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

Study Results

Results pending

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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Recruitment Status

NO_LONGER_AVAILABLE

Study Classification

EXPANDED_ACCESS

Brief Summary

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Expanded access Protocol to treat LHON subjects with EPI743

Detailed Description

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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.

Conditions

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Leber's Hereditary Optic Neuropathy

Interventions

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EPI-743

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

Intervention Type DRUG

Other Intervention Names

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Vincerinone (TM) Vatiquinone

Eligibility Criteria

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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

Minimum Eligible Age

8 Years

Maximum Eligible Age

65 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Responsible Party

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Responsibility Role SPONSOR

Locations

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Doheny Eye Institute / UCLA

Los Angeles, California, United States

Site Status

Countries

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United States

Other Identifiers

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EPI-2010-004

Identifier Type: -

Identifier Source: org_study_id