Diseases of the Nervous System | Genetics - Adult | Genetics - Pediatric, Phase II-III
A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy (MIT-E)
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What is the purpose of this trial?
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.
- Trial withPTC Therapeutics, Inc.
- Ages18 years and younger
- GenderBoth
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- Last Updated04/21/2023
- Study HIC#2000031217