A Phase 2B Randomized, Placebo Controlled, Double Blind Clinical Trial of EPI-743 in Children With Leigh Syndrome
The purpose of this study is to evaluate the effects of EPI-743 in children with Leigh syndrome on disease severity, neuromuscular function, respiratory function, disease morbidity and mortality and disease associated biomarkers.
- drug : Placebo
- drug : EPI-743 5 mg/kg
- drug : EPI-743 15 mg/kg
Phase: Phase 2
Ages Eligible For Study:
- Clinical and MRI diagnosis of Leigh syndrome - Moderate disease severity based on NPMDS score - Age under 18 years - Documented evidence of disease progression within 12 month of enrollment - Availability of MRI that confirms necrotizing encephalopathy - Patient or guardian able to consent and comply with protocol requirements - Abstention from Coenzyme Q10, Vitamins C & E, lipoic acid and Idebenone