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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. Please contact Gregory Enns, MD at 650-498-5798 for more information.



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

Phase: Phase 2


Ages Eligible For Study:

N/A - 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

Primary Contact:

Gregory Enns, MD 650-498-5798

Stanford University School of Medicine 300 Pasteur Drive Stanford, CA 94305

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