A Phase 1/2a Study to Evaluate the Safety and Effects of Subretinal OpCT-001 Photoreceptor Precursor Cells Derived From iPSCs in Adults With Primary Photoreceptor Disease

What this Trial Is About

Researchers are evaluating the safety, tolerability, and clinical effects of OpCT-001, a cell therapy composed of photoreceptor precursor cells derived from human induced pluripotent stem cells (iPSCs), in adults with primary photoreceptor disease. This Phase 1/2a multisite study includes adults with conditions such as retinitis pigmentosa, Usher syndrome, inherited retinal disease, and various forms of retinal degeneration. The study aims to assess both safety and the impact on visual function and retinal structure in different clinical subgroups. Phase 1 involves up to four dose levels of OpCT-001 administered subretinally to approximately 24 legally blind participants using a dose-escalation design with cohorts of 3 to 6 participants each. Phase 2 plans to enroll up to 30 participants divided into two cohorts receiving two selected dose levels from Phase 1 data, with participants randomized 1:1 and masked to their dose assignments except for the surgical team. The study evaluates OpCT-001's safety and effects over these two parts. Participants will be closely monitored through Week 52 for the occurrence and severity of ocular and non-ocular treatment-emergent adverse events. Assessments include visual acuity testing, retinal imaging to evaluate retinal structure and cell engraftment, and genetic confirmation of diagnosis. Safety and tolerability data will guide dose selection, while visual and anatomical outcomes will help determine the therapy's potential impact. The total participation duration covers screening, treatment, and follow-up visits through one year post-treatment.

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