BENITIONSClinical Trial Intelligence

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

Phase 3 Completed NCT05218655

ConditionInherited Mitochondrial Disease
PhasePhase 3
SponsorPTC Therapeutics
StatusCompleted
Enrollment101 participants
InterventionsVatiquinone
Start dateJun 22, 2022
Completion dateApr 15, 2025
Results postedDec 22, 2025

Summary

The primary objective of this study is to assess the safety of vatiquinone in participants with inherited mitochondrial disease who had prior exposure to vatiquinone in a PTC/BioElectron sponsored (previously Edison) clinical study or treatment plan. The study will continue until vatiquinone becomes commercially available or the program is terminated.

Number of Participants With Treatment-emergent Adverse Events (TEAEs)

Baseline (Day 1) up to 30 days after last dose of study drug (956 days) · Participants

Vatiquinone91 Participants

Open in BENITIONS View on ClinicalTrials.gov

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