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

Pearson Syndrome Clinical Trial

Official title:

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

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT02104336
• Other study ID #: EPI743-13-024
• Status: Terminated
• Phase: Phase 2
• Start date: August 31, 2014
• Est. completion date: February 29, 2016

Study information

• Verified date: November 2020
• Source: Edison Pharmaceuticals Inc
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

Treatment of Pediatric Subjects with Pearson syndrome

Description:

If effective, treatment with EPI-743 should result in a significant reduction in occurrence of sepsis, metabolic crisis, hepatic failure and transfusion dependence.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 2
• Est. completion date: February 29, 2016
• Est. primary completion date: November 30, 2015
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A to 18 Years

Eligibility

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

Related Conditions & MeSH terms

• Lipid Metabolism, Inborn Errors
• Mitochondrial Diseases
• Muscular Diseases
• Pearson Syndrome
• Syndrome

Intervention

Drug:
• EPI-743
EPI-743 is the quinone oxidation product of alpha-tocotrienol

Locations

Country	Name	City	State
United States	Cleveland Clinic	Cleveland	Ohio
United States	Baylor College of Medicine	Houston	Texas
United States	Children's Hospital of Orange County	Orange	California
United States	Children's Hospital of Pittsburgh	Pittsburgh	Pennsylvania

Sponsors (1)

Lead Sponsor	Collaborator
Edison Pharmaceuticals Inc

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Number of Dose Limiting SAEs	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
Primary	Occurence of episodes of sepsis, metabolic crisis or hepatic faillure		1 year
Secondary	Transfusion avoidance		1 year
Secondary	Hematologic function		1 year
Secondary	Neuromuscular function	Neuromuscular function as assessed by Gross Motor Function Measure	1 year
Secondary	Disease severity	Disease severity as assessed by Newcastle Pediatric Mitochondrial Disease Scale	1 year
Secondary	Renal function		1 year
Secondary	Hepatic function		1 year
Secondary	Weight gain		1 year
Secondary	Hospitalizations		1 year
Secondary	Pancreatic function	Pancreatic function as assessed by insulin requirement and hemoglobin A1c	1 year
Secondary	Mortality		1 year