PTC Therapeutics
Trial Pipeline
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 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 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 PTC923 (CNSA-001) in Primary Tetrahydrobiopterin (BH4) Deficient Participants With Hyperphenylalaninemia
NCT03519711
Study of Ataluren in ≥2 to <5 Year-Old Male Participants With Duchenne Muscular Dystrophy
NCT02819557
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
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
Safety and Efficacy Study of EPI-743 in Children With Leigh Syndrome
NCT01721733
Study of Ataluren (PTC124™) in Cystic Fibrosis
NCT00803205
Phase Distribution
| Phase | Trial count |
|---|---|
| Phase 1 | 5 |
| Phase 2 | 5 |
| Phase 3 | 5 |
What the Pipeline for PTC Therapeutics Shows
According to the ClinicalTrials.gov registry, PTC Therapeutics is linked to 15 US clinical trials across every stage of research activity. Of those, 1 studies are currently recruiting — about 7% of the sponsor's indexed portfolio — and 11 are already marked complete, representing roughly 73% of the total. 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.
The phase mix for PTC Therapeutics reports 5 late-stage studies (Phase 3 and Phase 4 combined) and 10 earlier-phase studies (Phase 1 and Phase 2). 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.
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. 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.