PTC Therapeutics: all 72 studies

Page 1 of 1. Open to participants first (3), then studies with results posted, then the rest; newest registration first within each.

• Study of PTC844 in Participants With Active Rheumatoid ArthritisNot yet recruitingNCT07779616Phase 2
• Long-Term Efficacy Study of Vatiquinone for the Treatment of Friedreich's Ataxia (FA)Not yet recruitingNCT07681713Phase 3
• A Study of Sepiapterin in Participants With Phenylketonuria (PKU)RecruitingNCT06302348Phase 3
• Study to Assess the Effects of PTC857 Treatment in Participants With Amyotrophic Lateral Sclerosis ALSTerminatedNCT05349721Phase 2Results posted
• A Study of Unesbulin in Participants With Advanced Leiomyosarcoma (LMS)TerminatedNCT05269355Phase 2/3Results posted
• A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial DiseaseCompletedNCT05218655Phase 3Results posted
• A Study of PTC923 in Participants With PhenylketonuriaCompletedNCT05099640Phase 3Results posted
• A Study of SmartFlow Magnetic Resonance (MR) Compatible Ventricular Cannula for Administering Eladocagene Exuparvovec to Pediatric ParticipantsActive, not recruitingNCT04903288Phase 2Results posted
• A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich AtaxiaCompletedNCT04577352Phase 2/3Results posted
• A Study to Evaluate Efficacy and Safety of PTC299 (Emvododstat) in Hospitalized Participants With Coronavirus (COVID-19)TerminatedNCT04439071Phase 2/3Results posted
• A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory EpilepsyTerminatedNCT04378075Phase 2/3Results posted
• 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)CompletedNCT04336826Phase 2Results posted
• A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Who Have Been Treated With AtalurenCompletedNCT03796637Phase 2Results posted
• A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I)TerminatedNCT03783923Phase 3Results posted
• A Study of CNSA-001 in Women With Diabetic GastroparesisCompletedNCT03712124Phase 2Results posted
• A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)CompletedNCT03648827Phase 2Results posted
• A Study of PTC923 (CNSA-001) in Primary Tetrahydrobiopterin (BH4) Deficient Participants With HyperphenylalaninemiaCompletedNCT03519711Phase 1/2Results posted
• Long-Term Outcomes of Ataluren in Duchenne Muscular DystrophyCompletedNCT03179631Phase 3Results posted
• Study of Ataluren in ≥2 to <5 Year-Old Male Participants With Duchenne Muscular DystrophyCompletedNCT02819557Phase 2Results posted
• Study of Ataluren in Participants With Nonsense Mutation AniridiaCompletedNCT02647359Phase 2Results posted
• Safety and Biomarker Study of PTC-589 in Participants With Parkinson's DiseaseCompletedNCT02462603Phase 2Results posted
• Safety and Biomarker Study of EPI-589 in Participants With Amyotrophic Lateral Sclerosis (ALS)CompletedNCT02460679Phase 2Results posted
• Extension Study of Ataluren in Participants With Nonsense Mutation Cystic FibrosisTerminatedNCT02456103Phase 3Results posted
• Study of Ataluren in Nonsense Mutation Cystic Fibrosis (ACT CF)CompletedNCT02139306Phase 3Results posted
• Study of Ataluren (PTC124) in Cystic FibrosisTerminatedNCT02107859Phase 3Results posted
• An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation DystrophinopathyTerminatedNCT02090959Phase 3Results posted
• Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)CompletedNCT01826487Phase 3Results posted
• Study of Ataluren for Previously Treated Participants With Nonsense Mutation Duchenne/Becker Muscular Dystrophy (nmDBMD) in Europe, Israel, Australia, and CanadaCompletedNCT01557400Phase 3Results posted
• Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)TerminatedNCT01247207Phase 3Results posted
• Ataluren for Nonsense Mutation Methylmalonic AcidemiaTerminatedNCT01141075Phase 2Results posted
• Extension Study of Ataluren (PTC124) in Cystic FibrosisCompletedNCT01140451Phase 3Results posted
• Study of Ataluren (PTC124) in Nonambulatory Participants With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy (nmDMD/BMD)TerminatedNCT01009294Phase 2Results posted
• Study of Ataluren (PTC124) in Hemophilia A and BTerminatedNCT00947193Phase 2Results posted
• Phase 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)TerminatedNCT00847379Phase 2Results posted
• Study of Ataluren (PTC124™) in Cystic FibrosisCompletedNCT00803205Phase 3Results posted
• Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD)TerminatedNCT00759876Phase 2Results posted
• Phase 2B Study of PTC124 (Ataluren) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)CompletedNCT00592553Phase 2Results posted
• A Study of Ataluren in Pediatric Participants With Cystic FibrosisCompletedNCT00458341Phase 2Results posted
• A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich AtaxiaActive, not recruitingNCT05515536Phase 3
• A Study of Vatiquinone for the Treatment of Participants With Friedreich AtaxiaCompletedNCT05485987Phase 2
• A Long-Term Safety Study of PTC923 in Participants With PhenylketonuriaCompletedNCT05166161Phase 3
• A Phase 2 Open Label Extension Study in Participants With Nonsense Mutation AniridiaWithdrawnNCT04117880Phase 2
• A Study of Unesbulin (PTC596) in Combination With Dacarbazine in Participants With Advanced Leiomyosarcoma (LMS)CompletedNCT03761095Phase 1
• Study of PTC299 (Emvododstat) in Relapsed/Refractory Acute LeukemiasTerminatedNCT03761069Phase 1
• A Study of Deflazacort (Emflaza®) in Participants With Duchenne Muscular Dystrophy (DMD)WithdrawnNCT03642145Phase 3
• Deflazacort Expanded Access Program for Children, Adolescents and Adults With Duchenne Muscular DystrophyApproved for marketingNCT02592941
• Food Effect and Bioavailability of Deflazacort Formulations in Healthy VolunteersCompletedNCT02485431Phase 1
• Effects of Rifampin on the Pharmacokinetics of AtalurenCompletedNCT02409004Phase 1
• PTC596 in Patients With Advanced Solid TumorsCompletedNCT02404480Phase 1
• Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)CompletedNCT02369731
• Analysis of the Glutathione Cycle in Children With Rett SyndromeWithdrawnNCT02360436
• Long-Term Safety and Efficacy Evaluation of EPI-743 in Children With Leigh SyndromeCompletedNCT02352896Phase 2
• Emergency Administration of EPI-743 to a Single Patient With Leber's Hereditary Optic Neuropathy [LHON]No longer availableNCT02300753
• An Open-Label, Long-Term Extension Study to Evaluate the Safety and Tolerability DeflazacortCompletedNCT02295748Phase 1
• Evaluate Effects of Multiple Doses of Rifampin and Clarithromycin on the Single Dose Pharmacokinetics of DeflazacortCompletedNCT02286635Phase 1
• A Single Dose Evaluation of the Effects of Renal Impairment on Deflazacort PharmacokineticsCompletedNCT02286622Phase 1
• A Single Dose Evaluation of the Effects of Moderate Hepatic Impairment on Deflazacort PharmacokineticsCompletedNCT02286609Phase 1
• Protective Effects of EPI-743 on Noise-Induced Hearing LossCompletedNCT02257983Phase 2
• A Pharmacokinetic Study of Oral Deflazacort in Children and Adolescent Subjects With Duchenne Muscular DystrophyCompletedNCT02251600Phase 1
• An Exploratory Open Label Study of EPI-743 (Vincerinone TM) in Children With Autism Spectrum DisorderWithdrawnNCT02226458Phase 2
• Phase 2 Study of EPI-743 in Children With Pearson SyndromeTerminatedNCT02104336Phase 2
• Phase 2 Study of EPI-743 for Treatment of Rett SyndromeCompletedNCT01822249Phase 2
• Safety and Efficacy of EPI-743 in Patients With Friedreich's AtaxiaCompletedNCT01728064Phase 2
• Safety and Efficacy Study of EPI-743 in Children With Leigh SyndromeCompletedNCT01721733Phase 2
• EPI-743 for Mitochondrial Respiratory Chain DiseasesNo longer availableNCT01370447
• PTC299 for Treatment of Neurofibromatosis Type 2TerminatedNCT00911248Phase 2
• PTC299 for Treatment of Advanced CancerTerminatedNCT00704821Phase 1
• PTC299 and Hormonal Agent for Treatment of Metastatic Breast CancerCompletedNCT00508586Phase 1
• PTC124 for the Treatment of Cystic FibrosisCompletedNCT00351078Phase 2
• Safety and Efficacy Study of PTC124 in Duchenne Muscular DystrophyCompletedNCT00264888Phase 2
• Safety and Efficacy of Ataluren (PTC124) for Cystic FibrosisCompletedNCT00237380Phase 2
• PTC124 for Cystic FibrosisCompletedNCT00234663Phase 2

← PTC Therapeutics overview and focus areas