NCT02503683终止1 期
A Phase 1/2, Randomized, Single-blind, Placebo-Controlled, Single Ascending Dose and Multiple Dose, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Subcutaneously Administered ALN-AAT in Healthy Adult Subjects and Patients With ZZ Type Alpha-1 Antitrypsin Deficiency Liver Disease
Alnylam Pharmaceuticals1 个研究点 分布在 1 个国家目标入组 26 人开始时间: 2015年7月9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
- 终止
- 发起方
- 入组人数
- 26
- 试验地点
- 1
- 主要终点
- The safety of ALN-AAT evaluated by the proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs), and AEs leading to study drug discontinuation
研究概览
简要总结
The purpose of this study is to evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Subcutaneously Administered ALN-AAT in Healthy Adult Subjects and Patients with ZZ Type Alpha-1 Antitrypsin Deficiency Liver Disease.
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
- Single (Participant)
入排标准
- 年龄范围
- 18 Years 至 65 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Adequate complete blood counts, liver and renal function.
- •12-lead electrocardiogram (ECG) within normal limits
- •Female subjects must be of non-childbearing potential; e.g. post-menopausal or pre-menopausal with surgical sterilization
- •Male subjects agree to use appropriate contraception
- •Willing to provide written informed consent and willing to comply with study requirements
- •Nonsmokers for at least 5 years before screening
排除标准
- •Any uncontrolled or serious disease, or any medical or surgical condition, that may interfere with participation in the clinical study and/or put the subject at significant risk
- •Received an investigational agent within 90 days before the first dose of study drug or are in follow-up of another clinical study
- •Active serious mental illness or psychiatric disorder requiring current pharmacological intervention
- •History or evidence of alcohol or drug abuse within 12 months before screening.
- •History of intolerance to SC injection
研究组 & 干预措施
ALN-AAT
Active Comparator
干预措施: ALN-AAT (Drug)
Sterile Normal Saline (0.9% NaCl)
Placebo Comparator
干预措施: Sterile Normal Saline (0.9% NaCl) (Drug)
结局指标
主要结局
The safety of ALN-AAT evaluated by the proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs), and AEs leading to study drug discontinuation
时间窗: Part A (SAD phase): through day 70; Part B (MAD) phase: through Day 154; Part C: through Day 224
次要结局
- Profile of Pharmacokinetics (PK) of ALN-AAT(Part A (SAD) phase: up to 21 days; Part B (MAD) phase: up to 105 days; Part C: up to 161 days)
- The effect of ALN-AAT on serum levels of AAT protein(Part A (SAD) phase: through day 70; Part B (MAD) phase: through Day 154; Part C: through Day 224)
研究者
研究点 (1)
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