Trial of AGTC-402 In Patients with Congenital Achromatopsia caused by Mutations in the CNGA3 Gene (H-45519)
A Multiple Site Phase 1/2, Safety and Efficacy Trial of AGTC-402, A Recombinant Adeno-Associated Virus Vector Expressing CNGA3, In Patients with Congenital Achromatopsia caused by Mutations in the CNGA3 Gene
Global, non-randomized, open-label, Phase 1/2 dose escalation study. Adult and pediatric participants will be enrolled into six groups. Each will receive AGTC-402 (a recombinant adeno-associate virus vector that expresses the CNGA3 gene) in a volume of up to 0.30mL administered by subretinal injection in one eye on a single occasion. Safety will be monitored by evaluation of ocular and non-ocular adverse events and hematology and clinical parameters. Efficacy will be measured by evaluation in visual acuity and series of diagnostic imaging. Congenital Achromatopsia is an autosomal recessive inherited disease that causes severe vision loss.