clinical trial · NCT03602820
Long-term Follow-up Study in Subjects Who Received Voretigene Neparvovec-rzyl (AAV2-hRPE65v2)
Spark Therapeutics, Inc.·—·active not recruiting·n = 41
Inherited Retinal Dystrophy Due to RPE65 MutationsAAV2-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.
started
Jun 1, 2015
primary completion
Jan 1, 2030
completion
Jan 1, 2030
last updated
Apr 18, 2025
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.
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
sourced from ClinicalTrials.gov · pharmadog mirrors structured fields, not the full protocol