NCT07282457

Prospective, Randomized, Sham-controlled, Dose-finding I/II Trial of Safety and Efficacy of Modified Optogenetic Gene Therapy (ZM-02 Injection)

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

• Status: Not Yet Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 21 (estimated)
• Sponsor: Zhongmou Therapeutics · Industry
• Sex: All
• Age: 6 Years – 60 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Retinitis Pigmentosa (RP)

Interventions

Type	Name	Description
GENETIC	ZM-02 (low dose)	Single unilateral IVT injection of low-dose ZM-02 in the study eye
GENETIC	ZM-02 (high dose)	Single unilateral IVT injection of high-dose ZM-02 in the study eye
GENETIC	ZM-02 (selected dose)	Single unilateral IVT injection of selected-dose ZM-02 in the study eye
PROCEDURE	Sham injection	Sham IVT procedure in the study eye

Timeline

• Start date: 2026-06-25
• Primary completion: 2029-12-25
• Completion: 2031-12-25
• First posted: 2025-12-15
• Last updated: 2026-04-13