Phase I/IIa Dose Escalation Safety and Efficacy Study of Human Embryonic Stem Cell-Derived Retinal Pigment Epithelium Cells Transplanted Subretinally in Patients With Advanced Dry-Form Age-Related Macular Degeneration (Geographic Atrophy)
The main objective of the study is evaluation of the safety and tolerability of OpRegen - Human embryonic stem cell-derived retinal pigment epithelial (RPE) cells. The study will also include initial exploration of the ability of transplanted OpRegen cells to engraft, survive, and moderate disease progression.
• Age 50 and older;
• Diagnosis of dry (non-neovascular) age related macular degeneration in both eyes;
• Funduscopic findings of dry age-related macular degeneration (AMD) with progressive geographic atrophy in the macula;
• Best corrected central visual acuity equal or less than 20/200 in cohorts 1-3 and 20/64-20/250 in cohort 4 in the study eye by ETDRS vision testing;
• Vision in the non-operated eye must be better than or equal to that in the operated eye;
• Subjects with sufficiently good health to allow participation in all study-related procedures and complete the study follow up period (based on medical records);
• Ability to undergo a vitreoretinal surgical procedure under monitored anesthesia care;
• Blood counts, blood chemistry, coagulation and urinalysis without abnormal significance;
• Negative for tuberculosis (TB) (cohort 4), human immunodeficiency virus (HIV), hepatitis B (HBC), and hepatitis C virus (HCV), negative for cytomegalovirus (CMV) Immunoglobulin (IgM) and Epstein-Barr Virus (EBV) IgM or asymptomatic in the opinion of the investigator (cohort 4);
• No history of malignancy (other than a non-melanoma skin cancer). For cancers in remission for more then 5 years enrollment is allowed with concurred documented approval of principal investigator and oncologist prior to enrollment;
• Willing to defer all future blood and tissue donation;
• Able to understand study procedures and willing to sign informed consent.