A Phase 1/2 interventional study of OpCT-001 and OpCT-001 in Primary Photoreceptor Disease, Retinitis Pigmentosa (RP) and Usher Syndrome, sponsored by BlueRock Therapeutics. Recruiting at 5 sites in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-10-15.
Sponsored by BlueRock Therapeutics (part of Bayer) · Phase 1/2, Interventional, and Treatment
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.
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.
33 studies on the registry are indexed under Usher Syndromes; 13 are open to participants now.
This study's planned enrollment of 54 is above the median of 39 across 18 interventional studies indexed under Usher Syndromes.
Browse Usher Syndromes studies →BlueRock Therapeutics is the lead sponsor of 5 studies on the registry; 3 are open to participants now.
First submitted after BlueRock Therapeutics became part of Bayer.
Compared within Usher Syndromes, this study's primary condition as ClinicalTrials.gg classifies it; the registry's first condition term is Retinitis Pigmentosa. The counts are of registry records indexed under each term, refreshed daily. How we classify →
Key Inclusion Criteria:
Key Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Immunosuppression
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
OpCT-001 dose level 1 will be administered via subretinal injection.
Biological: OpCT-001
OpCT-001 dose level 2 will be administered via subretinal injection.
Biological: OpCT-001
OpCT-001 dose level 3 will be administered via subretinal injection.
Biological: OpCT-001
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).
Cell therapy product composed of photoreceptor precursor cells derived from human iPSCs.
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
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 the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
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