Phase 2 Study of EPI-743 in Children With Pearson Syndrome (NCT02104336) | Clinical Trial Compass
TerminatedPhase 2
Phase 2 Study of EPI-743 in Children With Pearson Syndrome
Stopped: Results from other studies did not support continuation of this trial
United States2 participantsStarted 2014-08-31
Plain-language summary
Treatment of Pediatric Subjects with Pearson syndrome
Who can participate
Age range18 Years
SexALL
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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
What they're measuring
1
Occurence of episodes of sepsis, metabolic crisis or hepatic faillure