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.
Age
18–any
Sex
ALL
Healthy volunteers
Not accepted
You may be eligible if
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.
You may not be if
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
Clareo Health | A Study to Investigate the Safety of OpCT-001 in Adults Who Have Primary Photoreceptor Disease (CLARICO)
