Clinical Trial: Phase 2 Study of EPI-743 in Children With Pearson Syndrome

Study Status: Terminated
Recruit Status: Terminated
Study Type: Interventional

Official Title: An Open-Label Phase 2 Safety and Efficacy Study of EPI-743 (VincerinoneTM) in Children With Pearson Syndrome

Brief Summary: Treatment of Pediatric Subjects with Pearson syndrome

Detailed Summary: If effective, treatment with EPI-743 should result in a significant reduction in occurrence of sepsis, metabolic crisis, hepatic failure and transfusion dependence.
Sponsor: Edison Pharmaceuticals Inc

Current Primary Outcome: Occurrence of episodes of sepsis, metabolic crisis or hepatic failure [ Time Frame: 1 year ]

Original Primary Outcome: Occurence of episodes of sepsis, metabolic crisis or hepatic faillure [ Time Frame: 1 year ]

Current Secondary Outcome:

  • Transfusion avoidance [ Time Frame: 1 year ]
  • Hematologic function [ Time Frame: 1 year ]
  • Neuromuscular function [ Time Frame: 1 year ]
    Neuromuscular function as assessed by Gross Motor Function Measure
  • Disease severity [ Time Frame: 1 year ]
    Disease severity as assessed by Newcastle Pediatric Mitochondrial Disease Scale
  • Renal function [ Time Frame: 1 year ]
  • Hepatic function [ Time Frame: 1 year ]
  • Weight gain [ Time Frame: 1 year ]
  • Hospitalizations [ Time Frame: 1 year ]
  • Pancreatic function [ Time Frame: 1 year ]
    Pancreatic function as assessed by insulin requirement and hemoglobin A1c
  • Mortality [ Time Frame: 1 year ]


Original Secondary Outcome:

  • Transfusion avoidance [ Time Frame: 1 year ]
  • Hematologic function [ Time Frame: 1 year ]
  • Neuromuscular function [ Time Frame: 1 year ]
    Neuromuscular function as assessed by Gross Motor Function Measure
  • Disease severity [ Time Frame: 1 year ]
    Disease severity as assessed by Newcastle Pediatric Mitochondrial Disease Scale
  • Renal function [ Time Frame: 1 year ]
  • Hepatic function [ Time Frame: 1 year ]
  • Weight gain [ Time Frame: 1 year ]
  • Hospitalizations [ Time Frame: 1 year ]
  • Pancreatic function [ Time Frame: 1 year ]
    Pancreatic function as assessed by insulin requirement and hemoglobin A1c
  • Mortalilty [ Time Frame: 1 year ]


Information By: Edison Pharmaceuticals Inc

Dates:
Date Received: April 2, 2014
Date Started: August 2014
Date Completion:
Last Updated: March 18, 2016
Last Verified: November 2015