NCT05874310招募中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
干预措施
试验速览
- 阶段
- 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.
研究组 & 干预措施
Intermediate dose FT-002
Experimental
Intraocular injection of a single Intermediate dose of FT-002
干预措施: FT-002 (Genetic)
Low dose FT-002
Experimental
Intraocular injection of a single low dose of FT-002
干预措施: FT-002 (Genetic)
High dose FT-002
Experimental
Intraocular injection of a single High dose of FT-002
干预措施: FT-002 (Genetic)
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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