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

Primary Purpose

Pearson Syndrome

Status

Terminated

Phase

Phase 2

Locations

United States

Study Type

Interventional

Intervention

EPI-743

About this trial

This is an interventional treatment trial for Pearson Syndrome focused on measuring Pearson, EPI743, EPI-743, Vincerinone, Mitochondrial disorder, Mitochondrial disease

Eligibility Criteria

undefined - 18 Years (Child, Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

Children's Hospital of Orange County
Cleveland Clinic
Children's Hospital of Pittsburgh
Baylor College of Medicine

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

EPI-743

Arm Description

15 mg/kg EPI-743 to be administered three times per day for 1 year

Outcomes

Primary Outcome Measures

Occurence of episodes of sepsis, metabolic crisis or hepatic faillure

Secondary Outcome Measures

Transfusion avoidance

Hematologic function

Neuromuscular function

Neuromuscular function as assessed by Gross Motor Function Measure

Disease severity

Disease severity as assessed by Newcastle Pediatric Mitochondrial Disease Scale

Renal function

Hepatic function

Weight gain

Hospitalizations

Pancreatic function

Pancreatic function as assessed by insulin requirement and hemoglobin A1c

Mortality

Full Information

NCT ID

NCT02104336

First Posted

April 2, 2014

Last Updated

November 16, 2020

Sponsor

Edison Pharmaceuticals Inc

1. Study Identification

Unique Protocol Identification Number

NCT02104336

Brief Title

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

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

November 2020

Overall Recruitment Status

Terminated

Why Stopped

Results from other studies did not support continuation of this trial

Study Start Date

August 31, 2014 (Actual)

Primary Completion Date

November 30, 2015 (Actual)

Study Completion Date

February 29, 2016 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Edison Pharmaceuticals Inc

4. Oversight

Data Monitoring Committee

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Pearson Syndrome

Keywords

Pearson, EPI743, EPI-743, Vincerinone, Mitochondrial disorder, Mitochondrial disease

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

2 (Actual)

8. Arms, Groups, and Interventions

Arm Title

EPI-743

Arm Type

Experimental

Arm Description

15 mg/kg EPI-743 to be administered three times per day for 1 year

Intervention Type

Drug

Intervention Name(s)

EPI-743

Other Intervention Name(s)

Vincerinone (TM)

Intervention Description

EPI-743 is the quinone oxidation product of alpha-tocotrienol

Primary Outcome Measure Information:

Title

Occurence of episodes of sepsis, metabolic crisis or hepatic faillure

Time Frame

1 year

Secondary Outcome Measure Information:

Title

Transfusion avoidance

Time Frame

1 year

Title

Hematologic function

Time Frame

1 year

Title

Neuromuscular function

Description

Neuromuscular function as assessed by Gross Motor Function Measure

Time Frame

1 year

Title

Disease severity

Description

Disease severity as assessed by Newcastle Pediatric Mitochondrial Disease Scale

Time Frame

1 year

Title

Renal function

Time Frame

1 year

Title

Hepatic function

Time Frame

1 year

Title

Weight gain

Time Frame

1 year

Title

Hospitalizations

Time Frame

1 year

Title

Pancreatic function

Description

Pancreatic function as assessed by insulin requirement and hemoglobin A1c

Time Frame

1 year

Title

Mortality

Time Frame

1 year

Other Pre-specified Outcome Measures:

Title

Number of Dose Limiting SAEs

Description

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

Time Frame

1 year

10. Eligibility

Sex

All

Maximum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

Eligibility Criteria

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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Matthew Klein, MD, FACS

Organizational Affiliation

Edison Pharmaceuticals

Official's Role

Study Chair

Facility Information:

Facility Name

Children's Hospital of Orange County

City

Orange

State/Province

California

ZIP/Postal Code

82868

Country

United States

Facility Name

Cleveland Clinic

City

Cleveland

State/Province

Ohio

Country

United States

Facility Name

Children's Hospital of Pittsburgh

City

Pittsburgh

State/Province

Pennsylvania

ZIP/Postal Code

15224

Country

United States

Facility Name

Baylor College of Medicine

City

Houston

State/Province

Texas

ZIP/Postal Code

77030

Country

United States

12. IPD Sharing Statement

Learn more about this trial

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

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