Sponsor Pipeline

PTC Therapeutics Clinical Trial Pipeline

Data updated daily from ClinicalTrials.gov — last refresh 2026-08-16
5
Active Trials
1
Recruiting
4
Phase 3
1
Changes, 30 Days
What Changed in the Last 30 Days
DetectedTrialChange
2026-08-15 NCT07681713
Long-Term Efficacy Study of Vatiquinone for the Treatment of Friedreich's Ataxia (FA)
Trial sites expanded: 0 -> 9 locations
Active Trials by Phase
Phase 3
4
Phase 2
1
Active Trials
NCT IDTitleConditionStatus
Phase 3
NCT06302348 A Study of Sepiapterin in Participants With Phenylketonuria (PKU) Phenylketonuria Recruiting
NCT05515536 A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich… Friedreich Ataxia Active, Not Recruiting
NCT05166161 A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria Phenylketonuria Active, Not Recruiting
NCT07681713 Long-Term Efficacy Study of Vatiquinone for the Treatment of Friedreich's Ataxia (FA) Friedreich's Ataxia Not Yet Recruiting
Phase 2
NCT04903288 A Study of SmartFlow Magnetic Resonance (MR) Compatible Ventricular Cannula for… AADC Deficiency Active, Not Recruiting
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