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drugset / Trial / NCT04577352

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

NCT04577352 ↗

Phase 2/3 Completed 146 enrolled PTC Therapeutics
RandomizedParallel-groupQuadruple-blindTreatment

Summary

The primary objective of the study is to evaluate the efficacy (using the modified Friedreich Ataxia Rating Scale \[mFARS\]) and safety of vatiquinone in participants with Friedreich ataxia (FA).

Timeline

Start
2020-12-17
Primary completion
2023-04-04
Completion
2023-10-02

Outcome

Missed primary endpoint

registry analysis (other test); Vatiquinone vs Placebo; p = 0.1440; LS Mean Difference -1.610 (95% CI -3.769 to 0.550) NCT04577352 ↗

registry analysis (other test); Vatiquinone vs Placebo; p = 0.0984; LS Mean Difference -1.657 (95% CI -3.622 to 0.308) NCT04577352 ↗

Drugs

EvaluationDrugModalityDoseRoute
Subject Vatiquinone Small molecule 200 mg Oral
Subject Vatiquinone Small molecule 400 mg Oral

Indications

Friedreich ataxia