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 ]

Measure of disease progression


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