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
试验速览
- 阶段
- 1 期
- 状态
- 尚未招募
- 发起方
- 入组人数
- 21
- 主要终点
- Safety and tolerability of ZM-02
研究概览
简要总结
This is a Phase 1/2, multi-center, randomized, sham-controlled, dose-escalation study evaluating ZM-02 in patients with advanced retinitis pigmentosa (RP).
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 6 Years 至 60 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female participants aged 6 to 60 years.
- •Clinical diagnosis of advanced retinitis pigmentosa (RP).
- •Best corrected visual acuity at or below the protocol-defined threshold in the study eye.
- •Presence of sufficient retinal structure in the study eye as determined by screening assessments.
- •Ability to understand and sign informed consent (and assent, when applicable).
排除标准
- •Presence of other ocular conditions that could interfere with study assessments.
- •History of significant ocular surgery in the study eye within a protocol-defined period.
- •Active ocular infection or inflammation.
- •Clinically significant systemic disease that could increase study risk or interfere with assessments.
- •Positive screening for clinically significant infectious diseases, as defined in the protocol.
- •Pregnant or breastfeeding individuals.
- •Any condition that, in the investigator's judgment, would make the participant unsuitable for study participation.
研究组 & 干预措施
High Dose ZM-02
干预措施: ZM-02 (high dose) (Genetic)
Low Dose ZM-02
干预措施: ZM-02 (low dose) (Genetic)
ZM-02 (selected dose)
干预措施: ZM-02 (selected dose) (Genetic)
Sham Comparator
干预措施: Sham injection (Procedure)
结局指标
主要结局
Safety and tolerability of ZM-02
时间窗: Baseline to Week 52.
Incidence of severity of ocular and systemic adverse events (AEs) and serious adverse events (SAEs) following a single intravitreal injection of ZM-02.
次要结局
- Change from Baseline in Best-Corrected Visual Acuity (BCVA) of Both Eyes(Baseline to Week 52.)
- Change from Baseline in Functional Vision (MLMT)(Baseline to Week 52.)
- Change from Baseline in Vision-Related Quality of Life (NEI VFQ-25)(Baseline to Week 52.)