Actively Recruiting
A Phase 1/2a Study of Subretinal Administration of OpCT-001 Photoreceptor Precursor Cells Derived From iPSCs in Patients With Primary Photoreceptor Disease
Led by BlueRock Therapeutics · Updated on 2026-04-13
54
Participants Needed
4
Research Sites
52 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are studying OpCT-001, a cell therapy made from photoreceptor precursor cells derived from human induced pluripotent stem cells (iPSCs), in adults with primary photoreceptor disease. This Phase 1/2a study aims to evaluate the safety, tolerability, and effects of OpCT-001 on visual function, functional vision, and retinal structure in about 54 adults with confirmed genetic diagnoses of photoreceptor diseases such as retinitis pigmentosa and Usher syndrome. The study is divided into two parts. Phase 1 involves up to 24 legally blind participants receiving OpCT-001 at up to 4 different dose levels via subretinal injection, using a dose-escalation design. Phase 2 will enroll up to 30 participants randomized to two dose levels selected from Phase 1 data, with masking applied to participants and most study staff. Both phases focus on monitoring safety and assessing effects on vision and retinal anatomy. Participants will receive OpCT-001 through subretinal injections and be followed for up to 52 weeks to monitor treatment-emergent adverse events and changes in retinal layer thickness using imaging methods like spectral domain optical coherence tomography. Visual acuity and retinal structure will be assessed regularly. The study includes genetic confirmation of disease, eye exams, and ongoing safety monitoring, with the total participation duration spanning about one year after treatment.
CONDITIONS
Brief Title
A Study to Investigate the Safety of OpCT-001 in Adults Who Have Primary Photoreceptor Disease (CLARICO)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Confirmed genetic diagnosis of primary photoreceptor disease
- Best corrected visual acuity in the study eye at Screening: LogMAR 3.9 to LogMAR 1.3 for Phase 1
- Best corrected visual acuity in the study eye at Screening: ETDRS letter score between 20 to 60 for Phase 2
- Retinal structure in the study eye shows suitable regions for cell administration
You will not qualify if you...
- Active ocular inflammation or infection
- Glaucoma or significant optic neuropathy
- Diabetic macular edema or diabetic retinopathy
- Clinically significant cystoid macular edema
- More than 8.00 diopters myopia in phakic participants
- Eye surgery within 3 months before Screening
- Monocular vision with no light perception in fellow eye
- Active malignancy or history of malignancy within 5 years except treated basal cell carcinoma
- Any current active infection risking immunosuppression
- History of cell therapy, gene therapy, or retinal implant
- Previous bone marrow or solid organ transplant
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 52 weeks from administration
Participants receive OpCT-001 via subretinal injection at one of up to four dose levels depending on cohort assignment.
Multiple visits for monitoring and assessments through Week 52
Duration - Up to 52 weeks after OpCT-001 administration
Participants are monitored for safety and changes in retinal structure after treatment.
Regular visits for safety and efficacy assessments through Week 52
Trial Site Locations
Total: 4 locations
1
University of Miami, Bascom Palmer Eye Institute
Miami, Florida, United States, 33136
Actively Recruiting
2
Mid Atlantic Retina/ Wills Eye
Philadelphia, Pennsylvania, United States, 19107
Actively Recruiting
3
Retina Foundation of the Southwest
Dallas, Texas, United States, 75231
Actively Recruiting
4
University of Wisconsin
Madison, Wisconsin, United States, 53705
Actively Recruiting
Research Team
P
Patient Engagement
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
4
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