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