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drugset / Trial / NCT02352896

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

NCT02352896 ↗

Phase 2 Completed 30 enrolled PTC Therapeutics
NaSingle-groupOpen-labelTreatment

Summary

EPI-743 in Leigh syndrome participants that participated in previous EPI743-12-002 (NCT01721733) study.

Timeline

Start
2014-01-31
Primary completion
2023-10-30
Completion
2023-10-30

Drugs

EvaluationDrugModalityDoseRoute
Subject Vatiquinone Small molecule 15 mg/kg Oral

Indications

Leigh syndrome