Phase 2 Study of EPI-743 in Children With Pearson Syndrome
NCT ID: NCT02104336
Last Updated: 2020-11-18
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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TERMINATED
PHASE2
2 participants
INTERVENTIONAL
2014-08-31
2016-02-29
Brief Summary
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Detailed Description
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Conditions
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Keywords
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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EPI-743
15 mg/kg EPI-743 to be administered three times per day for 1 year
EPI-743
EPI-743 is the quinone oxidation product of alpha-tocotrienol
Interventions
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EPI-743
EPI-743 is the quinone oxidation product of alpha-tocotrienol
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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
* 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
18 Years
ALL
No
Sponsors
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Edison Pharmaceuticals Inc
INDUSTRY
Responsible Party
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Principal Investigators
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Matthew Klein, MD, FACS
Role: STUDY_CHAIR
Edison Pharmaceuticals
Locations
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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
Countries
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Other Identifiers
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EPI743-13-024
Identifier Type: -
Identifier Source: org_study_id