This is a prospective, observational study designed to evaluate the long-term safety and efficacy of RGX-314. Eligible participants are those who were previously enrolled in a clinical study in which they received a single subretinal administration of RGX-314 in their study eye. Enrollment of each participant in the current study should occur after the participant has completed either the end of study or early termination visit in the previous (parent) clinical study. Participants will be followed for up to 5 years post-RGX-314 administration (inclusive of the parent study). After enrollment and a 6-month follow-up visit, participants will attend at least annual study visits through the end of the 5-year post-RGX-314 administration follow-up period. Additionally, an interventional fellow eye treatment substudy will evaluate the safety, efficacy, and immunogenicity of subretinal RGX-314 administration in the fellow eye of participants having bilateral disease who previously received a subretinal injection of RGX-314 in their study eye. Participants who qualify for the substudy will receive subretinal administration of RGX-314 in their fellow eye and complete 13 study visits in a 54-week period. Following completion of the substudy participants will continue in the observational portion of the study for up to 5 years post RGX-314 administration in their fellow eye.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
865
AAV8 vector containing a transgene for anti-VEGF Fab
Retinal Research Institute, LCC
Phoenix, Arizona, United States
California Retina Consultants CRC
Santa Barbara, California, United States
Johns Hopkins University
Baltimore, Maryland, United States
Ophthalmic Consultants of Boston
Boston, Massachusetts, United States
Sierra Eye Associates
Reno, Nevada, United States
University of Pennsylvania Department of Ophthalmology
Philadelphia, Pennsylvania, United States
MidAtlantic Retina
Philadelphia, Pennsylvania, United States
Charles Retina Institute
Germantown, Tennessee, United States
Retina Consultants of Texas Research Center
The Woodlands, Texas, United States
Incidence of ocular adverse events and any serious adverse events in the study eye
Time frame: 5 years inclusive of parent study
Fellow eye substudy: Incidence of ocular adverse events and any serious adverse events in the fellow eye
Time frame: 54 weeks
Change from baseline in best corrected visual acuity (BCVA) in the study eye
Mean change and proportion of participants losing greater than or equal to 5, 10, and 15 letters
Time frame: 5 years inclusive of parent study
Change from baseline in Central Retinal Thickness (CRT) in the study eye
Mean change from baseline in CRT as measured by spectral domain optical coherence tomography (SD-OCT)
Time frame: 5 years inclusive of parent study
Number of supplemental anti-VEGF injections in the study eye
Mean number of supplemental anti-VEGF injections based on chart review
Time frame: 5 years inclusive of parent study
Number of retinal specialist visits for the study eye
Mean number of retinal specialist visits attended for nAMD based on chart review
Time frame: 5 years inclusive of parent study
Fellow eye substudy: Change from baseline in best corrected visual acuity
Mean change from baseline in BCVA in the fellow eye
Time frame: 54 weeks
Fellow eye substudy: Change from baseline in Central Retinal Thickness in the fellow eye
Mean change from baseline in CRT in the fellow eye as measured by SD-OCT
Time frame: 54 weeks
Fellow eye substudy: Number of supplemental anti-VEGF injections in the fellow eye
Mean number of supplemental anti-VEGF injections in the fellow eye
Time frame: 54 weeks
Fellow eye substudy: Aqueous and Serum RGX-314 transgene product (TP) concentrations in the fellow eye
Fellow eye aqueous and serum RGX-314 TP concentrations at assessed time points
Time frame: 54 weeks
Fellow eye substudy: Immunogenicity measurements in the fellow eye
Fellow eye immunogenicity measurements at assessed timepoints
Time frame: 54 weeks
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