Emergency Administration of EPI-743 to a Single Patient With Leber's Hereditary Optic Neuropathy [LHON]

N/ANo Longer AvailableNCT02300753

Edison Pharmaceuticals Inc

Overview

Expanded access Protocol to treat LHON subjects with EPI743

Emergency use administration of EPI-743 to subjects with LHON. This Protocol was originally developed to treat a single subject but was expanded to treat several at a single institution.

Study Type

EXPANDED_ACCESS

Conditions

Leber's Hereditary Optic Neuropathy

Interventions

EPI-743DRUG

EPI-743 is a quinone oxidation product of alpha-tocotrienol

Eligibility

Sex: ALLMin age: 8 YearsMax age: 65 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Emergency treatment for subjects with LHON who are actively losing site in one eye Exclusion Criteria: * Allergy to EPI-743 or sesame oil * Clinical history of bleeding or abnormal PT/PTT * Hepatic insufficiency with LFTs greater than 2-times normal * Renal insufficiency requiring dialysis * Fat malabsorption syndromes * Any other concurrent inborn erros of metabolism * Severe end-organ hypo-perfusion syndrome secondary to cardiac failure resulting in lactic acidosis * Anemia with a HCT \<25

Locations (1)

Doheny Eye Institute / UCLA

Los Angeles, California, United States

Data from ClinicalTrials.gov

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