PTC Therapeutics

52 total trials 1 currently recruiting 33 completed

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

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 of Vatiquinone for the Treatment of Participants With Friedreich Ataxia

NCT05485987

COMPLETED Phase 3

A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease

NCT05218655

TERMINATED Phase 2

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

NCT05269355

TERMINATED Phase 2

Study to Assess the Effects of PTC857 Treatment in Participants With Amyotrophic Lateral Sclerosis ALS

NCT05349721

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 3

A Study of PTC923 in Participants With Phenylketonuria

NCT05099640

COMPLETED Phase 2

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

NCT04577352

TERMINATED Phase 2

A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy

NCT04378075

TERMINATED Phase 2

A Study to Evaluate Efficacy and Safety of PTC299 (Emvododstat) in Hospitalized Participants With Coronavirus (COVID-19)

NCT04439071

TERMINATED Phase 3

A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I)

NCT03783923

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 2

A Study of CNSA-001 in Women With Diabetic Gastroparesis

NCT03712124

COMPLETED Phase 1

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

NCT03519711

COMPLETED Phase 2

A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)

NCT03648827

TERMINATED Phase 1

Study of PTC299 (Emvododstat) in Relapsed/Refractory Acute Leukemias

NCT03761069

COMPLETED Phase 3

Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy

NCT03179631

COMPLETED Phase 2

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

NCT02819557

COMPLETED Phase 2

Study of Ataluren in Participants With Nonsense Mutation Aniridia

NCT02647359

COMPLETED Phase 2

Safety and Biomarker Study of EPI-589 in Participants With Amyotrophic Lateral Sclerosis (ALS)

NCT02460679

COMPLETED Phase 1

PTC596 in Patients With Advanced Solid Tumors

NCT02404480

TERMINATED Phase 3

Extension Study of Ataluren in Participants With Nonsense Mutation Cystic Fibrosis

NCT02456103

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

An Open-Label, Long-Term Extension Study to Evaluate the Safety and Tolerability Deflazacort

NCT02295748

COMPLETED Phase 1

A Pharmacokinetic Study of Oral Deflazacort in Children and Adolescent Subjects With Duchenne Muscular Dystrophy

NCT02251600

COMPLETED Phase 1

A Single Dose Evaluation of the Effects of Moderate Hepatic Impairment on Deflazacort Pharmacokinetics

NCT02286609

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

TERMINATED Phase 3

Study of Ataluren (PTC124) in Cystic Fibrosis

NCT02107859

TERMINATED Phase 3

An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy

NCT02090959

COMPLETED Phase 2

Long-Term Safety and Efficacy Evaluation of EPI-743 in Children With Leigh Syndrome

NCT02352896

COMPLETED Phase 3

Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)

NCT01826487

COMPLETED Phase 2

Safety and Efficacy of EPI-743 in Patients With Friedreich's Ataxia

NCT01728064

COMPLETED Phase 2

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

NCT01721733

COMPLETED Phase 3

Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)

NCT01247207

COMPLETED Phase 3

Extension Study of Ataluren (PTC124) in Cystic Fibrosis

NCT01140451

TERMINATED Phase 2

Study of Ataluren (PTC124) in Nonambulatory Participants With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy (nmDMD/BMD)

NCT01009294

TERMINATED Phase 2

Study of Ataluren (PTC124) in Hemophilia A and B

NCT00947193

COMPLETED Phase 3

Study of Ataluren (PTC124™) in Cystic Fibrosis

NCT00803205

TERMINATED Phase 2

PTC299 for Treatment of Neurofibromatosis Type 2

NCT00911248

TERMINATED Phase 2

Phase 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)

NCT00847379

TERMINATED Phase 2

Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD)

NCT00759876

TERMINATED Phase 1

PTC299 for Treatment of Advanced Cancer

NCT00704821

COMPLETED Phase 2

Phase 2B Study of PTC124 (Ataluren) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)

NCT00592553

COMPLETED Phase 1

PTC299 and Hormonal Agent for Treatment of Metastatic Breast Cancer

NCT00508586

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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.

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

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.