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

Stopped early · Phase 2

Conditions studied: Pearson Syndrome

In brief

Treatment of Pediatric Subjects with Pearson syndrome

Key facts

Study ID

NCT02104336

Run by

Edison Pharmaceuticals Inc

People needed

2

Starts

2014-08-31

Expected to finish

2016-02-29

Last updated by the study team

2020-11-18

Who can join

Age: any, up to 18. Sex: any. Healthy volunteers: not accepted.

You may qualify 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 qualify 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

Where it is running

• Children's Hospital of Orange County — Orange, California, United States
• Cleveland Clinic — Cleveland, Ohio, United States
• Children's Hospital of Pittsburgh — Pittsburgh, Pennsylvania, United States
• Baylor College of Medicine — Houston, Texas, United States

Full record on ClinicalTrials.gov

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