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

BlueRock Therapeutics

Status and phase: Enrolling; Phase 2; Phase 1

Conditions: Inherited Retinal Disease (IRD); Rod-Cone Disease; Retinal Degeneration; Cone-Rod Disease (C-RD); Rod-Cone Dystrophy; Cone-Rod Dystrophy; Usher Syndrome; Primary Photoreceptor Disease; Retinitis Pigmentosa (RP)

Treatments: Biological: OpCT-001

Study type: Interventional

Funder types: Industry

Identifiers

NCT06789445

OpCT-001-101

Details and patient eligibility

About

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.

Full 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.

Enrollment: 54 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

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 1.3. 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.

Key Exclusion Criteria:

• 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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Sequential Assignment

Masking: None (Open label)

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54 participants in 4 patient groups

Cohort 1

Experimental group

Description:

OpCT-001 dose level 1 will be administered via subretinal injection.

Treatment:

Biological: OpCT-001

Biological: OpCT-001

Cohort 2

Experimental group

Description:

OpCT-001 dose level 2 will be administered via subretinal injection.

Treatment:

Biological: OpCT-001

Biological: OpCT-001

Cohort 3 (optional)

Experimental group

Description:

OpCT-001 dose level 3 will be administered via subretinal injection.

Treatment:

Biological: OpCT-001

Biological: OpCT-001

Cohort 4

Experimental group

Description:

OpCT-001 dose level 4 will be administered via subretinal injection.

Treatment:

Biological: OpCT-001

Biological: OpCT-001