NCT06591793招募中1 期
A Phase 1/2 Multicenter, Open-label, Dose Escalation, Safety and Efficacy Study of Subretinal Administration of Dual AAV8.MYO7A, AAVB-081 in Subjects With Usher Syndrome Type IB (USH1B) 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
- 15
- 试验地点
- 3
- 主要终点
- To measure the number and severity of treatment related adverse events following treatment with AAVB-081
研究概览
简要总结
The purpose of the 081-101 study is to evaluate the safety and tolerability of a single subretinal injection of AAVB-081 in USH1B patients with retinitis pigmentosa due to a mutation in the MYO7A gene. The study will also assess the initial efficacy following AAVB-081 administration.
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 50 Years（Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Molecular diagnosis of USB1B due to MYO7A mutation
- •Willingness to adhere to protocol per informed consent
排除标准
- •Unwillingness to meet the requirements of the study
- •Participation in a clinical study with an Investigation Product in the past 6 months
- •Previous participation in another Gene Therapy trial
- •Any condition that would preclude subretinal surgery
- •Complicating ocular and/or systemic diseases
研究组 & 干预措施
Cohort 1
Experimental
AAVB-081 dose level 1
干预措施: AAVB-081 (Biological)
Cohort 2
Experimental
AAVB-081 dose level 2
干预措施: AAVB-081 (Biological)
Cohort 3
Experimental
AAVB-081 dose level 3
干预措施: AAVB-081 (Biological)
结局指标
主要结局
To measure the number and severity of treatment related adverse events following treatment with AAVB-081
时间窗: 61 months
次要结局
- Change from baseline in microperimetry following treatment with AAVB-081(61 months)
- Change from baseline in static perimetry following treatment with AAVB-081(61 months)
研究者
研究点 (3)
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