Study of Stem Cell-Derived Retinal Pigment Epithelium Transplantation for Geographic Atrophy in Dry Age-Related Macular Degeneration

What this Trial Is About

Age-related macular degeneration AMD is a common eye condition in people over 50, with the dry form potentially progressing to geographic atrophy GA that causes vision loss. Researchers aim to evaluate if replacing older eye cells with younger ones using stem cell techniques can help treat GA in dry AMD. This trial focuses on testing the safety and feasibility of transplanting retinal pigment epithelium RPE cells derived from a participants own induced pluripotent stem cells iPSC as a potential treatment for GA associated with AMD. Participants will undergo surgery to transplant iPSC-derived RPE cells grown on a biodegradable scaffold into one eye affected by GA. The cells are prepared from the participants blood and surgically placed under the retina through a small cut. During surgery, participants receive anesthesia and a gas bubble will be inserted in the eye to support healing. Immunosuppressive medications will be given to help prevent transplant rejection. This phase IIIa trial will enroll up to 20 participants in two cohorts based on visual acuity ranges, with follow-up evaluations planned for five years after surgery. Participants will have at least 14 study visits over 5 and a half years, including eye exams, imaging tests like fluorescein angiography and optical coherence tomography OCT, electrophysiology assessments, and vision tests. Safety will be monitored by tracking adverse events and changes in visual acuity at 12 months and beyond. Secondary outcomes include detailed retinal structure and function assessments at 12, 24, and 60 months. Participants will also be contacted yearly for up to 15 years to monitor long-term safety and effects.

Autologous Transplantation of Induced Pluripotent Stem Cell-Derived Retinal Pigment Epithelium for Geographic Atrophy Associated With Age-Related Macular Degeneration