PTC Therapeutics
PTC Therapeutics sponsors 52 registered US clinical trials on ClinicalTrials.gov, 1 of them currently recruiting, and 33 completed. 15 of these trials are in Phase 3-4 (later-stage) and 37 in Phase 1-2 (earlier-stage). The most-studied condition in this pipeline is Phenylketonuria, with 2 trials.
Trial Pipeline
52 total, page 1 of 2
A Study of Sepiapterin in Participants With Phenylketonuria (PKU)
NCT06302348
A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia
NCT05515536
A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria
NCT05166161
A Study of SmartFlow Magnetic Resonance (MR) Compatible Ventricular Cannula for Administering Eladocagene Exuparvovec to Pediatric Participants
NCT04903288
A Study of Vatiquinone for the Treatment of Participants With Friedreich Ataxia
NCT05485987
A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease
NCT05218655
A Study of Unesbulin in Participants With Advanced Leiomyosarcoma (LMS)
NCT05269355
Study to Assess the Effects of PTC857 Treatment in Participants With Amyotrophic Lateral Sclerosis ALS
NCT05349721
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)
NCT04336826
A Study of PTC923 in Participants With Phenylketonuria
NCT05099640
A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich Ataxia
NCT04577352
A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy
NCT04378075
A Study to Evaluate Efficacy and Safety of PTC299 (Emvododstat) in Hospitalized Participants With Coronavirus (COVID-19)
NCT04439071
A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I)
NCT03783923
A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Who Have Been Treated With Ataluren
NCT03796637
A Study of Unesbulin (PTC596) in Combination With Dacarbazine in Participants With Advanced Leiomyosarcoma (LMS)
NCT03761095
A Study of CNSA-001 in Women With Diabetic Gastroparesis
NCT03712124
A Study of PTC923 (CNSA-001) in Primary Tetrahydrobiopterin (BH4) Deficient Participants With Hyperphenylalaninemia
NCT03519711
A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
NCT03648827
Study of PTC299 (Emvododstat) in Relapsed/Refractory Acute Leukemias
NCT03761069
Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy
NCT03179631
Study of Ataluren in ≥2 to <5 Year-Old Male Participants With Duchenne Muscular Dystrophy
NCT02819557
Study of Ataluren in Participants With Nonsense Mutation Aniridia
NCT02647359
Safety and Biomarker Study of EPI-589 in Participants With Amyotrophic Lateral Sclerosis (ALS)
NCT02460679
PTC596 in Patients With Advanced Solid Tumors
NCT02404480
Extension Study of Ataluren in Participants With Nonsense Mutation Cystic Fibrosis
NCT02456103
Food Effect and Bioavailability of Deflazacort Formulations in Healthy Volunteers
NCT02485431
A Single Dose Evaluation of the Effects of Renal Impairment on Deflazacort Pharmacokinetics
NCT02286622
An Open-Label, Long-Term Extension Study to Evaluate the Safety and Tolerability Deflazacort
NCT02295748
A Pharmacokinetic Study of Oral Deflazacort in Children and Adolescent Subjects With Duchenne Muscular Dystrophy
NCT02251600
A Single Dose Evaluation of the Effects of Moderate Hepatic Impairment on Deflazacort Pharmacokinetics
NCT02286609
Evaluate Effects of Multiple Doses of Rifampin and Clarithromycin on the Single Dose Pharmacokinetics of Deflazacort
NCT02286635
Study of Ataluren in Nonsense Mutation Cystic Fibrosis (ACT CF)
NCT02139306
Study of Ataluren (PTC124) in Cystic Fibrosis
NCT02107859
An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy
NCT02090959
Long-Term Safety and Efficacy Evaluation of EPI-743 in Children With Leigh Syndrome
NCT02352896
Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
NCT01826487
Safety and Efficacy of EPI-743 in Patients With Friedreich's Ataxia
NCT01728064
Safety and Efficacy Study of EPI-743 in Children With Leigh Syndrome
NCT01721733
Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)
NCT01247207
Extension Study of Ataluren (PTC124) in Cystic Fibrosis
NCT01140451
Study of Ataluren (PTC124) in Nonambulatory Participants With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy (nmDMD/BMD)
NCT01009294
Study of Ataluren (PTC124) in Hemophilia A and B
NCT00947193
Study of Ataluren (PTC124™) in Cystic Fibrosis
NCT00803205
PTC299 for Treatment of Neurofibromatosis Type 2
NCT00911248
Phase 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)
NCT00847379
Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD)
NCT00759876
PTC299 for Treatment of Advanced Cancer
NCT00704821
Phase 2B Study of PTC124 (Ataluren) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)
NCT00592553
PTC299 and Hormonal Agent for Treatment of Metastatic Breast Cancer
NCT00508586
Phase Distribution
| Phase | Trial count |
|---|---|
| Phase 1 | 12 |
| Phase 2 | 25 |
| Phase 3 | 15 |
What the Pipeline for PTC Therapeutics Shows
According to the ClinicalTrials.gov registry, PTC Therapeutics is linked to 52 US clinical trials across every stage of research activity. Of those, 1 studies are currently recruiting, about 2% of the sponsor's indexed portfolio, and 33 are already marked complete, representing roughly 63% of the total.
The phase mix for PTC Therapeutics reports 15 late-stage studies (Phase 3 and Phase 4 combined) and 37 earlier-phase studies (Phase 1 and Phase 2).
The top therapeutic focus area indexed for PTC Therapeutics is Phenylketonuria with 2 linked trials, and 9 other condition areas appear in the top list above.
How to read these pipeline numbers
Recruiting share is one of the more practical signals here: it reflects how much of a sponsor's research is presently open to new participants, while the completed share indicates the depth of finished work that has already contributed registry results. Both counts come directly from the public ClinicalTrials.gov dataset and are refreshed on the registry side; this page mirrors the latest data pull without altering it.
A portfolio weighted toward Phase 3 usually reflects an organization advancing candidates toward regulatory review, where the research centers on comparative efficacy and broader safety across larger populations. A heavier Phase 1 and Phase 2 tilt generally indicates exploratory work, safety, dosing, and early signal detection, and is common among research-forward sponsors that seed many early programs. Phase 4 entries, when present, track interventions already in real-world use and typically focus on long-term safety, effectiveness across subgroups, or formulation comparisons.
That distribution is a quick read of where the organization concentrates its research attention; it does not imply product availability, market share, or any clinical endorsement. All numbers on this page come from ClinicalTrials.gov maintained by the National Library of Medicine, and counts can shift as new studies are registered or existing ones update their status. This information is provided for reference and educational purposes only, not as medical, investment, or regulatory advice, verify current details directly with ClinicalTrials.gov before relying on any figure here.
Read our methodology - how this data is sourced, computed, and verified.
Related
Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page aggregates every registered trial from this sponsor, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.