Clinical Trial: Phase 2 Study of EPI-743 for Treatment of Rett Syndrome
Study Status: Completed
Recruit Status: Completed
Study Type: Interventional
Official Title: A Phase 2A Randomized, Placebo Controlled Trial of EPI-743 in Children With Rett Syndrome
Brief Summary: Rett syndrome is a severe neurodevelopmental disorder that primarily affects female children. Rett syndrome is characterized by significant elevation in blood markers of oxidative stress. EPI-743 is a novel therapeutic with demonstrated efficacy and safety in the treatment of disorders characterized by oxidative stress. The purpose of this study is to examine the safety and efficacy of EPI-743 in a population of children with Rett syndrome.
Detailed Summary:
Sponsor: Edison Pharmaceuticals Inc
Current Primary Outcome: Rett Syndrome Clinical Severity Sore [ Time Frame: Change at six months from baseline ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
- Oxidative Stress Biomarkers [ Time Frame: Change at six months from baseline ]
- Head circumference [ Time Frame: Change at six months from baseline ]
- Rett syndrome behavioral questionnaire [ Time Frame: Change at six months from baseline ]
- PedsQL [ Time Frame: Change at six months from baseline ]
- Number of Drug-related adverse and serious adverse events [ Time Frame: Six months ]
- Respiratory Disturbance Index (RDI) [ Time Frame: Change at six months from baseline ]RDI will be determined on polysomnography study
Original Secondary Outcome:
- Oxidative Stress Biomarkers [ Time Frame: Change at six months from baseline ]
- Head circumference [ Time Frame: Change at six months from baseline ]
- Rett syndrome behavioral questionnaire [ Time Frame: Change at six months from baseline ]
- PedsQL [ Time Frame: Change at six months from baseline ]
- Drug-related adverse and serious adverse events [ Time Frame: Six months ]
- Respiratory Disturbance Index (RDI) [ Time Frame: Change at six months from baseline ]RDI will be determined on polysomnography study
Information By: Edison Pharmaceuticals Inc
Dates:
Date Received: March 25, 2013
Date Started: January 2013
Date Completion:
Last Updated: August 3, 2015
Last Verified: August 2015