This is a Phase 1/2, multi-center, randomized, sham-controlled, dose-escalation study evaluating ZM-02 in patients with advanced retinitis pigmentosa (RP).
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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
21
Single unilateral IVT injection of low-dose ZM-02 in the study eye
Single unilateral IVT injection of high-dose ZM-02 in the study eye
Single unilateral IVT injection of selected-dose ZM-02 in the study eye
Safety and tolerability of ZM-02
Incidence of severity of ocular and systemic adverse events (AEs) and serious adverse events (SAEs) following a single intravitreal injection of ZM-02.
Time frame: Baseline to Week 52.
Change from Baseline in Best-Corrected Visual Acuity (BCVA) of Both Eyes
Time frame: Baseline to Week 52.
Change from Baseline in Functional Vision (MLMT)
Change from baseline in the multi-luminance mobility test (MLMT) performance.
Time frame: Baseline to Week 52.
Change from Baseline in Vision-Related Quality of Life (NEI VFQ-25)
Time frame: Baseline to Week 52.
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Sham IVT procedure in the study eye