NCT01148953已完成1 期
A Phase 1, Randomized, Single-Blind, Placebo-Controlled, Dose Escalation Trial to Evaluate the Safety and Tolerability of a Single Dose of Intravenous ALN-TTR01 in Patients With TTR Amyloidosis
Alnylam Pharmaceuticals1 个研究点 分布在 1 个国家目标入组 32 人开始时间: 2010年6月最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- 入组人数
- 32
- 试验地点
- 1
- 主要终点
- The proportion of patients experiencing adverse events (AEs), serious adverse events (SAEs), and study drug discontinuation.
研究概览
简要总结
The purpose of this study is to determine the safety, tolerability, pharmacokinetics and pharmacodynamics of a single dose of ALN-TTR01 in patients with transthyretin (TTR) mediated amyloidosis (ATTR).
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 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of TTR amyloidosis
- •Adequate blood counts, liver and renal function
- •Women of child-bearing potential must have a negative pregnancy test, cannot be breast feeding, and must use an adequate method of birth control
- •Males agree to use appropriate contraception
- •Willing and able to comply with protocol-required visit schedule and visit requirements and provide written informed consent.
排除标准
- •Known human immunodeficiency virus (HIV) positive status
- •Receiving antibiotics for bacterial infection within 7 days of screening
- •Known or suspected systemic viral, parasitic or fungal infection
- •Receiving an investigational agent within 30 days prior to study drug administration
- •Poor cardiac function
- •Considered unfit for the study by the Principal Investigator
- •Known sensitivity to oligonucleotides
- •Employee or family member of the sponsor or the clinical study site personnel.
研究组 & 干预措施
ALN-TTR01
Active Comparator
干预措施: ALN-TTR01 (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 proportion of patients experiencing adverse events (AEs), serious adverse events (SAEs), and study drug discontinuation.
时间窗: Up to 28 Days
次要结局
- Pharmacokinetics (PK) of ALN-TTR01 (Cmax, tmax, t1/2, AUC0-last, CL)(Up to 70 days)
- Effect of ALN-TTR01 on Circulating TTR Levels (Determination of % Lowering of TTR to Pretreatment/Baseline TTR Level)(up to 70 days)
研究者
研究点 (1)
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