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

November 16, 2020 updated by: Edison Pharmaceuticals Inc

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

Treatment of Pediatric Subjects with Pearson syndrome

Study Overview

Status

Terminated

Conditions

Intervention / Treatment

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

Interventional

Enrollment (Actual)

2

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • California
      • Orange, California, United States, 82868
        • Children's Hospital of Orange County
    • Ohio
      • Cleveland, Ohio, United States
        • Cleveland Clinic
    • Pennsylvania
      • Pittsburgh, Pennsylvania, United States, 15224
        • Children's Hospital of Pittsburgh
    • Texas
      • Houston, Texas, United States, 77030
        • Baylor College of Medicine

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

No older than 18 years (ADULT, CHILD)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: TREATMENT
  • Allocation: NA
  • Interventional Model: SINGLE_GROUP
  • Masking: NONE

Arms and Interventions

Participant Group / 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 Names:
  • Vincerinone (TM)

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Occurence of episodes of sepsis, metabolic crisis or hepatic faillure
Time Frame: 1 year
1 year

Secondary Outcome Measures

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

Other Outcome Measures

Outcome Measure
Measure Description
Time Frame
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
1 year

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Edison Pharmaceuticals Inc

Investigators

  • Study Chair: Matthew Klein, MD, FACS, Edison Pharmaceuticals

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (ACTUAL)

August 31, 2014

Primary Completion (ACTUAL)

November 30, 2015

Study Completion (ACTUAL)

February 29, 2016

Study Registration Dates

First Submitted

April 2, 2014

First Submitted That Met QC Criteria

April 3, 2014

First Posted (ESTIMATE)

April 4, 2014

Study Record Updates

Last Update Posted (ACTUAL)

November 18, 2020

Last Update Submitted That Met QC Criteria

November 16, 2020

Last Verified

November 1, 2020

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • EPI743-13-024

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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