NCT06789445

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

• Status: Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 54 (estimated)
• Sponsor: BlueRock Therapeutics · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

Detailed description

Phase 1 of the study includes up to 4 planned dose levels to be administered across up to 4 cohorts. Participants are assigned to receive OpCT-001 in an open-label manner. Dose escalation in Phase 1 is being conducted using a standard 3+3 scheme in which a total of up to 24 legally blind participants (\~3 to 6 per cohort) will receive OpCT-001. Phase 2 is planned to enroll a maximum of 15 participants per cohort in 2 cohorts to evaluate 2 dose levels of OpCT-001 that will be selected based on Phase 1 safety and tolerability data. Phase 2 participants will be randomized 1:1 to either dose-level cohort. Phase 2 participants and investigators and study site personnel outside of the surgical team will be masked to the Phase 2 OpCT-001 dose-level assignments.

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

Interventions

Type	Name	Description
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.

Timeline

• Start date: 2025-03-10
• Primary completion: 2029-10-01
• Completion: 2030-10-01
• First posted: 2025-01-23
• Last updated: 2026-04-13

Locations

4 sites across 1 country: United States

Regulatory

• FDA-regulated drug study