A Study to Investigate the Safety of OpCT-001 in Adults Who Have Primary Photoreceptor Disease (CLARICO)

Phase 1

Recruiting

• Conditions: Primary Photoreceptor Disease; Retinitis Pigmentosa (RP); Usher Syndrome; Cone-Rod Dystrophy; Inherited Retinal Disease (IRD); Rod-Cone Dystrophy; Rod-Cone Disease; Cone-Rod Disease; Retinal Degeneration
• Interventions: Biological: OpCT-001

• Registration Number: NCT06789445
• Lead Sponsor: BlueRock Therapeutics

Brief 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 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.

Detailed Description

Phase 1 will include 4 planned dose levels to be administered across 4 cohorts (Cohorts 1 through 4). Participants will be assigned to receive OpCT-001.

Dose escalation in Phase 1 will be conducted using a standard 3+3 scheme in which a total of 12 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 (Cohorts 5 and 6) 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 Cohort 5 or Cohort 6. Phase 2 participants and the investigator/study site personnel outside of the surgical team will be masked to OpCT-001 dose assignments.

Study Timeline

• Study First Submitted: 2025-01-17
• Study First Submitted QC: 2025-01-17
• Study First Posted: 2025-01-23
• Study Start: 2025-03-10
• Status Verified: 2025-06
• Last Update Submitted: 2025-06-18
• Last Update Posted: 2025-06-24
• Primary Completion: 2029-10
• Study Completion: 2030-10-01

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 54

Inclusion Criteria

• Genetically confirmed diagnosis of primary photoreceptor (PR) disease
• BCVA at Screening for Phase 1: 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 (SE) 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 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 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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
Cohort 1	OpCT-001	OpCT-001 dose level 1 will be administered via subretinal injection.
Cohort 4	OpCT-001	OpCT-001 dose level 4 will be administered via subretinal injection.
Cohort 3	OpCT-001	OpCT-001 dose level 3 will be administered via subretinal injection.
Cohort 2	OpCT-001	OpCT-001 dose level 2 will be administered via subretinal injection.

Primary Outcome Measures

Name	Time	Method
Incidence and severity of ocular and non-ocular treatment-emergent adverse events (TEAEs) through the Week 52 visit	From enrollment to the end of treatment at 52 weeks

Secondary Outcome Measures

Name	Time	Method
Change from baseline over time through the Week 52 visit in the treated portion of the study eye (SE) retina in outer retinal layer thickness as measured by spectral domain optical coherence tomography (SD-OCT)	From enrollment to the end of treatment at 52 weeks

Trial Locations

Locations (2)

University of Wisconsin; 🇺🇸 Madison, Wisconsin, United States

Locations: University of Miami, Bascom Palmer Eye Institute; 🇺🇸 Miami, Florida, United States