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

NCT ID: NCT06789445

Last Updated: 2025-12-26

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE1/PHASE2

Total Enrollment

54 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-03-10

Study Completion Date

2030-10-31

Brief Summary

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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

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Phase 1 of the study will include up to 4 planned dose levels to be administered across up to 4 cohorts. 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 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 cohort. Phase 2 participants and the investigator/study site personnel outside of the surgical team will be masked to OpCT-001 dose assignments.

Conditions

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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

Keywords

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Primary Photoreceptor disease CLARICO Photoreceptor cells Inherited retinal disease Cell Therapy Cellular Therapy Usher Syndrome Cone-Rod Disease Rod-Cone Disease Retinitis Pigmentosa

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SEQUENTIAL

Phase 1 of the study will include up to 4 planned dose levels to be administered across up to 4 cohorts. 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 the Phase 1 safety and tolerability data.
Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Phase 1: None (Open Label) Phase 2: Investigator/study site personnel outside of the surgical team will be masked to dose level assignments

Study Groups

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Cohort 3

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

Group Type EXPERIMENTAL

OpCT-001

Intervention Type BIOLOGICAL

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

Cohort 4

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

Group Type EXPERIMENTAL

OpCT-001

Intervention Type BIOLOGICAL

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

Cohort 1

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

Group Type EXPERIMENTAL

OpCT-001

Intervention Type BIOLOGICAL

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

Cohort 2

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

Group Type EXPERIMENTAL

OpCT-001

Intervention Type BIOLOGICAL

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

Interventions

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OpCT-001

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

Intervention Type BIOLOGICAL

OpCT-001

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

Intervention Type BIOLOGICAL

Eligibility Criteria

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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.

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
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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BlueRock Therapeutics

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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University of Miami, Bascom Palmer Eye Institute

Miami, Florida, United States

Site Status RECRUITING

Retina Foundation of the Southwest

Dallas, Texas, United States

Site Status RECRUITING

University of Wisconsin

Madison, Wisconsin, United States

Site Status RECRUITING

Countries

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United States

Central Contacts

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Patient Engagement

Role: CONTACT

Phone: 1-877-380-3931

Email: [email protected]

Facility Contacts

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Patient Engagement

Role: primary

Patient Engagement

Role: primary

Patient Engagement

Role: primary

Related Links

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Other Identifiers

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OpCT-001-101

Identifier Type: -

Identifier Source: org_study_id