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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02104336
Recruitment Status : Terminated (Results from other studies did not support continuation of this trial)
First Posted : April 4, 2014
Last Update Posted : July 26, 2018
Sponsor:
Information provided by (Responsible Party):
Edison Pharmaceuticals Inc

Tracking Information
First Submitted Date  ICMJE April 2, 2014
First Posted Date  ICMJE April 4, 2014
Last Update Posted Date July 26, 2018
Study Start Date  ICMJE August 2014
Actual Primary Completion Date November 2015   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: October 27, 2014)
Occurrence of episodes of sepsis, metabolic crisis or hepatic failure [ Time Frame: 1 year ]
Original Primary Outcome Measures  ICMJE
 (submitted: April 3, 2014)
Occurence of episodes of sepsis, metabolic crisis or hepatic faillure [ Time Frame: 1 year ]
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: October 27, 2014)
  • 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 Measures  ICMJE
 (submitted: April 3, 2014)
  • 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 ]
Current Other Pre-specified Outcome Measures
 (submitted: April 3, 2014)
Number of Dose Limiting SAEs [ Time Frame: 1 year ]
Any adverse event greater than or equal to grade 3 that is deemed related to treatment with EPI-743 and not related to underlying disease
Original Other Pre-specified Outcome Measures Same as current
 
Descriptive Information
Brief Title  ICMJE Phase 2 Study of EPI-743 in Children With Pearson Syndrome
Official Title  ICMJE 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 Description If effective, treatment with EPI-743 should result in a significant reduction in occurrence of sepsis, metabolic crisis, hepatic failure and transfusion dependence.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Pearson Syndrome
Intervention  ICMJE Drug: EPI-743
EPI-743 is the quinone oxidation product of alpha-tocotrienol
Other Name: Vincerinone (TM)
Study Arms  ICMJE Experimental: EPI-743
15 mg/kg EPI-743 to be administered three times per day for 1 year
Intervention: Drug: EPI-743
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Terminated
Actual Enrollment  ICMJE
 (submitted: July 23, 2018)
2
Original Estimated Enrollment  ICMJE
 (submitted: April 3, 2014)
10
Actual Study Completion Date  ICMJE February 2016
Actual Primary Completion Date November 2015   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Genetically confirmed diagnosis of Pearson syndrome
  • Age less than 18
  • Availability of medical history for 12 months prior to enrollment
  • Abstention from use of CoQ10, vitamin E, lipoic acid and Idebenone 14 days prior to treatment with EPI-743

Exclusion Criteria:

  • Allergy to EPI-743, sesame oil or vitamin E
  • Clinical history of bleeding/ abnormal PT/PTT
  • Concurrent inborn errors of metabolism
  • Use of anticoagulant medications
  • Participation in any interventional study within 30 days of treatment
  • Use of erythropoietin 30 days prior to trial enrollment
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE up to 18 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02104336
Other Study ID Numbers  ICMJE EPI743-13-024
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Edison Pharmaceuticals Inc
Study Sponsor  ICMJE Edison Pharmaceuticals Inc
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Chair: Matthew Klein, MD, FACS Edison Pharmaceuticals
PRS Account Edison Pharmaceuticals Inc
Verification Date July 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP