Safety Study of an Adeno-associated Virus Vector for Gene Therapy of Leber’s Hereditary Optic Neuropathy (LHON)
An Open-label Dose Escalation Study of an Adeno-associated Virus Vector (scAAV2-P1ND4v2) for Gene Therapy of Leber’s Hereditary Optic Neuropathy (LHON) Caused by the G11778A Mutation in Mitochondrial DNA
Doctors at the Bascom Palmer Eye Institute of the University of Miami are recruiting participants with a specific type of Leber’s Hereditary Optic Neuropathy (LHON) to help assess the effectiveness of an investigational gene therapy to improve retina structure and function. The mutation targeted by this treatment is the G1178A mitochondrial mutation, where vision has been lost up to one year. The treatment, a genetically modified virus, is designed to impart the functional copy of the gene to cells of the eyes.
Participants will be assigned to receive the investigational drug in both eyes at one of 4 doses. Patients will be followed up to 2 years post treatment.
- Be 15 years of age or older
- Have vision loss of a certain duration of time due to LHON, in at least one eye
- Not have had previous eye surgery in the eye selected for injection
THE STUDY INVOLVES:
- Prescreening to confirm eligibility of the patient to participate
- A single intravitreal injection into one or both eyes
- Follow up will continue for 1 year.
LOCATIONS AND CONTACTS:
JOHN GUY, MD | 302-326-6036 | [email protected]
The trial takes place at the Bascom Palmer Eye Institute of the University of Miami. Map.
National Eye Institute (NEI)
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