A Phase I/IIa Trial for Autologous Transplantation of Induced Pluripotent Stem Cell-Derived Retinal Pigment Epithelium for Geographic Atrophy Associated with Age-Related Macular Degeneration

National Eye Institute

Investigators

Abstract

This Phase I/IIa study aims to evaluate the safety and feasibility of subretinal transplantation of iPSC-derived RPE, grown as a monolayer on a biodegradable poly lactic-co-glycolic acid (PLGA) scaffold, as a potential autologous cell-based therapy for GA associated with AMD. Five participants with GA, best-corrected visual acuity (BCVA) between 20/100 and inclusive of counting fingers (CF), and a fellow eye that has same or better BCVA will undergo subretinal transplantation of autologous iPSC-derived RPE in one eye and will be followed for five years after surgery. A second cohort of seven additional participants with GA, BCVA between 20/80 and CF (inclusive) in the eye being considered for RPE transplantation, and same or better visual acuity in the other eye may undergo the procedure to gather additional safety and potential efficacy data useful for planning future studies. Up to 20 participants may be enrolled. The primary outcome measure is the safety of RPE/PLGA transplantation, as determined by assessment of visual acuity change and summary of adverse events at 12 months after RPE/PLGA transplantation. Secondary outcome measures include visual acuity change and adverse event reporting at 24 and 60 months, and changes in the following at 12, 24 and 60 months as compared with baseline, assessed in the transplanted region, and compared where applicable with other areas in the macula, and/or with corresponding regions in the fellow eye. During the past year we have continued to screen, recruit patients, and also finalized the surgical transplantation efforts for the clinical trial.