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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.

Stanford is now accepting new patients for this trial.



  • drug : Placebo
  • drug : EPI-743 5 mg/kg
  • drug : EPI-743 15 mg/kg

Phase: Phase 2


Ages Eligible For Study:

6 Years - 17 Years

Inclusion Criteria

- 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

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Contact information

Stanford University
School of Medicine
300 Pasteur Drive
Stanford, CA 94305
Gregory Enns, MD

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