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A Study to Investigate the Safety of OpCT-001 in Adults Who Have Primary Photoreceptor Disease (CLARICO)
Sponsor: BlueRock Therapeutics
Summary
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 approximately 54 adults with primary photoreceptor (PR) disease. Phase 1 focuses 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.
Official title: A Phase 1/2a Study of Subretinal Administration of OpCT-001 Photoreceptor Precursor Cells Derived From iPSCs in Patients With Primary Photoreceptor Disease
Key Details
Gender
All
Age Range
18 Years - Any
Study Type
INTERVENTIONAL
Enrollment
54
Start Date
2025-03-10
Completion Date
2030-10
Last Updated
2026-04-13
Healthy Volunteers
No
Conditions
Interventions
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.
Locations (4)
University of Miami, Bascom Palmer Eye Institute
Miami, Florida, United States
Mid Atlantic Retina/ Wills Eye
Philadelphia, Pennsylvania, United States
Retina Foundation of the Southwest
Dallas, Texas, United States
University of Wisconsin
Madison, Wisconsin, United States