Phase 2 Study of EPI-743 in Children With Pearson Syndrome
Launched by EDISON PHARMACEUTICALS INC · Apr 3, 2014
Trial Information
Current as of July 16, 2025
Terminated
Keywords
ClinConnect Summary
If effective, treatment with EPI-743 should result in a significant reduction in occurrence of sepsis, metabolic crisis, hepatic failure and transfusion dependence.
Gender
ALL
Eligibility 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
About Edison Pharmaceuticals Inc
Edison Pharmaceuticals Inc. is a biopharmaceutical company dedicated to advancing innovative therapies for the treatment of rare and complex diseases, particularly those related to mitochondrial dysfunction. With a focus on developing targeted drug candidates that address unmet medical needs, Edison combines cutting-edge scientific research with a commitment to patient-centric solutions. The company leverages a robust portfolio of proprietary compounds and collaborates with leading researchers and institutions to drive clinical development and bring transformative treatments to market. Through its rigorous clinical trial programs, Edison Pharmaceuticals aims to improve the quality of life for patients and their families affected by debilitating conditions.
Contacts
Jennifer Cobb
Immunology at National Institute of Allergy and Infectious Diseases (NIAID)
Locations
Houston, Texas, United States
Orange, California, United States
Cleveland, Ohio, United States
Pittsburgh, Pennsylvania, United States
Patients applied
Trial Officials
Matthew Klein, MD, FACS
Study Chair
Edison Pharmaceuticals
Timeline
First submit
Trial launched
Trial updated
Estimated completion
Not reported
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