NCT05231785招募中1 期
A Randomized, Double-blind, Placebo-controlled Single Ascending Dose and Open-label Multi-dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ALN-APP in Adult Patients With Early-onset Alzheimer's Disease (EOAD)
Alnylam Pharmaceuticals8 个研究点 分布在 4 个国家目标入组 60 人开始时间: 2022年2月4日最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 发起方
- 入组人数
- 60
- 试验地点
- 8
- 主要终点
- Part B: Frequency of Adverse Events
研究概览
简要总结
The purpose of this study is to evaluate the safety, tolerability, pharmacodynamics (PD) and pharmacokinetics (PK) of a single dose and multiple doses of ALN-APP administered by intrathecal (IT) injection in adult patients with early-onset Alzheimer's Disease (EOAD). Maximum treatment duration for Part A: single dose. Maximum treatment duration for Part B: 12 months.
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
- Triple (Participant, Care Provider, Investigator)
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Has mild cognitive impairment or mild dementia due to EOAD
- •Has Clinical Dementia Rating (CDR) global score 0.5 or 1.0 and Mini Mental State Examination (MMSE) >20
排除标准
- •Has Non-Alzheimer's disease dementia
- •Has alanine aminotransferase (ALT) or aspartate aminotransferase (AST) >2×upper limit of normal (ULN)
- •Has estimated glomerular filtration rate (eGFR) <45 mL/min/1.73m^2 at Screening
- •Has recently received an investigational agent
- •Has recent treatment with amyloid-targeting antibody
- •Note: other protocol defined inclusion / exclusion criteria apply
研究组 & 干预措施
Part B:
Experimental
Participants will be administered multiple doses of ALN-APP.
干预措施: ALN-APP (Drug)
Part A: ALN-APP
Experimental
Participants will be administered a single dose of ALN-APP.
干预措施: ALN-APP (Drug)
Part A: Placebo
Placebo Comparator
Participants will be administered a single dose of placebo.
干预措施: Placebo (Drug)
结局指标
主要结局
Part B: Frequency of Adverse Events
时间窗: Up to 24 months
Part A: Frequency of Adverse Events
时间窗: Up to 12 months
次要结局
- Part A and Part B: Change from Baseline in Levels of Cerebrospinal Fluid (CSF) Soluble Amyloid Precursor Protein Alpha (sAPPα) and Soluble Amyloid Precursor Protein Beta (sAPPβ)(Part A up to 12 months; Part B up to 24 months)
- Part A and Part B: Maximum Observed Plasma Concentration (Cmax) of ALN-APP and of Potential Metabolites(Part A up to 12 months; Part B up to 24 months)
- Part A: Fraction of ALN-APP and Potential Metabolites Excreted in the Urine (fe)(Up to 1 day)
- Part A and Part B: Concentration of ALN-APP and Potential Metabolites at Time 't' (Ct) in Plasma and CSF(Part A up to 12 months; Part B up to 24 months)
- Part A and Part B: Area Under the Plasma Concentration-time Curve (AUC) of ALN-APP and of Potential Metabolites(Part A up to 12 months; Part B up to 24 months)
研究者
研究点 (8)
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