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

Launched by BLUEROCK THERAPEUTICS · Jan 17, 2025

Keywords

ClinConnect Summary

The CLARICO trial is a research study that is testing a new treatment called OpCT-001 for adults with specific eye conditions known as primary photoreceptor diseases, which include diseases like Retinitis Pigmentosa and Usher Syndrome. The main goal of the study is to find out if OpCT-001 is safe to use and how it might improve vision for people with these conditions. The trial has two parts: the first part focuses on safety by gradually increasing the dose of OpCT-001, while the second part will collect more safety information and look at how the treatment affects vision and the structure of the eye.

To participate in this study, you need to be an adult aged 18 or older with a confirmed genetic diagnosis of a primary photoreceptor disease. You'll also need to meet certain vision requirements and have specific conditions in your eye that are suitable for treatment. During the study, participants can expect regular check-ups to monitor their health and vision. It's important to note that certain health issues, such as active eye infections or significant eye diseases, will exclude you from joining the study. If you or a loved one is considering participating, this trial could be an opportunity to contribute to research that aims to improve treatments for these eye diseases.

Gender

ALL

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