PTC Therapeutics

15 total trials 1 currently recruiting 11 completed

Trial Pipeline

RECRUITING Phase 3

A Study of Sepiapterin in Participants With Phenylketonuria (PKU)

NCT06302348

ACTIVE NOT RECRUITING Phase 3

A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia

NCT05515536

ACTIVE NOT RECRUITING Phase 3

A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria

NCT05166161

ACTIVE NOT RECRUITING Phase 2

A Study of SmartFlow Magnetic Resonance (MR) Compatible Ventricular Cannula for Administering Eladocagene Exuparvovec to Pediatric Participants

NCT04903288

COMPLETED Phase 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)

NCT04336826

COMPLETED Phase 2

A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Who Have Been Treated With Ataluren

NCT03796637

COMPLETED Phase 1

A Study of Unesbulin (PTC596) in Combination With Dacarbazine in Participants With Advanced Leiomyosarcoma (LMS)

NCT03761095

COMPLETED Phase 1

A Study of PTC923 (CNSA-001) in Primary Tetrahydrobiopterin (BH4) Deficient Participants With Hyperphenylalaninemia

NCT03519711

COMPLETED Phase 2

Study of Ataluren in ≥2 to <5 Year-Old Male Participants With Duchenne Muscular Dystrophy

NCT02819557

COMPLETED Phase 1

Food Effect and Bioavailability of Deflazacort Formulations in Healthy Volunteers

NCT02485431

COMPLETED Phase 1

A Single Dose Evaluation of the Effects of Renal Impairment on Deflazacort Pharmacokinetics

NCT02286622

COMPLETED Phase 1

Evaluate Effects of Multiple Doses of Rifampin and Clarithromycin on the Single Dose Pharmacokinetics of Deflazacort

NCT02286635

COMPLETED Phase 3

Study of Ataluren in Nonsense Mutation Cystic Fibrosis (ACT CF)

NCT02139306

COMPLETED Phase 2

Safety and Efficacy Study of EPI-743 in Children With Leigh Syndrome

NCT01721733

COMPLETED Phase 3

Study of Ataluren (PTC124™) in Cystic Fibrosis

NCT00803205

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.

Related

Data sourced from official U.S. government datasets. See our methodology for details. Retrieved and formatted by PlainTrial Editorial