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

Inherited Retinal Dystrophy Due to RPE65 Mutations Clinical Trial

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

Status Active, not recruiting

Clinical Trial 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.

Clinical Trial 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 Design

Related Conditions & MeSH terms

Inherited Retinal Dystrophy Due to RPE65 Mutations
Retinal Dystrophies

Clinical Trial Details

NCT number	NCT03602820
Study type	Observational
Source	Spark Therapeutics
Contact
Status	Active, not recruiting
Phase
Start date	June 2015
Completion date	June 2030

View Details

See also
	Status	Clinical Trial	Phase
	Active, not recruiting	`NCT00999609` - Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis	Phase 3