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This Phase 1/Phase 2 study follows up on voretigene neparvovec-rzyl, an eye gene therapy, in patients with Leber congenital amaurosis type 2, an inherited form of blindness linked to the RPE65 gene. It included patients who had previously received AAV2-hRPE65v2 in one eye in a Phase 1 study and who have enough healthy retinal cells in the other eye. It was carried out in Phase 1/Phase 2 and is active but not currently recruiting participants.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
Inclusion Criteria: * Prior participation in Phase 1 study with unilateral, subretinal administration of AAV2-hRPE65v2. * Visual acuity equal to or greater than light perception. * Sufficient viable retinal cells in contralateral, previously uninjected eye, as determined by non-invasive means, such as optical coherence tomography (OCT) and/or ophthalmoscopy. Must have either: 1) an area of retina within the posterior pole of \> 100 µm shown on OCT; 2) ≥ 3 disc areas of retina without atrophy or pigmentary degeneration within the posterior pole; or 3) remaining visual field within 50 degrees of fixation. * Willingness to adhere to protocol and long-term follow-up as evidenced by written informed consent or parental permission and subject assent (where applicable). Exclusion Criteria: * Unable or unwilling to meet requirements of the study. * Participation in any other study of an investigational drug within the past six months. * Use of retinoid compounds or precursors that could potentially interact with the biochemical activity of the RPE65 enzyme; individuals who discontinue use of these compounds for 18 months may become eligible. * Prior intraocular surgery within six months. * Known sensitivity to medications planned for use in the peri-operative period. * Pre-existing eye conditions, such as glaucoma, or complicating systemic diseases that would preclude the planned surgery or could interfere with the interpretation of study. Complicating systemic diseases would include those in which the disease itself, or the treatment for the disease, can alter ocular function. Examples are malignancies whose treatment could affect central nervous system function (for example: radiation treatment of the orbit; leukemia with CNS/optic nerve involvement). Subjects with diabetes or sickle cell disease would be excluded if they had any manifestation of advanced retinopathy (e.g. macular edema or proliferative changes). Also excluded would be subjects with immunodeficiency (acquired or congenital) as there could be susceptibility to opportunistic infection (such as CMV retinitis). * Individuals of childbearing potential who are pregnant or unwilling to use effective contraception for four months following vector administration. * Any other condition that would not allow the potential subject to complete follow-up examinations during the course of the study and, in the opinion of the investigator, makes the potential subject unsuitable for the study. * Subjects will NOT be excluded based on their gender, race, or ethnicity.