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.
Confirmed genetic diagnosis of primary photoreceptor (PR) disease
Best corrected visual acuity (BCVA) in the study eye at Screening for Phase 1: Logmarithm of the minimum angle of resolution (LogMAR) 3.9 to LogMAR 1.3. BCVA at Screening for Phase 2: ETDRS letter score between 20 to 60, inclusive.
Retinal structure examination in the study eye demonstrating regions suitable for cell administration.
Clinically relevant, active ocular inflammation or infection
Glaucoma or other significant optic neuropathy
Diabetic macular edema or diabetic retinopathy
Clinically significant cystoid macular edema
BlueRock Therapeutics
Lead sponsor