Dysautonomia International

A Study to Investigate the Safety of OpCT-001 in Adults Who Have Primary Photoreceptor Disease (CLARICO)

Purpose

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.

Conditions

Primary Photoreceptor Disease
Retinitis Pigmentosa (RP)
Usher Syndrome
Inherited Retinal Disease (IRD)
Rod-Cone Dystrophy
Rod-Cone Disease
Retinal Degeneration
Cone-Rod Disease (C-RD)
Cone-Rod Dystrophy

Eligibility

Eligible Ages: Over 18 Years
Eligible Sex: All
Accepts Healthy Volunteers: No

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.

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

Study Design

Phase: Phase 1/Phase 2
Study Type: Interventional
Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Intervention Model Description: Phase 1 of the study will include up to 4 planned dose levels to be administered across up to 4 cohorts. 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 the Phase 1 safety and tolerability data.
Primary Purpose: Treatment
Masking: None (Open Label)
Masking Description: Phase 1: None (Open Label) Phase 2: Investigators and study site personnel outside of the surgical team will be masked to dose level assignments

Arm Groups

Arm	Description	Assigned Intervention
Experimental Cohort 1	OpCT-001 dose level 1 will be administered via subretinal injection.	Biological: OpCT-001 Cell therapy product composed of photoreceptor precursor cells derived from human induced pluripotent stem cells (iPSCs).
Experimental Cohort 2	OpCT-001 dose level 2 will be administered via subretinal injection.	Biological: OpCT-001 Cell therapy product composed of photoreceptor precursor cells derived from human iPSCs.
Experimental Cohort 3 (optional)	OpCT-001 dose level 3 will be administered via subretinal injection.	Biological: OpCT-001 Cell therapy product composed of photoreceptor precursor cells derived from human iPSCs.
Experimental Cohort 4	OpCT-001 dose level 4 will be administered via subretinal injection.	Biological: OpCT-001 Cell therapy product composed of photoreceptor precursor cells derived from human iPSCs.

Recruiting Locations

University of Southern California /Children's Hospital of Los Angeles
Los Angeles, California 90007