NCT02352493已完成1 期
A Phase 1/2 Single-ascending and Multiple-ascending Dose, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Study of Subcutaneously Administered ALN-CC5 in Healthy Adult Volunteers and Patients With Paroxysmal Nocturnal Hemoglobinuria
Alnylam Pharmaceuticals3 个研究点 分布在 1 个国家目标入组 62 人开始时间: 2015年1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
- 62
- 试验地点
- 3
- 主要终点
- Number of Participants With Adverse Events
研究概览
简要总结
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of ALN-CC5 in healthy adult volunteers and subjects with PNH
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
- Double (Participant, Investigator)
入排标准
- 年龄范围
- 18 Years 至 45 Years（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 of child bearing potential agreeing to use a protocol specified method of contraception
- •Male subjects agreeing to use protocol specified methods of contraception
- •Willing to provide written informed consent and willing to comply with study requirements
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
- •History of multiple drug allergies or intolerance to subcutaneous injection
- •Parts A and B of the study: Used prescription medications within 14 days or 7 half-lives of administration of the first dose of study drug.
- •History of meningococcal infection
研究组 & 干预措施
Sterile Normal Saline (0.9% NaCl)
Placebo Comparator
干预措施: Sterile Normal Saline (0.9% NaCl) (Drug)
ALN-CC5
Active Comparator
干预措施: ALN-CC5 (Drug)
结局指标
主要结局
Number of Participants With Adverse Events
时间窗: Part A: through day 658; Part B: through day 532; Part C: through day 280
Adverse events were reported for single-ascending doses (SAD) or multiple ascending doses (MAD) of ALN-CC5 when administered to healthy adult subjects and of multiple doses (MD) in patients with paroxysmal nocturnal hemoglobinuria (PNH)
次要结局
- Pharmacodynamic (PD) Effect of ALN-CC5: Percentage Reduction From Baseline in Complement Alternative Pathway (CAP)(Part A: through day 70; Part B: through day 140; Part C: through day 140)
- Pharmacokinetic (PK) Effect of ALN-CC5: T Max (23-mer)(Part A: 0-48 hrs, Day 0; Part B (weekly dosing cohorts): 0-48 hrs, Day 28; Part B (biweekly, weekly/monthly/biweekly/monthly cohorts): 0-48 hrs, Day 84; Part C: 0- 24 hrs, Day 84)
- Pharmacokinetic (PK) Effect of ALN-CC5: AUC 0-t (23-mer)(Part A: 0-48 hrs, Day 0; Part B (weekly dosing cohorts): 0-48 hrs, Day 28; Part B (biweekly, weekly/monthly/biweekly/monthly cohorts): 0-48 hrs, Day 84; Part C: 0- 24 hrs, Day 84)
- Pharmacokinetic (PK) Effect of ALN-CC5: Cmax (25-mer)(Part A: 0-48 hrs, Day 0; Part B (weekly dosing cohorts): 0-48 hrs, Day 28; Part B (biweekly, weekly/monthly/biweekly/monthly cohorts): 0-48 hrs, Day 84; Part C: 0- 24 hrs, Day 84)
- Pharmacodynamic (PD) Effect of ALN-CC5: Percentage Reduction From Baseline in C5 Protein Levels(Part A: through day 70; Part B: through day 140; Part C: through day 140)
- Pharmacodynamic (PD) Effect of ALN-CC5: Percentage Reduction From Baseline in Complement Classical Pathway (CCP)(Part A: through day 70; Part B: through day 140; Part C: through day 140)
- Pharmacokinetic (PK) Effect of ALN-CC5: Cmax (23-mer)(Part A: 0-48 hrs, Day 0; Part B (weekly dosing cohorts): 0-48 hrs, Day 28; Part B (biweekly, weekly/monthly/biweekly/monthly cohorts): 0-48 hrs, Day 84; Part C: 0- 24 hrs, Day 84)
- Pharmacokinetic (PK) Effect of ALN-CC5: T Max (25-mer)(Part A: 0-48 hrs, Day 0; Part B (weekly dosing cohorts): 0-48 hrs, Day 28; Part B (biweekly, weekly/monthly/biweekly/monthly cohorts): 0-48 hrs, Day 84; Part C: 0- 24 hrs, Day 84)
- Pharmacokinetic (PK) Effect of ALN-CC5: AUC 0-t (25-mer)(Part A: 0-48 hrs, Day 0; Part B (weekly dosing cohorts): 0-48 hrs, Day 28; Part B (biweekly, weekly/monthly/biweekly/monthly cohorts): 0-48 hrs, Day 84; Part C: 0- 24 hrs, Day 84)
研究者
研究点 (3)
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