Emergency Administration of EPI-743 to a Single Patient With Leber's Hereditary Optic Neuropathy [LHON]
- Conditions
- Leber's Hereditary Optic Neuropathy
- Registration Number
- NCT02300753
- Lead Sponsor
- Edison Pharmaceuticals Inc
- 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.
Recruitment & Eligibility
- Status
- NO_LONGER_AVAILABLE
- Sex
- All
- Target Recruitment
- Not specified
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
Study & Design
- Study Type
- EXPANDED_ACCESS
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (1)
Doheny Eye Institute / UCLA
🇺🇸Los Angeles, California, United States