NCT02104336

Brief Summary

Treatment of Pediatric Subjects with Pearson syndrome

Trial Health

57
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
2

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Aug 2014

Shorter than P25 for phase_2

Geographic Reach
1 country

4 active sites

Status
terminated

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

April 2, 2014

Completed
2 days until next milestone

First Posted

Study publicly available on registry

April 4, 2014

Completed
5 months until next milestone

Study Start

First participant enrolled

August 31, 2014

Completed
1.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

November 30, 2015

Completed
3 months until next milestone

Study Completion

Last participant's last visit for all outcomes

February 29, 2016

Completed
Last Updated

November 18, 2020

Status Verified

November 1, 2020

Enrollment Period

1.2 years

First QC Date

April 2, 2014

Last Update Submit

November 16, 2020

Conditions

Pearson Syndrome

Keywords

PearsonEPI743EPI-743VincerinoneMitochondrial disorderMitochondrial disease

Outcome Measures

Primary Outcomes (1)

  • Occurence of episodes of sepsis, metabolic crisis or hepatic faillure

    1 year

Secondary Outcomes (10)

  • Transfusion avoidance

    1 year

  • Hematologic function

    1 year

  • Neuromuscular function

    1 year

  • Disease severity

    1 year

  • Renal function

    1 year

  • +5 more secondary outcomes

Other Outcomes (1)

  • Number of Dose Limiting SAEs

    1 year

Study Arms (1)

EPI-743

EXPERIMENTAL

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

Drug: EPI-743

Interventions

EPI-743DRUG

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

Also known as: Vincerinone (TM)
EPI-743

Eligibility Criteria

AgeUp to 18 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (4)

Children's Hospital of Orange County

Orange, California, 82868, United States

Location

Cleveland Clinic

Cleveland, Ohio, United States

Location

Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, 15224, United States

Location

Baylor College of Medicine

Houston, Texas, 77030, United States

Location

MeSH Terms

Conditions

VLCAD deficiencyMitochondrial Diseases

Interventions

alpha-tocotrienol quinone

Condition Hierarchy (Ancestors)

Metabolic DiseasesNutritional and Metabolic Diseases

Study Officials

  • Matthew Klein, MD, FACS

    Edison Pharmaceuticals

    STUDY CHAIR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

April 2, 2014

First Posted

April 4, 2014

Study Start

August 31, 2014

Primary Completion

November 30, 2015

Study Completion

February 29, 2016

Last Updated

November 18, 2020

Record last verified: 2020-11

Locations

US(4)