A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy

Summary

This is a parallel-arm, double-blind, placebo-controlled study with a screening phase that includes a 28-day run-in phase to establish baseline seizure frequency, followed by a 24-week, randomized, placebo-controlled phase. After completion of the randomized, placebo-controlled phase, participants may enter a 48-week, long-term, extension phase during which they will receive open-label treatment with vatiquinone.

Timeline

Start

2020-09-28

Primary completion

2023-03-18

Completion

2023-12-27

Outcome

Missed primary endpoint

Stopped (Business): “Sponsor decision.”

registry analysis (other test); Double-blind Period: Vatiquinone vs Double-blind Period: Placebo; p = 0.173; Median Difference (Final Values) -8.31 (95% CI -31.3 to 16.4); ANCOVA NCT04378075 ↗

release “MIT-E trial of vatiquinone for the treatment of mitochondrial disease associated seizures (MDAS) failed to achieve its primary endpoint” ptcbio.com ↗

Drugs

Indications