Long-term Follow-up Study in Subjects Who Received Voretigene Neparvovec-rzyl (AAV2-hRPE65v2)
Active, not recruiting · NCT03602820
Conditions: Inherited Retinal Dystrophy Due to RPE65 Mutations
At a glance
- Research payments reported, 2025
- $715,731
- U.S. investigators named
- 2
- Sites paid
- 3
- Planned or actual enrollment
- 41
About the trial
- Sponsor
- Genentech, Inc.
- Tested
- AAV2-hRPE65v2 (biological)
- Dates
- Started 2015-06; completion 2030-01
- Results posted
- Not yet
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.
From ClinicalTrials.gov, last updated 2026-08-19. Full record, eligibility and contacts (opens in a new tab)
Industry funding
Spark Therapeutics, Inc. reported $715,731 in research payments for this study in 2025 (36 payments).
Products named: Luxturna
Research payments usually go to hospitals, universities and research sites to run a study, not to the investigators personally. A payment is not evidence of wrongdoing.
U.S. investigators named on this study
- Stephen R. Russell, MD Ophthalmology · Iowa City, IA
- Albert M. Maguire, MD Retina Specialist · Philadelphia, PA
Clinicians the company named as principal investigators in its Open Payments reports.
Sites paid to run it
Sources: CMS Open Payments research payments (program year 2025) and ClinicalTrials.gov, U.S. National Library of Medicine.