OpCT-001: Cell therapy product composed of photoreceptor precursor cells derived from human induced pluripotent stem cells (iPSCs).
OpCT-001: Cell therapy product composed of photoreceptor precursor cells derived from human iPSCs.
Study 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.
Eligibility
Sex
ALL
Min age
18 Years
Max age
—
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 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
Primary outcome measure(s)
Incidence and severity of ocular and non-ocular treatment-emergent adverse events (TEAEs) through the Week 52 visit — From OpCT-001 administration through the Week 52 visit
Trial sites (5)
Facility
City
Region
Status
University of Southern California /Children's Hospital of Los Angeles
Los Angeles
California
Recruiting
University of Miami, Bascom Palmer Eye Institute
Miami
Florida
Recruiting
Mid Atlantic Retina/ Wills Eye
Philadelphia
Pennsylvania
Recruiting
Retina Foundation of the Southwest
Dallas
Texas
Recruiting
University of Wisconsin
Madison
Wisconsin
Recruiting
Official registry record
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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