NCT01617967已完成2 期
A Phase 2, Open-Label, Multi-Dose, Dose Escalation Trial to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intravenous Infusions of ALN-TTR02 in Patients With TTR Amyloidosis
Alnylam Pharmaceuticals1 个研究点 分布在 1 个国家目标入组 29 人开始时间: 2012年5月最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 29
- 试验地点
- 1
- 主要终点
- Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Study Drug Discontinuation
研究概览
简要总结
This was a multiple dose, dose escalation study designed to determine the safety, tolerability, pharmacokinetics and pharmacodynamics of patisiran (ALN-TTR02) in participants with transthyretin (TTR) mediated amyloidosis (ATTR).
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
- 18 Years 至 85 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Body mass index must be between 17 kg/m^2 and ≤ 33 kg/m^2;
- •Women of child-bearing potential must have a negative pregnancy test, cannot be breast feeding, and must use appropriate contraception;
- •Males agree to use appropriate contraception;
- •Diagnosis of TTR amyloidosis;
- •Adequate blood counts, liver and renal function;
- •Willing to give written informed consent and are willing to comply with the study requirements.
排除标准
- •Known human immunodeficiency virus (HIV) positive status or known or suspected systemic bacterial, viral, parasitic, or fungal infection;
- •Received an investigational agent, other than tafamidis or diflunisal, within 30 days prior to first dose study drug administration;
- •Prior liver transplant;
- •Poor cardiac function;
- •Considered unfit for the study by the Principal Investigator;
- •Employee or family member of the sponsor or the clinical study site personnel.
研究组 & 干预措施
Patisiran (ALN-TTR02)
Experimental
Two administrations of patisiran will be administered once every 4 weeks [Q4W]) in 4 sequential cohorts with escalating doses followed by optional cohorts with an alternative dosing regimen (once every 3 weeks [Q3W]), and an alternative premedication regimen.
干预措施: Patisiran (Drug)
结局指标
主要结局
Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Study Drug Discontinuation
时间窗: Up to 56 days post first dose
The number of participants experiencing at least one adverse event (AE), at least one serious adverse event (SAE) and study drug discontinuation (due to any reason).
次要结局
- Pharmacokinetic Parameters of Patisiran - Renal Clearance (CLR)(Predose (within 1 h of planned dosing start) and post-infusion at 0-6 h (pooled) on Day 0 and Day 21/28 depending on dosing frequency)
- Pharmacokinetic Parameters of Patisiran - Beta Elimination Half-life (t1/2 Beta)(Predose, end of infusion and post-infusion at 5 minutes (min), 10 min, 30 min, 1 hour (h), 2 h, 4 h, 6 h, 24 h and 48 h on Day 0 and Day 21/28 depending on dosing frequency)
- Pharmacokinetic Parameters of Patisiran - Systemic Clearance (CL)(Predose, end of infusion and post-infusion at 5 minutes (min), 10 min, 30 min, 1 hour (h), 2 h, 4 h, 6 h, 24 h and 48 h on Day 0 and Day 21/28 depending on dosing frequency)
- Percentage Change From Baseline in Serum Transthyretin (TTR) Protein(Baseline to Day 21/28 and Day 42/56 depending on dosing regimen (Q3W/Q4W))
- Pharmacokinetic Parameters of Patisiran - Maximum Observed Plasma Concentration (Cmax)(Predose, end of infusion and post-infusion at 5 minutes (min), 10 min, 30 min, 1 hour (h), 2 h, 4 h, 6 h, 24 h and 48 h on Day 0 and Day 21/28 depending on dosing regimen (Q3W/Q4W))
- Pharmacokinetic Parameters of Patisiran - Apparent Volume of Distribution at Steady State (Vss)(Predose, end of infusion and post-infusion at 5 minutes (min), 10 min, 30 min, 1 hour (h), 2 h, 4 h, 6 h, 24 h and 48 h on Day 0 and Day 21/28 depending on dosing frequency)
- Pharmacokinetic Parameters of Patisiran - Area Under the Concentration Curve From Time 0 to Last Measurable Time Point (AUC0-last)(Predose, end of infusion and post-infusion at 5 minutes (min), 10 min, 30 min, 1 hour (h), 2 h, 4 h, 6 h, 24 h and 48 h on Day 0 and Day 21/28 depending on dosing regimen (Q3W/Q4W))
研究者
研究点 (1)
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