Clinical Trial: Safety and Efficacy Study of EPI-743 in Children With Leigh Syndrome
Study Status: Completed
Recruit Status: Completed
Study Type: Interventional
Official Title: A Phase 2B Randomized, Placebo Controlled, Double Blind Clinical Trial of EPI-743 in Children With Leigh Syndrome
Brief 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 Summary:
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.
Sponsor: Edison Pharmaceuticals Inc
Current Primary Outcome: Newcastle Pediatric Mitochondrial Disease Scale (NPMDS) Sections 1-3 [ Time Frame: 6 months ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
- Neuromuscular function [ Time Frame: 6 months ]Gross Motor Function Measure; Barry Albright Dystonia Scale
- Respiratory function [ Time Frame: 6 months ]Need for tracheostomy
- Disease morbidity [ Time Frame: 6 months ]Total number of hospitalizations
- Glutathione cycle biomarkers [ Time Frame: 6 months ]Blood levels of glutathione will be compared between placebo and treatment group
- Number of dose limiting serious adverse events [ Time Frame: 6 months ]
- Mortality [ Time Frame: 6 months ]Number of deaths
Original Secondary Outcome:
- Neuromuscular function [ Time Frame: 6 months ]Gross Motor Function Measure; Barry Albright Dystonia Scale
- Respiratory function [ Time Frame: 6 months ]Need for tracheostomy
- Disease morbidity [ Time Frame: 6 months ]Total number of hospitalizations
- Glutathione cycle biomarkers [ Time Frame: 6 months ]Blood levels of glutathione will be compared between placebo and treatment group
- Number of dose limiting serious adverse events [ Time Frame: 6 months ]
- MortalityNumber of deaths
Information By: Edison Pharmaceuticals Inc
Dates:
Date Received: November 1, 2012
Date Started: October 2012
Date Completion:
Last Updated: August 1, 2016
Last Verified: August 2016