Clinical trial

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

Name

AAV2-hRPE65v2-LTFU-01

Description

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.

Trial arms

Trial start

2015-06-01

Estimated PCD

2030-03-01

Trial end

2030-06-01

Status

Active (not recruiting)

Treatment

AAV2-hRPE65v2

Other names:

Voretigene Neparvovec-rzyl

Size

Primary endpoint

Mobility testing, Bilateral

15 years

Eligibility criteria

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

Protocol

{'studyType': 'OBSERVATIONAL', 'patientRegistry': False, 'designInfo': {'observationalModel': 'COHORT', 'timePerspective': 'PROSPECTIVE'}, 'enrollmentInfo': {'count': 41, 'type': 'ACTUAL'}}

Updated at

2024-02-26

1 organization

1 product

1 indication

Organization

Spark Therapeutics

Product

AAV2-hRPE65v2

Indication

Leber Congenital Amaurosis