NCT02352896Phase 2Completed

Long-Term Safety and Efficacy Evaluation of EPI-743 in Children With Leigh Syndrome

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 completion2023-10-30Actual

Enrollment · participants30Actual

Source updated2024-10-23Last update listed in the registry record

Version history

Changes to this trial

Last 12 months

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No recorded changes are available for this view and time period. Our history may be incomplete; this does not establish that the trial stayed unchanged.

Study details

Conditions studied

Leigh Syndrome

Treatments or interventions studied

EPI-743DrugAlso known as Vincerinone, Vatiquinone

Primary outcomes · main study measures

measure: Disease Severity as Measured by Newcastle Pediatric Mitochondrial Disease Scale (NPMDS) Sections 1-3 Score; description: NPMDS is a validated scale to assess the mitochondrial disease progression.; timeFrame: Baseline up to Month 36; measure: Number of Participants With Dose-Limiting Serious Adverse Events (SAEs); timeFrame: Baseline up to Month 36

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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