A Multiple-Site, Phase 1/2, Safety and Efficacy Trial of a Recombinant Adeno-associated Virus Vector Expressing CNGB3 (rAAV2tYF-PR1.7-hCNGB3) in Patients with Congenital Achromatopsia Caused by Mutations in the CNGB3 Gene (AGTC B3)
Sponsor: Applied Genetic Technologies Corporation
Principal Investigator: Kimberly Stepien, MD
Study Coordinator: Nickie Stangel
This study will evaluate the safety and efficacy of AGTC-401 in patients with congenital achromatopsia caused by mutations in the CNGB3 gene
Study participants will be enrolled into 7 non-randomized groups. Each participant will receive AGTC-401 in a volume of up to 0.30 mL administered by subretinal injection in one eye on a single occasion during the study.
Diagnosis and Key Eligibility Criteria:
Male or females with documented mutations in both alleles of the CNGB3 gene with confirmation by segregation analysis for subjects with compound heterozygous mutations wherever possible, and retinal disease consistent with a clinical diagnosis of achromatopsia. Ages ≥18 years for groups 1-6 and ≥6 for groups 4a, 5a, and 7. Prior use of any AAV gene therapy product is not allowed.
Duration of study: Approximately 5 years
For more information, contact Nickie Stangel 608-263-8783