NCT04239625进行中（未招募）2 期
A Phase 2 Multicenter, Double-Masked, Randomized, Placebo-Controlled Study to Investigate the Long Term Safety, Tolerability, Pharmacokinetics and Effects of ALK-001 on the Progression of Stargardt Disease
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 入组人数
- 200
- 试验地点
- 1
- 主要终点
- Pharmacokinetic profile of ALK-001 derived from the concentrations of ALK-001 and metabolites in plasma
研究概览
简要总结
The purpose of this open-label, multicenter study is to determine the long-term safety, pharmacokinetics and effects of ALK-001 (C20-D3-retinyl acetate) on the progression of Stargardt disease. This study is an extension of NCT02402660 and enrolls participants who are at least 8 years old. Enrollment is by invitation only.
Funding Source - FDA OOPD
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 8 Years 至 70 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Clinical diagnosis of Stargardt disease (STGD1)
- •Has at least two ABCA4 disease-causing mutations, unless authorized by sponsor
- •Has a best-corrected visual acuity (BCVA) greater than approximately 20/160 in at least one eye
- •Healthy as judged by investigator
- •Able and willing to comply with study requirements, restrictions and instructions and is likely to complete the 24-month study
- •Has been invited to participate in this extension, and has signed and dated the informed consent forms (or assent where appropriate) to participate
- •Female of childbearing potential has signed the attestation on contraception requirements
排除标准
- •Is lactating or pregnant
- •Has a medical condition likely to prevent compliance with the protocol and/or interfere with absorption of ALK-001 or performance of study procedures
- •Has abnormal laboratory result(s) at screening
- •Has an ocular disorder that may confound ocular assessments
- •Has a history of ocular intervention within 90 days of screening
研究组 & 干预措施
ALK-001
Experimental
干预措施: ALK-001 (Drug)
结局指标
主要结局
Pharmacokinetic profile of ALK-001 derived from the concentrations of ALK-001 and metabolites in plasma
时间窗: Up to 24 months
Safety and tolerability of ALK-001 assessed by incidence and/or clinically-significant changes of a combination of ocular and non-ocular adverse events
时间窗: From baseline to 24 months
次要结局
未报告次要终点
研究者
研究点 (1)
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