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
Overview
- Phase
- Not Applicable
- Intervention
- Not specified
- Conditions
- Inherited Retinal Dystrophy Due to RPE65 Mutations
- Sponsor
- Spark Therapeutics, Inc.
- Enrollment
- 41
- Primary Endpoint
- Mobility testing, Bilateral
- Status
- Active, not recruiting
- Last Updated
- last year
Overview
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.
Investigators
Eligibility Criteria
Inclusion Criteria
- •Subjects who participated in prior subretinal AAV2-hRPE65v2 gene therapy clinical studies
Exclusion Criteria
- •Subjects who will not consent for study.
- •Subjects who the investigators believe are not capable of performing study assessments
Outcomes
Primary Outcomes
Mobility testing, Bilateral
Time Frame: 15 years
Mobility testing will be videotaped at each study visit at which it is conducted. Independent reviewers may grade subjects' mobility videos. Graders will use a defined combination of speed and accuracy to grade each course attempt at a given light intensity. The course will be re-configured between each attempt, using twelve standardized templates, to reduce the impact of a potential learning effect. Each subject will be tested, using both eyes, under at least two and sometimes three different (specified) lighting conditions. The levels of light, selected to span lighting conditions that individuals encounter in daily life, range from a studio with floodlights (400 lux) or a brightly lit office (250 lux) down to a poorly lit sidewalk at night (1 lux).
Secondary Outcomes
- Full-field light sensitivity threshold (FST) testing(15 years)
- Visual acuity(15 years)
- Mobility testing, Monocular(15 years)