Long-term Follow-up Study in Subjects Who Received Voretigene Neparvovec-rzyl (AAV2-hRPE65v2)
Long-term Follow-up Study in Subjects Who Received Voretigene Neparvovec-rzyl (AAV2-hRPE65v2)
Brief Summary:
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.
Detailed 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.
Study Type: Observational
Actual Enrollment: 41 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: A Long-Term Follow-Up Study in Subjects Who Received an Adenovirus-Associated Viral Vector Serotype 2 Containing the Human RPE65 Gene (AAV2-hRPE65v2, Voretigene Neparvovec-rzyl) Administered Via Subretinal Injection
Actual Study Start Date: June 2015
Estimated Primary Completion Date: March 2030
Estimated Study Completion Date: June 2030
| Category | Value |
|---|---|
| Study type(s) | Observational |
| Actual enrolment | 41 |
| Actual Study start date | 01 June 2015 |
| Estimated Study Completion Date | 01 June 2030 |