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MIT-E: Vatiquinone in Mitochondrial Disease With Refractory Epilepsy
jRCT2011210075Completed
- Sponsor
- PTC Therapeutics, Inc.
- Start
- Mar 2022
- Modality
- Small Molecules
01
Overview
Randomised, double-blind trial in Mitochondriopathy, Alpers disease, Leigh syndrome and 3 more comparing Vatiquinone with Placebo → Vatiquinone (2 arms). Primary endpoint: Percent change from baseline in frequency of observable motor seizures per 28 days. Started Mar 2022; status: completed.
02
Drugs
vatiquinone
03
Conditions
MitochondriopathyAlpers diseaseLeigh syndromemitochondrial encephalomyopathiesPontocerebellar Hypoplasia Type 6 (PCH6)
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