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Priya Life Science Sponsor trial record
Sponsor · clinical trials

PTC Therapeutics clinical trials

Source: ClinicalTrials.gov, U.S. National Library of Medicine — current trials in our USA, France, Australia, Spain, Canada, Brazil, Ireland, UK, Sweden, Poland, Taiwan, Israel, Belgium, Germany and Italy registers with PTC Therapeutics as lead sponsor. Synced 10 Nov 2026
5Current trials
2Recruiting now
1Drug in these trials
15Trial registers
At a glance

PTC Therapeutics is the lead sponsor of 5 current clinical trials in our USA, France, Australia, Spain, Canada, Brazil, Ireland, UK, Sweden, Poland, Taiwan, Israel, Belgium, Germany and Italy registers. 2 are recruiting patients now. The most common phase is Phase 3. The drugs named most often in its trials are Vatiquinone. The most-studied condition is Phenylketonuria.

ClinicalTrials.gov trials by status

  • Recruiting240%
  • Not yet open120%
  • Active, closed240%

Trials by phase

  • Phase 3360%
  • Phase 2240%
01

Clinical trials sponsored by PTC Therapeutics

5 on ClinicalTrials.gov
StudyPhaseStatusStartRegisters
A Study of Sepiapterin in Participants With Phenylketonuria (PKU)
NCT06302348 · Phenylketonuria
Phase 3 Recruiting 2024-03 Ireland, UK, USA, France, Sweden, Poland, Australia
Study of PTC844 in Participants With Active Rheumatoid Arthritis
NCT07779616 · ClinicalTrials.gov ↗ · Rheumatoid Arthritis
Phase 2 Recruiting 2026-09 USA
Long-Term Efficacy Study of Vatiquinone for the Treatment of Friedreich's Ataxia (FA)
NCT07681713 · Friedreich's Ataxia
Phase 3 Not yet open 2026-08 USA, France, Spain, Belgium, Canada, Brazil
A Study of SmartFlow Magnetic Resonance (MR) Compatible Ventricular Cannula for Administering Eladocagene Exuparvovec to Pediatric Participants
NCT04903288 · AADC Deficiency
Phase 2 Active, closed 2021-05 USA, Taiwan, Israel
A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia
NCT05515536 · Friedreich Ataxia
Phase 3 Active, closed 2022-12 USA, Germany, France, Spain, Italy, Canada, Brazil, Australia

Registers list studies that are recruiting, about to open, active but closed to enrolment, or enrolling by invitation. The UK and USA registers cover industry-sponsored studies only. Titles and statuses are copied from the registry as published.

02

Drugs in PTC Therapeutics trials

1 compound

Drugs and biologicals named as interventions in these trials, with the number of this sponsor's trials that name each one. Placebos and generic comparators are excluded.

03

Conditions in PTC Therapeutics trials

5 conditions
Phenylketonuria1AADC Deficiency1Friedreich's Ataxia1Rheumatoid Arthritis1Friedreich Ataxia1
04

By trial register

RegisterTrialsRecruiting
Clinical trials in USA52
Clinical trials in France31
Clinical trials in Australia21
Clinical trials in Spain20
Clinical trials in Canada20
Clinical trials in Brazil20
Clinical trials in Ireland11
Clinical trials in UK11
Clinical trials in Sweden11
Clinical trials in Poland11
Clinical trials in Taiwan10
Clinical trials in Israel10
Clinical trials in Belgium10
Clinical trials in Germany10
Clinical trials in Italy10

A trial with sites in more than one country appears in each of those registers.

05

Other sponsors in Phenylketonuria

SponsorTrials
Otsuka Pharmaceutical Development & Commercialization2

Sponsors with the most current trials registered for this condition.

06

Questions about PTC Therapeutics trials

How many clinical trials does PTC Therapeutics sponsor?

PTC Therapeutics is the lead sponsor of 5 current trials on ClinicalTrials.gov in our USA, France, Australia, Spain, Canada, Brazil, Ireland, UK, Sweden, Poland, Taiwan, Israel, Belgium, Germany and Italy registers, as of the registry sync on 10 November 2026. Completed and withdrawn studies are not counted.

Are PTC Therapeutics trials recruiting?

Yes. 2 of its 5 ClinicalTrials.gov trials are recruiting. Eligibility and site contacts are on each trial's record. Only the study team can confirm whether a patient is eligible.

Which drugs is PTC Therapeutics testing?

Its current trials name 1 drug or biological as interventions. The most frequent are Vatiquinone (2). The number in brackets is how many of its trials name each drug.

Does PTC Therapeutics have clinical trials in Ireland?

Yes. 1 trial in our Ireland register (ClinicalTrials.gov). Some may be the same study registered in both. See the Irish clinical trials register for sites and contacts.

Where does this information come from?

Every trial, status, phase and condition on this page is copied from public ClinicalTrials.gov and EU CTIS records by a daily sync, grouped by the sponsor name each registry records. It says nothing about the organisation beyond its registered trials, and it is not medical advice.

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How this page is built

Everything on this page is copied from public registry records — it is not written from memory, and nothing here describes the organisation beyond its registered trials. Each trial links to its own record so you can check it yourself.

This page is built automatically: a daily sync job reads the registry records behind our trial registers and groups every current trial by its lead sponsor name, ignoring legal-form suffixes such as Inc. or Ltd. A small, hand-checked list joins the same organisation's different registry names (for example its EU legal entity). There is no AI drafting and no per-sponsor editorial pass. The “last verified” date above is the timestamp of that sync, not a review date. Our general editorial approach is described in our editorial standards and corrections policy. The the registries data on this page was last retrieved on 10 Nov 2026. How every register is built: methodology · fixes we have made: corrections log.

Please verify before you rely on this. This page is general information for life-science and pharmaceutical professionals. It is not medical advice, and it has not been reviewed by a clinician — our editorial team holds life-science qualifications, not clinical ones. It is not exhaustive and may not reflect the most recent label change. Always check the official prescribing information (US Prescribing Information or EU SmPC) and speak to your doctor or pharmacist before acting on anything here. Drugs in the same class are not automatically interchangeable, and approvals, brand names and indications differ between the US, the EU/Ireland (EMA/HPRA) and other regions. Spotted an error? Tell us — we correct promptly and log it.