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

Phase 2

Terminated

• Conditions: Pearson Syndrome
• Interventions: Drug: EPI-743

• Registration Number: NCT02104336
• Lead Sponsor: Edison Pharmaceuticals Inc

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 Timeline

• Study First Submitted: 2014-04-02
• Study First Submitted QC: 2014-04-03
• Study First Posted: 2014-04-04
• Study Start: 2014-08-31
• Primary Completion: 2015-11-30
• Study Completion: 2016-02-29
• Disposition First Submitted: 2020-08-17
• Disposition First Submitted QC: 2020-08-24
• Disposition First Posted: 2020-08-31
• Status Verified: 2020-11
• Last Update Submitted: 2020-11-16
• Last Update Posted: 2020-11-18

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 2

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

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

Primary Outcome Measures

Name	Time	Method
Occurence of episodes of sepsis, metabolic crisis or hepatic faillure	1 year

Secondary Outcome Measures

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

Trial Locations

Locations (4)

Children's Hospital of Pittsburgh; 🇺🇸 Pittsburgh, Pennsylvania, United States

Children's Hospital of Orange County; 🇺🇸 Orange, California, United States

Baylor College of Medicine; 🇺🇸 Houston, Texas, United States

Cleveland Clinic; 🇺🇸 Cleveland, Ohio, United States