Tests OpCT-001 cell therapy safety in adults with inherited retinal disease
Tests a cell therapy injected under the retina in adults with retinitis pigmentosa.
Plain-language title generated from the registry record
Registry title: A Study to Investigate the Safety of OpCT-001 in Adults Who Have Primary Photoreceptor Disease (CLARICO)
Key facts
- Where
- Dallas, Los Angeles, Madison, Miami, and Philadelphia, United StatesSee the 5 sites
- Phase
- Phase 1/2
Where in United States
- Retina Foundation of the SouthwestDallas, United StatesTaking people
- University of Southern California /Children's Hospital of Los AngelesLos Angeles, United StatesTaking people
- University of WisconsinMadison, United StatesTaking people
- University of Miami, Bascom Palmer Eye InstituteMiami, United StatesTaking people
- Mid Atlantic Retina/ Wills EyePhiladelphia, United StatesTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- BlueRock Therapeutics
- Trial code
- NCT06789445
- Record updated
- 15 October 2026
- Original description
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.Read full original descriptionShow less
A Phase 1/2a Study of Subretinal Administration of OpCT-001 Photoreceptor Precursor Cells Derived From iPSCs in Patients With Primary Photoreceptor Disease
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.
Inclusion: 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.
Exclusion: Ocular surgery ≤3 months before Screening; Previously received a bone marrow or solid organ transplant; Clinically relevant, active ocular inflammation or infection; Currently active malignancy, or history of malignancy within 5 years before OpCT-001 administration. Exception: Basal cell carcinoma that has been definitively treated; Any current and active infection (bacterial/viral/fungal) that could put the participant at risk from immunosuppression; History of any cell therapy, gene therapy, or retinal implant at any time; Glaucoma or other significant optic neuropathy; Diabetic macular edema or diabetic retinopathy; Clinically significant cystoid macular edema; In phakic participants: Spherical equivalent refractive error of greater than 8.00 diopters myopia; Monocular vision (ie, no light perception in the fellow eye).