PTC Therapeutics
Open enrollment with an early-phase pipeline tilt
According to ClinicalTrials.gov-derived indexes, PTC Therapeutics sits in the top decile of US sponsors by registered trial count (#363 of 15,742 sponsors by registered trial count).
PTC Therapeutics: 52 sponsored US clinical trials, 1 recruiting.
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.
Top-decile sponsor footprint
PTC Therapeutics ranks in the top decile of sponsors by registered trial count (#363 of 15,742 sponsors by registered trial count). Volume at this scale usually reflects a long institutional pipeline, read phase tilt and therapeutic areas below before treating it as current-year activity.
Trial completion reliability score
33 of 48 decided trials (Completed vs. Terminated on ClinicalTrials.gov) reached their planned completion (68.8%), vs. national p10 68.4% / p90 100% among 1,234 sponsors with at least 10 decided trials. Still-open trials (Recruiting, Active-not-recruiting, Not-yet-recruiting) are excluded since they have not reached an outcome yet.
Open and recent trials
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 is open in the PTC Therapeutics pipeline
1 recruiting of 52 indexed (2% open); 33 completed (63%).
Phase mix: 15 late (III/IV) vs 37 early (I/II) (early-phase heavy).
Top condition: Phenylketonuria (2 trials), 9 others listed.
How open-pipeline share is computed
Recruiting share is recruiting_count divided by trial_count for this sponsor in the public registry export. Status can change between pulls; treat the figure as a research-attention snapshot, not product availability.
PTC Therapeutics: open-pipeline peers nationwide
Peers matched on 52 registered trials and 2% open enrollment, not the therapeutic-area sidebar.
PTC Therapeutics sits at or above the median trial volume and at or above the median recruiting share among these 9 nationwide peers -- the two do not always move together.
Sponsors near 52 trials (± recruiting share)
- Albany Medical College · 52 trials · 13% recruiting
- Burzynski Research Institute · 52 trials · 0% recruiting
- Dr. Reddy's Laboratories Limited · 52 trials · 0% recruiting
- Spectrum Pharmaceuticals · 52 trials · 2% recruiting
Sponsors near 2% open enrollment (± trial volume)
- Société des Produits Nestlé (SPN) · 2% recruiting · 53 trials
- Sanofi Pasteur, a Sanofi Company · 2% recruiting · 153 trials
- Carelon Research · 2% recruiting · 50 trials
- Alcon Research · 2% recruiting · 275 trials
Related
What to do with this PTC Therapeutics page
52 registered trials is a research-attention snapshot, not an endorsement of PTC Therapeutics or any single study.
- 1 of PTC Therapeutics's trials are currently recruiting, check each one's own eligibility criteria before contacting a site. Browse recruiting trials
- Phenylketonuria is the condition PTC Therapeutics studies most, compare against other sponsors active in the same condition before evaluating a specific study. See Phenylketonuria trials
- Bring the specific trial ID (NCT number) to your own doctor before enrolling, this page is a directory, not medical guidance. Read the trial-finding guide
Trial and recruiting counts reflect ClinicalTrials.gov registry status as of the data vintage above; individual trial eligibility and enrollment status can change.
Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. PTC Therapeutics pipeline card: 52 registered trials from official public datasets. See our editorial standards & corrections policy, the methodology behind these numbers, the data changelog, or report a data error.