NCT05874310招募中早期 1 期
An Open-label, Single-center, Dose-escalation Clinical Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of Intraocular Administration of FT-002 in Subjects With RPGR Mutation-associated X-linked Retinitis Pigmentosa.
适应症
试验速览
- 阶段
- 早期 1 期
- 状态
- 招募中
- 入组人数
- 18
- 试验地点
- 2
- 主要终点
- Incidence of AEs
研究概览
简要总结
A clinical trial of gene therapy for patients with X-linked retinitis pigmentosa (XLRP).
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
- •1.Subjects that are willing and able to follow study procedures; 2.Males aged 8-45 years old at the time of signing the Informed Consent Form; 4.Subjects who are confirmed with variants of RPGR ;
排除标准
- •1.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
Incidence of AEs
时间窗: from FT-002 administration through up to 1 years
Number and proportion of Adverse Events
次要结局
- Change in retinal structure as assessed by Optical Coherence Tomography(from FT-002 administration through up to 1 years)
- Change in visual function(from FT-002 administration through up to 1 years)
研究者
研究点 (2)
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