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RECRUITINGPhase 1 / Phase 2INTERVENTIONAL

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

Important: This information is not medical advice. Talk to your doctor about whether a clinical trial is right for you.

About This Trial

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 up to approximately 54 adults with primary photoreceptor (PR) disease. Phase 1 will focus 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.

Who May Be Eligible (Plain English)

Key Who May Qualify: - 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 1.0. BCVA at Screening for Phase 2: ETDRS letter score 20 and 60. - Retinal structure examination in the study eye demonstrating regions suitable for cell administration. Key Who Should NOT Join This Trial: - History of/currently active clinically relevant, ocular inflammation or infection - Glaucoma or other significant optic neuropathy - Diabetic macular edema or diabetic retinopathy - Clinically significant cystoid macular edema - 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) - Presence of clinically significant anti-OpCT-001 Human Leukocyte Antigen (HLA) antibodies at Screening - 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 Always talk to your doctor about whether this trial is right for you.

Original Eligibility Criteria

View original clinical language
Key Inclusion Criteria: * 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 1.0. BCVA at Screening for Phase 2: ETDRS letter score 20 and 60. * Retinal structure examination in the study eye demonstrating regions suitable for cell administration. Key Exclusion Criteria: * History of/currently active clinically relevant, ocular inflammation or infection * Glaucoma or other significant optic neuropathy * Diabetic macular edema or diabetic retinopathy * Clinically significant cystoid macular edema * 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) * Presence of clinically significant anti-OpCT-001 Human Leukocyte Antigen (HLA) antibodies at Screening * 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

Treatments Being Tested

BIOLOGICAL

OpCT-001

Cell therapy product composed of photoreceptor precursor cells derived from human induced pluripotent stem cells (iPSCs).

BIOLOGICAL

OpCT-001

Cell therapy product composed of photoreceptor precursor cells derived from human iPSCs.

Locations (3)

University of Miami, Bascom Palmer Eye Institute
Miami, Florida, United States
Retina Foundation of the Southwest
Dallas, Texas, United States
University of Wisconsin
Madison, Wisconsin, United States