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

• Status: Terminated
• Trial end date: 2016-02-29
• Target enrollment: 0
• Participant gender: All

Summary

Treatment of Pediatric Subjects with Pearson syndrome

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Edison Pharmaceuticals Inc
• Treatments: Ubiquinone

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