Phase 1 Study of C.001 Given by Subretinal Injection for Retinal Diseases Caused by Retinal Pigment Epithelium Damage

What this Trial Is About

Researchers are evaluating the safety and tolerability of C.001, a biological treatment delivered by subretinal injection, in patients with retinal diseases involving degeneration of the retinal pigment epithelium such as geographic atrophy and Stargardt disease. This Phase 1, first-in-human study aims to assess safety over a three-month period and explore visual function and retinal structure changes. The study is open-label and conducted at a single center, focusing on patients with significant retinal pigment epithelium-mediated maculopathy. Participants will receive a single dose of C.001 administered via subretinal injection. The study includes three sequential dose groups low, medium, and high dose, with a sentinel dosing strategy and safety reviews by an independent Data Safety Monitoring Board before escalating doses. There is a required minimum four-week observation period between dose escalations. Follow-up lasts up to 12 months after treatment to monitor safety and study effects on vision and retinal anatomy. Throughout the study, participants will undergo safety assessments, including monitoring for ocular and systemic adverse events. Visual function will be evaluated using ETDRS letter scores, and anatomical changes will be examined with optical coherence tomography OCT. The total participation duration may extend up to 12 months, with regular evaluations to track safety and exploratory outcomes.

Phase 1 Study of C.001 in Retinal Degeneration