Overview

Safety and Efficacy Study of EPI-743 in Children With Leigh Syndrome

Status:
Completed
Trial end date:
2015-05-31
Target enrollment:
Participant gender:
Summary
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.
Phase:
Phase 2
Details
Lead Sponsor:
Edison Pharmaceuticals Inc
PTC Therapeutics
Collaborator:
Axio Research. LLC
Treatments:
Ubiquinone