Home » Clinical Trials » Inherited retinal dystrophy » NCT03597399
NCT03597399

A Post-Authorization, Multicenter, Longitudinal, Observational Safety Registry Study for Patients Treated With Voretigene Neparvovec in US

Study Summary

The objective of this study is to collect long-term safety information (i.e., for 5 years after treatment) associated with voretigene neparvovec-rzyl (vector and/or transgene), its subretinal injection procedure, the concomitant use of corticosteroids, or a combination of these procedures and products. The enrollment period will last for two years from the first treatment following product approval (through 31March2020) and include a minimum of 40 patients.

Want to learn more about this trial?

Request More Info

Interventions

AAV2-hRPE65v2,voretigene neparvovec-rzylBIOLOGICAL
Subretinal administration of gene therapy vector AAV2-hRPE65v2 (voretigene neparvovec-rzyl) to both eyes via surgical procedures on separate days.

Study Locations

FacilityCityStateCountry
Children's Hospital of Los AngelesLos AngelesCaliforniaUnited States
Bascom Palmer Eye InstituteMiamiFloridaUnited States
University of Iowa Hospitals & ClinicsIowa CityIowaUnited States
Massachusetts Eye and Ear InstituteBostonMassachusettsUnited States
Kellogg Eye CenterAnn ArborMichiganUnited States
Cincinnati Eye InstituteCincinnatiOhioUnited States
Casey Eye InstitutePortlandOregonUnited States
Children's Hospital of Philadelphia (CHOP)PhiladelphiaPennsylvaniaUnited States
Scheie Eye InstitutePhiladelphiaPennsylvaniaUnited States
Cullen Eye InstituteHoustonTexasUnited States

Official Trial Information

View on ClinicalTrials.gov

Data sourced from ClinicalTrials.gov. Last updated: April 14, 2026