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

April 7, 2026 updated by: BlueRock Therapeutics

A Phase 1/2a Study of Subretinal Administration of OpCT-001 Photoreceptor Precursor Cells Derived From iPSCs in Patients With Primary Photoreceptor Disease

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Estimated)

54

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • United States
    • Florida
      • Miami, Florida, United States, 33136
        • Recruiting
        • University of Miami, Bascom Palmer Eye Institute
        • Contact:
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19107
    • Texas
      • Dallas, Texas, United States, 75231
    • Wisconsin
      • Madison, Wisconsin, United States, 53705

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Sequential Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Cohort 1
OpCT-001 dose level 1 will be administered via subretinal injection.
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.
Experimental: Cohort 2
OpCT-001 dose level 2 will be administered via subretinal injection.
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.
Experimental: Cohort 4
OpCT-001 dose level 4 will be administered via subretinal injection.
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.
Experimental: Cohort 3 (optional)
OpCT-001 dose level 3 will be administered via subretinal injection.
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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Incidence and severity of ocular and non-ocular treatment-emergent adverse events (TEAEs) through the Week 52 visit
Time Frame: From OpCT-001 administration through the Week 52 visit
From OpCT-001 administration through the Week 52 visit

Secondary Outcome Measures

Outcome Measure
Time Frame
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)
Time Frame: From baseline through the Week 52 visit
From baseline through the Week 52 visit

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

BlueRock Therapeutics

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

March 10, 2025

Primary Completion (Estimated)

October 1, 2029

Study Completion (Estimated)

October 1, 2030

Study Registration Dates

First Submitted

January 17, 2025

First Submitted That Met QC Criteria

January 17, 2025

First Posted (Actual)

January 23, 2025

Study Record Updates

Last Update Posted (Actual)

April 13, 2026

Last Update Submitted That Met QC Criteria

April 7, 2026

Last Verified

April 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • OpCT-001-101

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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