Phase 12a Study of Subretinal OpCT-001 Cell Therapy for Adults With Primary Photoreceptor Disease

What this Trial Is About

Researchers are evaluating OpCT-001, a cell therapy made from photoreceptor precursor cells derived from human induced pluripotent stem cells iPSCs, in adults with primary photoreceptor disease. This Phase 12a multisite study aims to assess the safety, tolerability, and effects on visual function and retinal anatomy in approximately 54 participants. The study includes adults with various inherited retinal diseases such as retinitis pigmentosa and Usher syndrome. The study is divided into two parts Phase 1 involves a dose-escalation design with up to four dose levels of OpCT-001 given by subretinal injection to up to 24 legally blind participants. Phase 2 will enroll up to 15 participants per cohort in two cohorts to evaluate two selected dose levels from Phase 1. In Phase 2, participants will be randomized to one of the two doses, and masking applies to participants, investigators, and site personnel outside the surgical team. Participants will be closely monitored through Week 52 after treatment to assess safety and effects on vision and retinal structure using tests like spectral domain optical coherence tomography SD-OCT. Researchers will measure the incidence and severity of treatment-emergent adverse events and changes in retinal layer thickness. The study includes genetic confirmation of diagnosis, vision tests, retinal structure exams, and ongoing safety follow-up over approximately one year post-treatment.

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