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A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy (MIT-E)

Conditions

Diseases of the Nervous System | Genetics - Adult | Genetics - Pediatric

Phase II-III

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 with
    PTC Therapeutics, Inc.
  • Start Date
    04/28/2022
  • End Date
    03/31/2023

I'm interested in volunteering

If you would prefer to contact a member of the Help us Discover team about this trial and other similar trials, please email helpusdiscover@yale.edu or call 877.978.8343

  • Last Updated
    08/16/2022
  • Study HIC
    #2000031217