Ophthalmology and Visual Sciences

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A Phase 1/2a Study of Subretinal Administration of OpCT-001 Photoreceptor Precursor Cells Derived From iPSCs in Patients with Primary Photoreceptor Disease

  • Sponsor: BlueRock Therapeutics
  • Principal Investigator: Kimberly Stepien, MD
  • Study Coordinators: Nickie Stangel; Matt Gilles

Study Objective:

An 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.

Study Design:

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.

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 1.0. BCVA at Screening for Phase 2: ETDRS letter score 20 and 60.
  • Retinal structure examination in the study eye demonstrating regions suitable for cell administration.

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 (i.e., no light perception in the fellow eye)
  • Presence of clinically significant anti-OpCT-001 Human Leukocyte Antigen (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 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

Contact Nickie Stangel at (608) 263-8783 or Matt Gilles at (608) 890-2211
For more information about this study, please visit clinicaltrials.gov