Clinical trial NCT06789445: A Study to Investigate the Safety of OpCT-001 in Adults Who Have Primary Photoreceptor Disease (CLARICO)
Official title: A Phase 1/2a Study of Subretinal Administration of OpCT-001 Photoreceptor Precursor Cells Derived from IPSCs in Patients with Primary Photoreceptor Disease
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE1
PHASE2
Conditions: Primary Photoreceptor Disease
Retinitis Pigmentosa (RP)
Usher Syndrome
Cone-Rod Dystrophy
Inherited Retinal Disease (IRD)
Rod-Cone Dystrophy
Rod-Cone Disease
Cone-Rod Disease
Retinal Degeneration
Keywords: Primary Photoreceptor disease
CLARICO
Photoreceptor cells
Inherited retinal disease
Cell Therapy
Cellular Therapy
Usher Syndrome
Cone-Rod Disease
Rod-Cone Disease
Retinitis Pigmentosa
Lead sponsor: BlueRock Therapeutics
Sponsor class: INDUSTRY
First posted: 2025-01-23
Enrollment: 54
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SEQUENTIAL
Intervention model description: Phase 1 of the study will include 4 planned dose levels to be administered across 4 cohorts (Cohorts 1 through 4). 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 the Phase 1 safety and tolerability data.
Primary purpose: TREATMENT

Brief 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 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.

Eligibility criteria: 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

Primary outcomes:
measure: Incidence and severity of ocular and non-ocular treatment-emergent adverse events (TEAEs) through the Week 52 visit
timeFrame: From enrollment to the end of treatment at 52 weeks
Secondary outcomes:
measure: 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)
timeFrame: From enrollment to the end of treatment at 52 weeks
Other outcomes:
Locations:
city: Miami
country: United States
facility: Locations: University of Miami, Bascom Palmer Eye Institute
state: Florida
Study officials:
References: