NCT06789445 · BlueRock Therapeutics
A Study to Investigate the Safety of OpCT-001 in Adults Who Have Primary Photoreceptor Disease (CLARICO)
(CLARICO)
What this study is 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 up to approximately 54 adults with primary photoreceptor (PR) disease. Phase 1 will focus on safety and features a gradually increasing doses design.
View original scientific description
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.
Interventions
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.
Primary outcome measures
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
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
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 defini
Where
- Los Angeles, California
- Miami, Florida
- Philadelphia, Pennsylvania
- Dallas, Texas
- Madison, Wisconsin
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Jul 6, 2026 · Source of record for eligibility and locations