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

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

Status
NO_LONGER_AVAILABLE
Phases
Unknown
Study type
Expanded Access
Source
ClinicalTrials.gov
Registry ID
NCT02300753
Enrollment
Unknown
Registered
2014-11-25
Start date
Unknown
Completion date
Unknown
Last updated
2014-11-25

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Leber's Hereditary Optic Neuropathy

Brief summary

Expanded access Protocol to treat LHON subjects with EPI743

Detailed description

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.

Interventions

DRUGEPI-743

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

Sponsors

Edison Pharmaceuticals Inc
Lead SponsorINDUSTRY

Eligibility

Sex/Gender
ALL
Age
8 Years to 65 Years

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

Countries

United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026