Clinical Trial Update: A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy [Phase 2, Phase 3]

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Summary

A Phase 2/3 study evaluating Vatiquinone for mitochondrial disease in participants with refractory epilepsy has been terminated, and results have been posted.

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Diseases / indications

• Alpers-huttenlocher Syndrome
• Drug-resistant Epilepsy
• Leigh Syndrome
• Mitochondrial Disease
• Mitochondrial Encephalomyopathy Lactic Acidosis and Stroke-like Episodes
• Pontocerebellar Hypoplasia Type

Drugs mentioned

• Vatiquinone

Snippet

Sponsor: PTC Therapeutics | Conditions: Mitochondrial Diseases, Drug Resistant Epilepsy | Phase 2, Phase 3 | Status: TERMINATED | Results posted: 2026-03-25