Trials / NCT06789445
A Study to Investigate the Safety of OpCT-001 in Adults Who Have Primary Photoreceptor Disease (CLARICO)
Recruiting now Phase 1 / Phase 2 Gene therapy, Cell therapy
- Sponsor
- BlueRock Therapeutics
- Treatment
- OpCT-001; OpCT-001
- Gene
- Not stated
- Conditions
- Primary Photoreceptor Disease, Retinitis Pigmentosa (RP), Usher Syndrome, Inherited Retinal Disease (IRD), Rod-Cone Dystrophy, Rod-Cone Disease, Retinal Degeneration, Cone-Rod Disease (C-RD), Cone-Rod Dystrophy
- Ages
- 18 Years to no upper limit
- Enrollment
- 54 participants
- Started
- 2025-03-10
- Main results expected
- 2029-10
- Results posted
- Not yet
- Record last updated
- 2026-10-15
What the study is testing
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.
Where it runs
United States (5)
- University of Southern California /Children's Hospital of Los Angeles, Los Angeles, California (recruiting now)
- University of Miami, Bascom Palmer Eye Institute, Miami, Florida (recruiting now)
- Mid Atlantic Retina/ Wills Eye, Philadelphia, Pennsylvania (recruiting now)
- Retina Foundation of the Southwest, Dallas, Texas (recruiting now)
- University of Wisconsin, Madison, Wisconsin (recruiting now)