Saves the questions and what to expect into your notes, next to the visit they belong to.
Keep this study
Lead
Edison Pharmaceuticals Inc
If effective, treatment with EPI-743 should result in a significant reduction in occurrence of sepsis, metabolic crisis, hepatic failure and transfusion dependence.
Age
0–18
Sex
ALL
Healthy volunteers
Not accepted
You may be eligible if
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
You may not be if
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
Clareo Health | Phase 2 Study of EPI-743 in Children With Pearson Syndrome
