NCT05858983招募中1 期
A Multi-center, Open-label, Dose-escalation Phase I/II Clinical Study to Evaluate the Safety, Tolerability and Efficacy of FT-001 Administered Via Subretinal Injection in Subjects With Biallelic RPE65 Mutation-associated Retinal Dystrophy
适应症
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 9
- 试验地点
- 1
- 主要终点
- Safety of FT-001(incidence of ocular and non-ocular AEs and SAEs)
研究概览
简要总结
The goal of this clinical trial is to evaluate the safety, tolerability and efficacy of subretinal administration of FT-001 in subjects with biallelic RPE65 mutation-associated retinal dystrophy.
详细描述
This study is a multi-center, open-label, phase I/II clinical study to evaluate the safety, tolerability, efficacy, immunogenicity, and in vivo biodistribution characteristics of FT-001 in subjects with biallelic RPE65 mutation-associated retinal dystrophy. Assessments will include visual acuity, vector shedding, immunogenicity and adverse events. Participants will be monitored for 5 years after treatment.
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
- All
- 接受健康志愿者
- 否
入选标准
- •Subjects who are able to understand and sign the ICF
- •Female or male aged 8-45 years old when signing the ICF
- •Clinically diagnosed with biallelic RPE65 mutation-associated retinal dystrophy
排除标准
- •Other interfering eye diseases
- •Presence of any systemic or ocular disease that can cause or likely to cause vision loss
- •There is evidence of obviously uncontrolled concomitant diseases
- •Known to have active or suspected autoimmune diseases
- •With active systemic infection under treatment
- •Pregnant or lactating women
- •Other conditions unsuitable for the study as determined by the investigator
结局指标
主要结局
Safety of FT-001(incidence of ocular and non-ocular AEs and SAEs)
时间窗: 52 weeks
Incidence of ocular and non-ocular AEs and SAEs
次要结局
- Changes in visual function from baseline(52 weeks)
研究者
研究点 (1)
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