NCT07161544招募中1 期
An Open-label, Multicenter, Two Part, Ascending Dose Followed by a Controlled Trial to Assess the Safety and Efficacy of a Subretinal Administration of AAVB-039 in Participants With Stargardt Disease (STGD1) (CELESTE)
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
- 75
- 试验地点
- 12
- 主要终点
- To measure the number and severity of treatment related adverse events following treatment with AAVB-039
研究概览
简要总结
The purpose of the 039-101 study is to evaluate the safety and tolerability of a single subretinal injection of AAVB-039 in participants with Stargardt disease secondary to a biallelic mutation of the ABCA4 gene. The study will also assess initial efficacy following AAVB-039 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
- 8 Years 至 55 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Molecular diagnosis of Stargardt disease due to ABCA4 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 another Investigation Medicinal Product
- •Previous participation in another gene or cell therapy trial
- •Any condition that would preclude subretinal surgery
- •Complicating ocular and/or systemic diseases
研究组 & 干预措施
Cohort 3
Experimental
AAVB-039 dose level 3 (high dose)
干预措施: AAVB-039 (Biological)
Cohort 1
Experimental
AAVB-039 dose level 1 (low dose)
干预措施: AAVB-039 (Biological)
Cohort 2
Experimental
AAVB-039 dose level 2 (mid dose)
干预措施: AAVB-039 (Biological)
Cohorts 4-5
Experimental
AAVB-039 dose level 1, 2 or 3
干预措施: AAVB-039 (Biological)
结局指标
主要结局
To measure the number and severity of treatment related adverse events following treatment with AAVB-039
时间窗: 0-60 months
次要结局
- Change in ellipsoid zone (EZ) loss following treatment with AAVB-039(0-60 months)
- Change in the area of atrophy (assessed via short wavelength fundus autofluorescence (SW-FAF), following treatment with AAVB-039(0-60 months)
- Change in macular sensitivity assessed via microperimetry following treatment with AAVB-039(0-60 months)
- Change in visual acuity following treatment with AAVB-039(0-60 months)
研究者
研究点 (12)
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