PTC Therapeutics registers Phase 3 PROVE-FA trial for vatiquinone in Friedreich's Ataxia

PTC Therapeutics posted one new registry record this week.

• A Phase 3 study titled "Evaluation of Long-Term Efficacy of Vatiquinone for the Treatment of Friedreich's Ataxia," known as PROVE-FA (NCT07681713), was first posted on 2026-07-02 and is listed as not yet recruiting, with an estimated start date of 2026-08-15 and estimated primary completion of 2029-03-15.¹ The study plans to enroll 120 participants who will receive vatiquinone capsules dosed by body weight, either 200 mg or 400 mg orally three times a day, for 24 months.¹ The primary outcome is change from baseline in the Total Modified Friedreich's Ataxia Rating Scale (mFARS) score at Month 24.¹