Trials / Active Not Recruiting
Active Not RecruitingNCT03602820
Long-term Follow-up Study in Subjects Who Received Voretigene Neparvovec-rzyl (AAV2-hRPE65v2)
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
- Phase
- —
- Study type
- Observational
- Enrollment
- 41 (actual)
- Sponsor
- Spark Therapeutics, Inc. · Industry
- Sex
- All
- Age
- —
- Healthy volunteers
- Not accepted
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.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| BIOLOGICAL | AAV2-hRPE65v2 |
Timeline
- Start date
- 2015-06-01
- Primary completion
- 2030-01-01
- Completion
- 2030-01-01
- First posted
- 2018-07-27
- Last updated
- 2025-04-18
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT03602820. Inclusion in this directory is not an endorsement.