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

Edison Pharmaceuticals

Status and phase: Terminated; Phase 2

Conditions: Pearson Syndrome

Treatments: Drug: EPI-743

Study type: Interventional

Funder types: Industry

Identifiers

NCT02104336

EPI743-13-024

Details and patient eligibility

About

Treatment of Pediatric Subjects with Pearson syndrome

Full description

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

Enrollment: 2 patients

Sex: All

Ages: Under 18 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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2 participants in 1 patient group

EPI-743

Experimental group

Description:

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

Treatment:

Drug: EPI-743