Prospective, Randomized, Sham-controlled, Dose-finding I/II Trial of Safety and Efficacy of Modified Optogenetic Gene Therapy (ZM-02 Injection)
brief summary
This is a Phase 1/2, multi-center, randomized, sham-controlled, dose-escalation study evaluating ZM-02 in patients with advanced retinitis pigmentosa (RP).
detailed description
Retinitis pigmentosa (RP) is the most common inherited retinal disease, characterized by progressive loss of visual function that can lead to severe visual impairment or blindness with no effective treatment.
This Phase 1/2 study is designed to evaluate the safety, tolerability, and preliminary efficacy of a single unilateral intravitreal injection of ZM-02 at escalating dose levels in patients with advanced RP. The study includes a sham-controlled design and will assess safety outcomes as well as multiple functional and structural measures of visual performance over long-term follow-up.
official title
A Prospective, Randomized, Sham-controlled, Dose-finding Phase 1/2 Two-part Trial to Evaluate the Safety, Tolerability, and Efficacy of ZM-02 Injection in Patients With Advanced Retinitis Pigmentosa