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.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
54
University of Miami, Bascom Palmer Eye Institute
Miami, Florida, United States
RECRUITINGMid Atlantic Retina/ Wills Eye
Philadelphia, Pennsylvania, United States
RECRUITINGRetina Foundation of the Southwest
Dallas, Texas, United States
RECRUITINGUniversity of Wisconsin
Madison, Wisconsin, United States
RECRUITINGIncidence 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
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