NCT06492850招募中1 期
A Phase I/II Dose-escalation and Dose-expansion Study to Evaluate the Safety and Efficacy of FT-002 Subretinal Injection in Subjects With RPGR Gene Mutation-associated X-linked Retinitis Pigmentosa.
适应症
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 32
- 试验地点
- 1
- 主要终点
- To evaluate the safety and tolerance of FT-002
研究概览
简要总结
The aim of this study was to evaluate the safety, tolerability, and efficacy of one-time subretinal injection of FT-002 in male subjects (8-45 years of age) with RPGR (Retinitis Pigmentosa GTPase Regulator) gene mutation-associated X-linked retinitis pigmentosa, of XLRP. This study includes Phase I (dose escalation phase) and Phase II (dose expansion phase).
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 8 Years 至 45 Years（Child, Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Subjects that are willing and able to follow study procedures including scheduled visits, treatment plan, and laboratory tests, and sign a written informed consent form;
- •Age: Phase I dose escalation stage, 18-45 years old male (including boundary value) at the time of signing the ICF; Phase II dose extension stage, males 8-45 years old (including boundary values) at the time of signing the ICF;
- •Clinically diagnosed XLRP, the main symptoms include but are not limited to night blindness, visual field loss, vision loss, etc.;
排除标准
- •Have other retinal degenerative diseases, such as retinal degeneration caused by other known Inherited retinal disease gene variants or previously received an gene therapy product.
结局指标
主要结局
To evaluate the safety and tolerance of FT-002
时间窗: from FT-002 administration through up to 1 years
Incidence and severity of AEs
次要结局
- To evaluate the efficacy of FT-002(from FT-002 administration through up to 1 years)
研究者
研究点 (1)
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