NCT01721733

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

A Phase 2B Randomized, Placebo Controlled, Double Blind Clinical Trial of EPI-743 in Children With Leigh Syndrome

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 35 (actual)
• Sponsor: PTC Therapeutics · Industry
• Sex: All
• Age: 6 Years – 17 Years
• Healthy volunteers: Not accepted

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.

Detailed description

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.

Conditions

• Leigh Syndrome

Interventions

Type	Name	Description
DRUG	Placebo
DRUG	EPI-743 15 mg/kg
DRUG	EPI-743 5 mg/kg

Timeline

• Start date: 2012-10-31
• Primary completion: 2015-02-28
• Completion: 2015-05-31
• First posted: 2012-11-06
• Last updated: 2020-08-31

Locations

4 sites across 1 country: United States