Clinical Trial: Long-Term Safety and Efficacy Evaluation of EPI-743 in Children With Leigh Syndrome

Study Status: Enrolling by invitation
Recruit Status: Enrolling by invitation
Study Type: Interventional

Official Title: EPI743-13-023: Long-Term Safety and Efficacy Evaluation of EPI-743 in Children With Leigh Syndrome

Brief Summary: EPI-743 in Leigh syndrome subjects that participated in previous EPI743-12-002 study.

Detailed Summary: To monitor the long-term safety and neurodevelopmental effects of EPI-743 on children who complete the EPI743-12-002 placebo controlled trial.
Sponsor: Edison Pharmaceuticals Inc

Current Primary Outcome: Long term effect of EPI743 on disease severity as measured by NPMDS [ Time Frame: Up to 3 years ]

Original Primary Outcome: Long term effect of EPI743 on disease severity as measured by NPMDS [ Time Frame: 2 years ]

Current Secondary Outcome:

  • Effect of EPI743 on neurodevelopment as measured by Bayley or Wechsler Scales [ Time Frame: 2 years ]
    neurodevelopment
  • Effect of EPI743 on neuromuscular function as assesed by Barry Albright Dystonia [ Time Frame: 2 years ]
  • Effect of EPI743 on Respiratory Function as measured by oxygen saturation [ Time Frame: Up to 3 years ]
  • Effect of EPI743 on disease morbidity and mortality as measure by mortality, medical encounters and hospitallizations [ Time Frame: Up to 3 years ]
  • Effect of EPI743 on Safety as measure by differences in adverse events [ Time Frame: Up to 3 years ]


Original Secondary Outcome:

  • Effect of EPI743 on neurodevelopment as measured by Bayley or Wechsler Scales [ Time Frame: 2 years ]
    neurodevelopment
  • Effect of EPI743 on neuromuscular function as assesed by Barry Albright Dystonia [ Time Frame: 2 years ]
  • Effect of EPI743 on Respiratory Function as measured by oxygen saturation [ Time Frame: 2 years ]
  • Effect of EPI743 on respiratory function as measured by oxygen saturation, episodes of pneumonia, etc. [ Time Frame: 2 years ]
  • Effect of EPI743 on disease morbidity and mortality as measure by mortality, medical encounters and hospitallizations [ Time Frame: 2 years ]
  • Effect of EPI743 on Safety as measure by differences in adverse events [ Time Frame: 2 years ]


Information By: Edison Pharmaceuticals Inc

Dates:
Date Received: August 21, 2014
Date Started: January 2014
Date Completion: April 2017
Last Updated: September 14, 2015
Last Verified: September 2015