NCT04378075

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

Efficacy and Safety Study of Vatiquinone for the Treatment of Mitochondrial Disease Subjects With Refractory Epilepsy

• Status: Terminated
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 68 (actual)
• Sponsor: PTC Therapeutics · Industry
• Sex: All
• Age: 20 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Mitochondrial Diseases
• Drug Resistant Epilepsy
• Leigh Disease
• Leigh Syndrome
• Mitochondrial Encephalopathy (MELAS)
• Pontocerebellar Hypoplasia Type 6 (PCH6)
• Alpers Disease
• Alpers Syndrome

Interventions

Type	Name	Description
DRUG	Vatiquinone	Vatiquinone will be administered per the treatment arm description.
OTHER	Placebo	Vatiquinone-matching placebo will be administered per the treatment arm description

Timeline

• Start date: 2020-09-28
• Primary completion: 2023-03-18
• Completion: 2023-12-27
• First posted: 2020-05-07
• Last updated: 2026-03-31
• Results posted: 2026-03-25

Locations

27 sites across 9 countries: United States, Canada, France, Italy, Japan, Poland, Spain, Sweden, United Kingdom

Regulatory

• FDA-regulated drug study