Primary Photoreceptor DiseasePhase 1/2CLARICONCT06789445
A Study to Investigate the Safety of OpCT-001 in Adults Who Have Primary Photoreceptor Disease (CLARICO)
A Phase 1/2 study testing OpCT-001 in people with Primary Photoreceptor Disease and Retinitis Pigmentosa (RP), run by BlueRock Therapeutics at 5 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Primary Photoreceptor Disease, Retinitis Pigmentosa (RP), Usher Syndrome
- What is being tested?
- OpCT-001
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 54 participants
- Where
- 5 sitesUnited States
- Study dates
- 10 Mar 2025to Oct 2030
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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
Show all 11Show fewer
- 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 locations and contacts
5 sites in 1 country. Contact the study team or a site near you to ask about taking part.
5 sites in 1 country
United States5 sites, 5 recruiting
- Recruiting
Retina Foundation of the Southwest
Dallas, Texas, 75231
Patient Engagement, 877-380-3931, clinicaltrials@bluerocktx.com
Jennifer Herrera, 214-363-3911 ext. 135, jherrera@retinafoundation.org
- Recruiting
University of Southern California /Children's Hospital of Los Angeles
Los Angeles, California, 90007
Patient Engagement, 877-380-3931, clinicaltrials@bluerocktx.com
Hilary Schwartz-Gale, hschwartz@chla.usc.edu
- Recruiting
University of Wisconsin
Madison, Wisconsin, 53705
Patient Engagement, 877-380-3931, clinicaltrials@bluerocktx.com
- Recruiting
University of Miami, Bascom Palmer Eye Institute
Miami, Florida, 33136
Patient Engagement, 877-380-3931, clinicaltrials@bluerocktx.com
- Recruiting
Mid Atlantic Retina/ Wills Eye
Philadelphia, Pennsylvania, 19107
Patient Engagement, 877-380-3931, clinicaltrials@bluerocktx.com
Study Coordinator, 215-928-3092, alemus@midatlanticretina.com
About this study
Official title: 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.
Read the 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
- Being tested
- OpCT-001
- Keywords
- CLARICO, Photoreceptor cells, Cell Therapy, Cellular Therapy, Usher Syndrome, Retinitis Pigmentosa, Inherited retinal disease (IRD), Primary Photoreceptor disease (PPD), Rod-Con Disease (R-CD), Cone-Rose disease (C-RD)
- Medical subject headings
- Retinitis Pigmentosa, Usher Syndromes, Cone-Rod Dystrophies, Retinal Degeneration
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
OpCT-001Biological
Cell therapy product composed of photoreceptor precursor cells derived from human induced pluripotent stem cells (iPSCs).
OpCT-001Biological
Cell therapy product composed of photoreceptor precursor cells derived from human iPSCs.
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 | Experimental | OpCT-001 dose level 1 will be administered via subretinal injection. Receives: Biological: OpCT-001 |
| Cohort 2 | Experimental | OpCT-001 dose level 2 will be administered via subretinal injection. Receives: Biological: OpCT-001 |
| Cohort 3 (optional) | Experimental | OpCT-001 dose level 3 will be administered via subretinal injection. Receives: Biological: OpCT-001 |
| Cohort 4 | Experimental | OpCT-001 dose level 4 will be administered via subretinal injection. Receives: Biological: OpCT-001 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence and severity of ocular and non-ocular treatment-emergent adverse events (TEAEs) through the Week 52 visit
Measured: From OpCT-001 administration through the Week 52 visit
Secondary outcomes (1)
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)
Measured: From baseline through the Week 52 visit
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Jan 2025
- Study start10 Mar 2025
- Primary completionOct 2029 (estimated)
- Study completionOct 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Primary Photoreceptor DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Model description
- 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.
- Masking
- None (open label)
- Enrolment
- 54 participants (estimated)
Sponsor and investigators
- Lead sponsor
- BlueRock Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Bayer
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06789445
- Sponsor's protocol ID
- OpCT-001-101
- Acronym
- CLARICO
- Status last verified
- Oct 2026
- First submitted
- 17 Jan 2025
- Last updated
- 15 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1/2a Study of Subretinal Administration of OpCT-001 Photoreceptor Precursor Cells Derived From iPSCs in Patients With Primary Photoreceptor Disease
- ClinicalTrials.gov ID
- NCT06789445
- Acronym / sponsor ID
- CLARICO / OpCT-001-101
- Conditions
- Primary Photoreceptor Disease, Retinitis Pigmentosa (RP), Usher Syndrome, Inherited Retinal Disease (IRD), Rod-Cone Dystrophy, Rod-Cone Disease
- Keywords
- CLARICO, Photoreceptor cells, Cell Therapy, Cellular Therapy, Usher Syndrome, Retinitis Pigmentosa, Inherited retinal disease (IRD), Primary Photoreceptor disease (PPD)
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 54 participants (estimated)
- Lead sponsor
- BlueRock TherapeuticsIndustry (pharma, biotech, devices)
- Collaborators
- Bayer
- Study start
- 10 Mar 2025 (actual)
- Primary completion
- Oct 2029 (estimated)
- Study completion
- Oct 2030 (estimated)
- First posted
- 23 Jan 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 4
- Model description
- 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.
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 | Experimental | Biological: OpCT-001OpCT-001 dose level 1 will be administered via subretinal injection. |
| Cohort 2 | Experimental | Biological: OpCT-001OpCT-001 dose level 2 will be administered via subretinal injection. |
| Cohort 3 (optional) | Experimental | Biological: OpCT-001OpCT-001 dose level 3 will be administered via subretinal injection. |
| Cohort 4 | Experimental | Biological: OpCT-001OpCT-001 dose level 4 will be administered via subretinal injection. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- 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 (11 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| OpCT-001Cell therapy product composed of photoreceptor precursor cells derived from human induced pluripotent stem cells (iPSCs). | Biological | Cohort 1 |
| OpCT-001Cell therapy product composed of photoreceptor precursor cells derived from human iPSCs. | Biological | Cohort 2, Cohort 3 (optional), Cohort 4 |
Primary outcome
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
1 secondary outcome
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Oct 2026.
- Related InfoLink provided by the sponsor.
Related trials for Primary Photoreceptor Disease
Recruiting
Retinal Photoreceptor Outer Segment Length Before and After Light Stimulation - a Pilot Study
Macular Degeneration, Dry, Primary Open-angle Glaucoma
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing OpCT-001 in people with Primary Photoreceptor Disease and Retinitis Pigmentosa (RP), run by BlueRock Therapeutics at 5 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 15 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Patient Engagement (1-877-380-3931, clinicaltrials@bluerocktx.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Oct 2026.