A Phase 2/3 Multicenter, Randomized, Double-masked, Parallel-group, Placebo-controlled Study to Investigate the Safety, Pharmacokinetics, Tolerability, and Efficacy of ALK-001 in Geographic Atrophy Secondary to Age-related Macular Degeneration
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 200
- 试验地点
- 3
- 主要终点
- Growth Rate of GA Lesions, as Assessed by Fundus Autofluorescence (FAF)
研究概览
简要总结
This is a double-masked, multicenter, randomized, placebo-controlled clinical trial, evaluating the efficacy and safety of ALK-001 in participants with Geographic Atrophy (GA) secondary to age-related macular degeneration (AMD).
Up to 200 participants will receive ALK-001 while up to 100 participants will receive a placebo.
详细描述
There is no oral treatment available for Geographic Atrophy secondary to AMD. AMD is characterized by an age-related degeneration of the retina.
The root cause for this degeneration or why some people develop AMD while others do not, is unknown. Over 20 years ago, it was hypothesized that the dimerization of vitamin A may be a significant contributor to the etiology of AMD. The eye indeed uses vitamin A as a cofactor to sense light, and a striking chemical signature of the aging and degenerating retina is the accumulation of vitamin A dimers in the retinal pigment epithelium (RPE) and the underlying Bruch's membrane. In rodent models, high levels of vitamin A dimers correlate with poor retinal health, and a variety of mechanisms have been proposed by which vitamin A dimers may induce retinal toxicity. It has been argued that these mechanisms participate in the development and progression of AMD.
ALK-001, the study drug, is a modified form of vitamin A. When taken once a day as a capsule, it replaces natural vitamin A in the body with one that forms vitamin A dimers more slowly. This study will measure the extent to which treatment with ALK-001 slows the progression of Geographic Atrophy.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 60 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •- At least one eye with geographic atrophy secondary to dry age-related macular degeneration (AMD)
排除标准
- •- Medical condition, which may interfere with the progression of GA, prevent performance of study procedures, compliance with protocol, or continuous participation in the study
研究组 & 干预措施
ALK-001
Capsule
干预措施: ALK-001 oral capsule (Drug)
Placebo
Capsule
干预措施: Placebo oral capsule (Drug)
结局指标
主要结局
Growth Rate of GA Lesions, as Assessed by Fundus Autofluorescence (FAF)
时间窗: Baseline to 24 months
次要结局
- Number of Participants With Adverse Events(Baseline to 24 months)
- Changes in Visual Acuity(Baseline to 24 months)