Leigh Syndrome Clinical Trial
Official title:
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.
The purpose of this study is to evaluate the effects of EPI-743 in patient with Leigh
syndrome on disease severity, neuromuscular function, respiratory function, disease morbidity
and mortality and biomarkers associated with the disease.
This study is a six month prospective randomized double-blind, placebo-controlled trial with
a six month extension phase of two dose levels of EPI743. The planned enrollment is for
approximately 30 children with genetically confirmed Leigh syndrome. After 6 months of
treatment, those children that were randomized to the placebo treatment arm will be
re-randomized to one of the 2 active treatment arms.
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