Long-term Follow-up Study in Subjects Who Received Voretigene Neparvovec-rzyl (AAV2-hRPE65v2)

Spark Therapeutics

Status: Active, not recruiting

Conditions: Inherited Retinal Dystrophy Due to RPE65 Mutations

Treatments: Biological: AAV2-hRPE65v2

Study type: Observational

Funder types: Industry

Identifiers

NCT03602820

AAV2-hRPE65v2-LTFU-01

Details and patient eligibility

About

Multi-site, non-randomized, observational study, for up to 15 years after subretinal AAV2-hRPE65v2 administration for each subject. The study is a non-interventional, follow-up study of subjects who participated in previous AAV2-hRPE65v2 gene therapy clinical trials.

Full description

This is an observational follow-up study of subjects who participated in previous Phase 1 and Phase 3 clinical trials of AAV2-hRPE65v2 gene therapy (voretigene neparvovec-rzyl) to evaluate long term durability and safety for 15 years after subretinal administration.

Enrollment: 41 patients

Sex: All

Volunteers: No Healthy Volunteers

Inclusion criteria

1. Subjects who participated in prior subretinal AAV2-hRPE65v2 gene therapy clinical studies

Exclusion criteria

1. Subjects who will not consent for study.
2. Subjects who the investigators believe are not capable of performing study assessments