NCT04378075Phase 2, phase 3Terminated

A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy

Compare the available registry edits below. Primary completion is the date data collection ends for the main study measures; it does not tell you when results will be published.

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Primary completion2023-03-18Actual

Enrollment · participants68Actual

Source updated2026-03-31Last update listed in the registry record

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Changes to this trial

Last 12 months

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Posted 2026-03-25 · 1 field changed

Main study measure wording changed

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− Before

Measure: Percent Change From Baseline to Week 24 in the Number of Observable Motor Seizures ~~per~~ 28 Days

+ After

Measure: Percent Change From Baseline to Week 24 in the Number of Observable Motor Seizures Per 28 Days During the Double-blind Period

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Study details

Conditions studied

Mitochondrial Diseases; Drug Resistant Epilepsy; Leigh Disease; Leigh Syndrome; Mitochondrial Encephalopathy (MELAS); Pontocerebellar Hypoplasia Type 6 (PCH6); Alpers Disease; Alpers Syndrome

Treatments or interventions studied

VatiquinoneDrugAlso known as PTC743, EPI-743

Primary outcomes · main study measures

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measure: Percent Change From Baseline to Week 24 in the Number of Observable Motor Seizures Per 28 Days During the Double-blind Period; description: The 28-day motor seizure frequency in the double-blind period was calculated as the (number of motor seizures)/ (the number of valid days where motor seizure count information is present) \* 28 within the double-blind period. The baseline of the seizure frequency used the 28 days observations immediately prior to treatment start date for this calculation.; timeFrame: Baseline to Week 24

History coverage

Research sample: selected versions from May 2023 onward plus a preceding baseline where available. Only selected fields were retained; endpoint measures were truncated to 300 characters. This is not complete trial or sponsor history. Daily snapshots begin when collection starts.

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