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

Edison Pharmaceuticals

Status

Conditions: Leber's Hereditary Optic Neuropathy

Treatments: Drug: EPI-743

Study type: Expanded Access

Funder types: Industry

Identifiers

NCT02300753

EPI-2010-004

Details and patient eligibility

About

Expanded access Protocol to treat LHON subjects with EPI743

Full 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.

Sex: All

Ages: 8 to 65 years old

Volunteers: No Healthy Volunteers

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