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
Sponsor: Applied Genetic Technologies Corp
Principal Investigator: Kimberly Stepien, MD
Study Coordinator: Nickie Stangel
To evaluate the safety and efficacy of AGTC-402 administered to one eye by subretinal injection in individuals with achromatopsia caused by mutations in the CNGA3 gene.
The study involves 12 visits the first year, and annual visits over the next 4 years. Subjects will be enrolled sequentially in to one of seven dosing groups. Subjects will either receive varying dose levels of study agent or the maximum tolerated dose.
Male or female subjects with documented mutations in both alleles of the CNGA3 gene;
Retinal disease consistent with a clinical diagnosis of achromatopsia;
At least 18 years of age for Groups 1, 2, 3, 4, and 5 and at least 6 years of age for Groups 3a, 4a and 6;
Able to perform tests of visual and retinal function;
Visual acuity in the study eye not better than 55 ETDRS letters (Snellen equivalent 20/80) based on the average of two examinations at the baseline visit;
Acceptable laboratory parameters;
For females of childbearing potential: A negative pregnancy test within 2 days before administration of study agent.
Best-corrected visual acuity difference between the two eyes of > 15 ETDRS letters (3 lines);