A Study to Investigate the Safety of OpCT-001 in Adults Who Have Primary Photoreceptor Disease (CLARICO)
A Phase 1/2a Study of Subretinal Administration of OpCT-001 Photoreceptor Precursor Cells Derived From iPSCs in Patients With Primary Photoreceptor Disease
About This Trial
Study OpCT-001-101 is a Phase 1/2a first-in-human, multisite, 2-part interventional study to evaluate the safety, tolerability, and the effect on clinical outcomes of OpCT-001 in up to approximately 54 adults with primary photoreceptor (PR) disease. Phase 1 will focus on safety and features a dose-escalation design. Phase 2 is designed to gather additional safety data and assess the effect of OpCT-001 on measures of visual function, functional vision, and anatomic measures of engraftment in different clinical subgroups.
Who May Be Eligible (Plain English)
Original Eligibility Criteria
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Treatments Being Tested
OpCT-001
Cell therapy product composed of photoreceptor precursor cells derived from human induced pluripotent stem cells (iPSCs).
OpCT-001
Cell therapy product composed of photoreceptor precursor cells derived from human iPSCs.