NCT04278131招募中1 期
Phase 1/2, Safety and Efficacy Trial of BS01, a Recombinant Adeno-Associated Virus Vector Expressing ChronosFP in Patients With Retinitis Pigmentosa
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 20
- 试验地点
- 1
- 主要终点
- Primary Outcome Measure
研究概览
简要总结
A Phase 1/2, Safety and Efficacy Trial of BS01, a Recombinant Adeno-Associated Virus Vector Expressing ChronosFP (AAV2-CAG-ChronosFP) in Patients with Retinitis Pigmentosa
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
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Confirmed diagnosis of retinitis pigmentosa
- •At least one eye with vision no better than 20/160 visual acuity on the EDTRS scale or, for patients with peripheral vision loss, a visual field no greater than 30°.
排除标准
- •Prior receipt of any AAV gene therapy product
- •Large amplitude nystagmus
研究组 & 干预措施
Cohort 1
Experimental
BSO1 Cohort 1 dose
干预措施: BS01 (Drug)
Cohort 2
Experimental
BS01 Cohort 2 dose
干预措施: BS01 (Drug)
Cohort 3
Experimental
BS01 Cohort 3 dose
干预措施: BS01 (Drug)
Cohort 4
Experimental
BS01 Cohort4 dose
干预措施: BS01 (Drug)
Cohort 5
Experimental
BS01 Cohort4 dose
干预措施: BS01 (Drug)
Cohort 6
Experimental
Treat opposite eye
干预措施: BS01 (Drug)
结局指标
主要结局
Primary Outcome Measure
时间窗: 12 months
Number of subjects with adverse events, changes in hematology/chemistry
次要结局
- Secondary Outcome Measures(12 months)
研究者
研究点 (1)
Loading locations...
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