A Phase 1/2a Study of Subretinal Administration of OpCT-001 Photoreceptor Precursor Cells Derived From iPSCs in Patients With Primary Photoreceptor Disease
BlueRock Therapeutics
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. 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.
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-leve…
Eligibility
- Age range
- 18+ years
- Sex
- All
- Healthy volunteers
- No
Key Inclusion Criteria: * 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: * Clinically relevant, active ocular inflammation or infection * Glaucoma or other significant optic neuropathy * Diabetic macular edema or di…
Interventions
- BiologicalOpCT-001
Cell therapy product composed of photoreceptor precursor cells derived from human induced pluripotent stem cells (iPSCs).
- BiologicalOpCT-001
Cell therapy product composed of photoreceptor precursor cells derived from human iPSCs.
Locations (4)
- University of Miami, Bascom Palmer Eye InstituteMiami, Florida
- Mid Atlantic Retina/ Wills EyePhiladelphia, Pennsylvania
- Retina Foundation of the SouthwestDallas, Texas
- University of WisconsinMadison, Wisconsin