- Principal Investigator: Kimberly Stepien
- Study Coordinator: Bonnie Verges (608-265-5292) and Angie Adler (608-265-7557)
- Sponsor: BlueRock Therapeutics
Brief Summary
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 up to approximately 54 adults with primary photoreceptor (PR) disease. Phase 1 will focus 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.
Detailed Description
Phase 1 will include 4 planned dose levels to be administered across 4 cohorts (Cohorts 1 through 4). Participants will be assigned to receive OpCT-001.
Dose escalation in Phase 1 will be conducted using a standard 3+3 scheme in which a total of 12 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 (Cohorts 5 and 6) 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 Cohort 5 or Cohort 6. Phase 2 participants and the investigator/study site personnel outside of the surgical team will be masked to OpCT-001 dose assignments.
Key Inclusion Criteria:
- Genetically confirmed diagnosis of primary photoreceptor (PR) disease
- BCVA at Screening for Phase 1: LogMAR 3.9 to LogMAR 1.0. BCVA at Screening for Phase 2: ETDRS letter score 20 and 60.
- Retinal structure examination in the study eye (SE) demonstrating regions suitable for cell administration.
Key Exclusion Criteria:
- History of/currently active clinically relevant, ocular inflammation or infection
- Glaucoma or other significant optic neuropathy
- Diabetic macular edema or diabetic retinopathy
- Clinically significant cystoid macular edema
- 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)
- Presence of clinically significant anti-OpCT-001 HLA antibodies at Screening
- 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 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
Contact Bonnie Verges or Angie Adler with questions at (608) 265-5292 or (608) 265-7557
For more information about this study, please visit clinicaltrials.gov