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

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

Treatment of Pediatric Subjects with Pearson syndrome

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

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