus: Completed, Estimated PCD: 2022-03-05
Clinical trial
A Phase 2/3 Study to Evaluate the Efficacy and Safety of Unesbulin in Unresectable or Metastatic, Relapsed or Refractory LeiomyosarcomaStatus: Active (not recruiting), Estimated PCD: 2024-06-30
Clinical trial
A Phase 3 Study of PTC923 in Subjects With PhenylketonuriaStatus: Completed, Estimated PCD: 2023-04-03
Clinical trial
A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Parallel Study to Assess the Efficacy, Safety, Tolerability, PK, and Biomarker Effects of PTC857 in Adult Subjects With Amyotrophic Lateral Sclerosis (CARDINALS)Status: Active (not recruiting), Estimated PCD: 2024-07-31
Clinical trial
An Open-Label Study Evaluating the Safety and Pharmacokinetics of Ataluren in Children From ≥6 Months to <2 Years of Age With Nonsense Mutation Duchenne Muscular DystrophyStatus: Completed, Estimated PCD: 2023-08-07
Clinical trial
A Phase 3b Open-Label Study of Long-Term Neurocognitive Outcomes in Children With Phenylketonuria Treated With SepiapterinStatus: Not yet recruiting, Estimated PCD: 2031-02-28
Clinical trial
A Randomized, Parallel-Arm, Double-Blind, Placebo-Controlled Study With Open-Label Extension to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Friedreich Ataxia (MOVE-FA)Status: Completed, Estimated PCD: 2023-04-04
Clinical trial
A Phase 2A, Randomized, Placebo-Controlled, Dose-Ranging Study to Evaluate the Safety and Efficacy of PTC518 in Subjects With Huntington's DiseaseStatus: Recruiting, Estimated PCD: 2024-03-31
Clinical trial
An Open-Label Trial to Address the Safety of the SmartFlow MR-Compatible Ventricular Cannula for Administering Eladocagene Exuparvovec to Pediatric SubjectsStatus: Active (not recruiting), Estimated PCD: 2023-05-22
Clinical trial
A Phase 3 Open-Label Study of PTC923 (Sepiapterin) in PhenylketonuriaStatus: Recruiting, Estimated PCD: 2025-02-07
Clinical trial
An Open-Label Study to Evaluate Pharmacokinetics, Safety, and Efficacy of Vatiquinone in Children With Friedreich Ataxia Younger Than 7 Years of AgeStatus: Active (not recruiting), Estimated PCD: 2024-10-05
Clinical trial
Emergency Use Protocol for EPI-743 in Acutely Ill Patients With Inherited Mitochondrial Respiratory Chain Disease Within 90 Days of End-of-Life CareStatus:
Clinical trial
Efficacy and Safety Study of Vatiquinone for the Treatment of Mitochondrial Disease Subjects With Refractory EpilepsyStatus: Completed, Estimated PCD: 2023-03-18
Clinical trial
An Open-Label, Safety Study for Previously Treated Vatiquinone (PTC743) Subjects With Inherited Mitochondrial DiseaseStatus: , Estimated PCD: 2025-03-31
Clinical trial
Long-Term Safety and Efficacy Evaluation of Vatiquinone (EPI-743) in Children With Leigh SyndromeStatus: Completed, Estimated PCD: 2023-10-30
Clinical trial
Evaluation of the Efficacy and Safety of PTC299 in Hospitalized Subjects With COVID-19 (FITE19)Status: Terminated, Estimated PCD: 2022-07-20
Clinical trial
A Phase 1/2, Open-Label, Randomized Parallel Arm, Intra-patient Dose Escalation Study to Evaluate the Safety, Pharmacokinetics and Preliminary Efficacy of CNSA-001(Sepiapterin) in Primary Tetrahydrobiopterin Deficient Patients With HyperphenylalaninemiaStatus: Completed, Estimated PCD: 2020-10-02
Clinical trial
A Phase 2b, Double-Blind, Randomized Extension Study to Evaluate the Long-Term Safety and Efficacy of PTC518 in Participants With Huntington's DiseaseStatus: Recruiting, Estimated PCD: 2027-06-30
Clinical trial
Long-Term Open-Label Study to Assess the Safety and Efficacy of Vatiquinone in Patients With Friedreich AtaxiaStatus: , Estimated PCD: 2027-12-31
Clinical trial
An Open-Label, Safety Study for Ataluren (PTC124) Patients With Nonsense Mutation DystrophinopathyStatus: , Estimated PCD: 2024-04-30