We're building a better ClinicalTrials.gov. Check it out and tell us what you think!
ClinicalTrials.gov Menu

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 : November 18, 2020
Information provided by (Responsible Party):
Edison Pharmaceuticals Inc

Brief Summary:
Treatment of Pediatric Subjects with Pearson syndrome

Condition or disease Intervention/treatment Phase
Pearson Syndrome Drug: EPI-743 Phase 2

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.

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 2 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-Label Phase 2 Safety and Efficacy Study of EPI-743 (VincerinoneTM) in Children With Pearson Syndrome
Actual Study Start Date : August 31, 2014
Actual Primary Completion Date : November 30, 2015
Actual Study Completion Date : February 29, 2016

Arm Intervention/treatment
Experimental: EPI-743
15 mg/kg EPI-743 to be administered three times per day for 1 year
Drug: EPI-743
EPI-743 is the quinone oxidation product of alpha-tocotrienol
Other Name: Vincerinone (TM)

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

Secondary Outcome Measures :
  1. Transfusion avoidance [ Time Frame: 1 year ]
  2. Hematologic function [ Time Frame: 1 year ]
  3. Neuromuscular function [ Time Frame: 1 year ]
    Neuromuscular function as assessed by Gross Motor Function Measure

  4. Disease severity [ Time Frame: 1 year ]
    Disease severity as assessed by Newcastle Pediatric Mitochondrial Disease Scale

  5. Renal function [ Time Frame: 1 year ]
  6. Hepatic function [ Time Frame: 1 year ]
  7. Weight gain [ Time Frame: 1 year ]
  8. Hospitalizations [ Time Frame: 1 year ]
  9. Pancreatic function [ Time Frame: 1 year ]
    Pancreatic function as assessed by insulin requirement and hemoglobin A1c

  10. Mortality [ Time Frame: 1 year ]

Other Outcome Measures:
  1. 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

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

Layout table for eligibility information
Ages Eligible for Study:   up to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02104336

Layout table for location information
United States, California
Children's Hospital of Orange County
Orange, California, United States, 82868
United States, Ohio
Cleveland Clinic
Cleveland, Ohio, United States
United States, Pennsylvania
Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States, 15224
United States, Texas
Baylor College of Medicine
Houston, Texas, United States, 77030
Sponsors and Collaborators
Edison Pharmaceuticals Inc
Layout table for investigator information
Study Chair: Matthew Klein, MD, FACS Edison Pharmaceuticals
Layout table for additonal information
Responsible Party: Edison Pharmaceuticals Inc
ClinicalTrials.gov Identifier: NCT02104336    
Other Study ID Numbers: EPI743-13-024
First Posted: April 4, 2014    Key Record Dates
Last Update Posted: November 18, 2020
Last Verified: November 2020
Keywords provided by Edison Pharmaceuticals Inc:
Mitochondrial disorder
Mitochondrial disease
Additional relevant MeSH terms:
Layout table for MeSH terms
Muscular Diseases
Lipid Metabolism, Inborn Errors
Mitochondrial Diseases
Pathologic Processes
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lipid Metabolism Disorders
Metabolic Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases