drugset / Trial / NCT01721733
Safety and Efficacy Study of EPI-743 in Children With Leigh Syndrome
RandomizedCrossoverQuadruple-blindTreatment
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.
Timeline
- Start
- 2012-10-31
- Primary completion
- 2015-02-28
- Completion
- 2015-05-31
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Comparator | Vatiquinone | Small molecule | 5 mg/kg | — |
| Comparator | Vatiquinone | Small molecule | 15 mg/kg | — |