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PTC Therapeutics clinical trials
The registry lists 72 studies whose lead sponsor is PTC Therapeutics or one of 1 company grouped under it. 3 are recruiting or about to start. Per ClinicalTrials.gov, last updated Oct 23, 2026.
Keep track of what changes. Save the PTC Therapeutics studies recruiting or about to start, and get an email each week — studies that open, close, add sites or post results.
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Registry sponsor names on this page
Each name exactly as ClinicalTrials.gov lists it, and how it relates to PTC Therapeutics.
- PTC Therapeutics65 studiesSame company
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- Edison Pharmaceuticals Inc7 studies →Acquired — became part of PTC Therapeutics, Oct 25, 2019 · source ↗
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Focus areas
Conditions PTC Therapeutics leads ten or more studies in, by study count. A broader condition is left out when almost all its studies are already in a narrower one listed here.
Studies
Recruiting first, then most recently updated. Showing 50 of 72. Browse all 72 →
- A Study of Sepiapterin in Participants With Phenylketonuria (PKU)RecruitingNCT06302348Phase 3
- 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
- Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)TerminatedNCT01247207Phase 3
- A Long-Term Safety Study of PTC923 in Participants With PhenylketonuriaCompletedNCT05166161Phase 3
- A Study of SmartFlow Magnetic Resonance (MR) Compatible Ventricular Cannula for Administering Eladocagene Exuparvovec to Pediatric ParticipantsActive, not recruitingNCT04903288Phase 2
- A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich AtaxiaCompletedNCT04577352Phase 2/3
- A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory EpilepsyTerminatedNCT04378075Phase 2/3
- Long-Term Outcomes of Ataluren in Duchenne Muscular DystrophyCompletedNCT03179631Phase 3
- Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)CompletedNCT02369731
- A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial DiseaseCompletedNCT05218655Phase 3
- 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
- Study to Assess the Effects of PTC857 Treatment in Participants With Amyotrophic Lateral Sclerosis ALSTerminatedNCT05349721Phase 2
- A Study of Unesbulin in Participants With Advanced Leiomyosarcoma (LMS)TerminatedNCT05269355Phase 2/3
- Long-Term Safety and Efficacy Evaluation of EPI-743 in Children With Leigh SyndromeCompletedNCT02352896Phase 2
- 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 2
- A Study of Unesbulin (PTC596) in Combination With Dacarbazine in Participants With Advanced Leiomyosarcoma (LMS)CompletedNCT03761095Phase 1
- EPI-743 for Mitochondrial Respiratory Chain DiseasesNo longer availableNCT01370447
- A Study of PTC923 in Participants With PhenylketonuriaCompletedNCT05099640Phase 3
- A Study of PTC923 (CNSA-001) in Primary Tetrahydrobiopterin (BH4) Deficient Participants With HyperphenylalaninemiaCompletedNCT03519711Phase 1/2
- A Study to Evaluate Efficacy and Safety of PTC299 (Emvododstat) in Hospitalized Participants With Coronavirus (COVID-19)TerminatedNCT04439071Phase 2/3
- A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I)TerminatedNCT03783923Phase 3
- Study of Ataluren in Participants With Nonsense Mutation AniridiaCompletedNCT02647359Phase 2
- Safety and Biomarker Study of PTC-589 in Participants With Parkinson's DiseaseCompletedNCT02462603Phase 2
Sponsor listed in the registry: Edison Pharmaceuticals Inc · First submitted before Edison Pharmaceuticals Inc became part of PTC Therapeutics
- A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)CompletedNCT03648827Phase 2
- A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Who Have Been Treated With AtalurenCompletedNCT03796637Phase 2
- Analysis of the Glutathione Cycle in Children With Rett SyndromeWithdrawnNCT02360436
Sponsor listed in the registry: Edison Pharmaceuticals Inc · First submitted before Edison Pharmaceuticals Inc became part of PTC Therapeutics
- Study of PTC299 (Emvododstat) in Relapsed/Refractory Acute LeukemiasTerminatedNCT03761069Phase 1
- An Exploratory Open Label Study of EPI-743 (Vincerinone TM) in Children With Autism Spectrum DisorderWithdrawnNCT02226458Phase 2
Sponsor listed in the registry: Edison Pharmaceuticals Inc · First submitted before Edison Pharmaceuticals Inc became part of PTC Therapeutics
- A Study of CNSA-001 in Women With Diabetic GastroparesisCompletedNCT03712124Phase 2
- Study of Ataluren for Previously Treated Participants With Nonsense Mutation Duchenne/Becker Muscular Dystrophy (nmDBMD) in Europe, Israel, Australia, and CanadaCompletedNCT01557400Phase 3
- Phase 2 Study of EPI-743 in Children With Pearson SyndromeTerminatedNCT02104336Phase 2
Sponsor listed in the registry: Edison Pharmaceuticals Inc · First submitted before Edison Pharmaceuticals Inc became part of PTC Therapeutics
- Protective Effects of EPI-743 on Noise-Induced Hearing LossCompletedNCT02257983Phase 2
Sponsor listed in the registry: Edison Pharmaceuticals Inc · First submitted before Edison Pharmaceuticals Inc became part of PTC Therapeutics
- Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD)TerminatedNCT00759876Phase 2
- Extension Study of Ataluren (PTC124) in Cystic FibrosisCompletedNCT01140451Phase 3
- Safety and Biomarker Study of EPI-589 in Participants With Amyotrophic Lateral Sclerosis (ALS)CompletedNCT02460679Phase 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
- Study of Ataluren in ≥2 to <5 Year-Old Male Participants With Duchenne Muscular DystrophyCompletedNCT02819557Phase 2
- An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation DystrophinopathyTerminatedNCT02090959Phase 3
- Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)CompletedNCT01826487Phase 3
- Study of Ataluren (PTC124) in Nonambulatory Participants With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy (nmDMD/BMD)TerminatedNCT01009294Phase 2
- Phase 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)TerminatedNCT00847379Phase 2
- Ataluren for Nonsense Mutation Methylmalonic AcidemiaTerminatedNCT01141075Phase 2
- Study of Ataluren (PTC124) in Hemophilia A and BTerminatedNCT00947193Phase 2
- Safety and Efficacy of Ataluren (PTC124) for Cystic FibrosisCompletedNCT00237380Phase 2
- Study of Ataluren in Nonsense Mutation Cystic Fibrosis (ACT CF)CompletedNCT02139306Phase 3
- Study of Ataluren (PTC124™) in Cystic FibrosisCompletedNCT00803205Phase 3
- Extension Study of Ataluren in Participants With Nonsense Mutation Cystic FibrosisTerminatedNCT02456103Phase 3