clinical trial · NCT04378075
A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy
Mitochondrial DiseasesDrug Resistant EpilepsyLeigh DiseaseLeigh SyndromeMitochondrial Encephalopathy (MELAS)Pontocerebellar Hypoplasia Type 6 (PCH6)Alpers DiseaseAlpers SyndromeVatiquinonePlacebo
brief 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.
started
Sep 28, 2020
primary completion
Mar 18, 2023
completion
Dec 27, 2023
last updated
Mar 31, 2026
official title
Efficacy and Safety Study of Vatiquinone for the Treatment of Mitochondrial Disease Subjects With Refractory Epilepsy
sourced from ClinicalTrials.gov · pharmadog mirrors structured fields, not the full protocol