ACDN-01-001: Open-Label, Single Ascending Dose Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of Subretinal ACDN-01 in Participants With ABCA4-related Retinopathy
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 15
- 试验地点
- 10
- 主要终点
- Safety and tolerability of ACDN-01 as measured by the number and severity of adverse events and serious adverse events.
研究概览
简要总结
This study is an open-label, single ascending dose clinical trial in participants who have ABCA4-related retinopathies. This is the first-in-human clinical trial in which ACDN-01 will be evaluated for safety, tolerability, and preliminary efficacy following a single subretinal injection of ACDN-01.
详细描述
This is an open-label, single ascending dose study of ACDN-01 in adult participants with ABCA4-related retinopathy. The study is designed to evaluate the safety, tolerability, and evidence of biological effect of SAD levels (low, medium, and high) of ACDN-01 when delivered subretinally.
Participants will be followed on study for 2 years for the primary safety and preliminary efficacy endpoints, after which they will continue in the study in a 3-year long-term follow-up period, for a total study duration of 5 years.
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
- 12 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Presence of mutations in the ABCA4 gene
- •ABCA4 retinopathy phenotype (Stargardt disease type 1 or cone-rod dystrophy)
- •Area of atrophy located in the macula of the study eye
- •BCVA of 20/50 (0.4 logMAR) or worse
排除标准
- •The presence of pathogenic or likely pathogenic mutations in other genes known to cause cone-rod dystrophy or Stargardt maculopathy
- •Retinal disease other than ABCA4-related retinopathy
- •Presence of a medical condition (systemic or ophthalmic), psychiatric condition, including substance abuse disorder, or physical examination or laboratory finding that may in the opinion of the principal investigator and sponsor preclude adherence to the scheduled study visits, safe participation in the study, or affect the results of the study.
研究组 & 干预措施
Low dose of ACDN-01
One time low dose of ACDN-01.
干预措施: ACDN-01 (Drug)
Mid-dose of ACDN-01
One time mid-dose of ACDN-01.
干预措施: ACDN-01 (Drug)
High dose of ACDN-01
One time high dose of ACDN-01.
干预措施: ACDN-01 (Drug)
结局指标
主要结局
Safety and tolerability of ACDN-01 as measured by the number and severity of adverse events and serious adverse events.
时间窗: 12 months
To evaluate the safety and tolerability of a single dose of ACDN-01 when administered to participants with ABCA4-related retinopathy.
次要结局
- Maximum tolerated dose for subsequent clinical evaluation will be determined by review of all available safety and tolerability data.(12 months)
- To evaluate for evidence of preliminary efficacy based on fundus autofluorescence (FAF).(24 months)
- To evaluate for evidence of preliminary efficacy based on optical coherence tomography (OCT).(24 months)