Sponsor pipeline

PTC Therapeutics clinical trial pipeline

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Data updated daily from ClinicalTrials.gov, last refresh 2026-10-16

What changed in the last 30 days

1 trial changed in the last 30 days: 0 stopped, 1 with a notable change, 0 with minor changes only.

  • Stopped: an ongoing trial was terminated, suspended or withdrawn.
  • Notable: the trial’s plan changed such as a big move in its timeline or size, a new primary endpoint, sponsor or phase, or recruiting opened or resumed.
  • Minor: routine progress and housekeeping, such as sites added or enrollment closed.

Active trials by phase

Phase 2
2
Phase 3
3

Active trials

NCT IDTitleConditionStatusUpdated
Phase 3
NCT06302348 A Study of Sepiapterin in Participants With Phenylketonuria (PKU) Phenylketonuria Recruiting 2026-08-28
NCT07681713 Long-Term Efficacy Study of Vatiquinone for the Treatment of Friedreich's Ataxia (FA) Friedreich's Ataxia Not Yet Recruiting 2026-08-14
NCT05515536 A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich… Friedreich Ataxia Active, Not Recruiting 2025-10-30
Phase 2
NCT07779616 Study of PTC844 in Participants With Active Rheumatoid Arthritis Rheumatoid Arthritis Not Yet Recruiting 2026-08-21
NCT04903288 A Study of SmartFlow Magnetic Resonance (MR) Compatible Ventricular Cannula for… AADC Deficiency Active, Not Recruiting 2026-04-24

Disease areas by active trials

Rheumatoid Arthritis (1)

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