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
BlueRock Therapeutics
54 participants
Mar 10, 2025
INTERVENTIONAL
Conditions
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.
Eligibility
Inclusion Criteria3
- 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 Criteria11
- Clinically relevant, active ocular inflammation or infection
- 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
- Ocular surgery ≤3 months before Screening
- Monocular vision (ie, no light perception in the fellow eye)
- 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
- Previously received a bone marrow or solid organ transplant
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Interventions
Cell therapy product composed of photoreceptor precursor cells derived from human induced pluripotent stem cells (iPSCs).
Cell therapy product composed of photoreceptor precursor cells derived from human iPSCs.
Locations(4)
View Full Details on ClinicalTrials.gov
For the most up-to-date information, visit the official listing.
NCT06789445