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A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia

NCT05515536 ↗

Phase 3 Active not recruiting 130 enrolled PTC Therapeutics
NaSingle-groupOpen-labelTreatment

Summary

The primary objective of this study is to assess the long-term safety of vatiquinone in participants with Friedreich ataxia (FA) previously exposed to vatiquinone.

Timeline

Start
2022-12-08
Primary completion
2027-12-31
Completion
2027-12-31

Outcome

Missed primary endpoint

release “today reported topline results from the MOVE-FA trial of vatiquinone in patients with Friedreich ataxia. The study did not meet its primary endpoint” ptcbio.com ↗

Drugs

EvaluationDrugModalityDoseRoute
Subject Vatiquinone Small molecule 15 mg/kg Oral
Subject Vatiquinone Small molecule 200 mg Oral
Subject Vatiquinone Small molecule 400 mg Oral

Indications

Friedreich ataxia