NCT02452372已完成1 期
A Phase 1, Single-ascending Dose, Multiple-ascending Dose, and Multi-dose Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Study of Subcutaneously Administered ALN AS1 in Patients With Acute Intermittent Porphyria (AIP)
Alnylam Pharmaceuticals1 个研究点 分布在 1 个国家目标入组 40 人开始时间: 2015年5月6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
- 已完成
- 发起方
- 入组人数
- 40
- 试验地点
- 1
- 主要终点
- The safety of givosiran 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 and tolerability of givosiran (ALN-AS1) in AIP patients as well as to characterize pharmacokinetics (PK) and pharmacodynamics (PD) of ALN-AS1 in AIP patients.
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
- 否
入选标准
- •Diagnosis of AIP
- •Urine PBG at Screening indicating patient is a high excreter
- •No clinically significant health concerns
- •Women of child bearing potential must have a negative pregnancy test, not be nursing, and use effective contraception
- •Willing to provide written informed consent and willing to comply with study requirements.
排除标准
- •Porphyria attack within 6 months of screening
- •Started a new prescription medication within 3 months of screening
- •Clinically significant abnormal laboratory results
- •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
- •Inclusion Criteria:
- •Diagnosis of AIP
- •Patient experienced a porphyria attack or was taking medication to prevent attacks recently
- •No clinically significant health concerns
- •Women of child bearing potential must have a negative pregnancy test, not be nursing, and use effective contraception
- •Willing to provide written informed consent and willing to comply with study requirements.
- •Exclusion Criteria:
- •Stared a new prescription medication within 3 months of screening
- •Clinically significant abnormal laboratory results
- •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
研究组 & 干预措施
givosiran (ALN-AS1)
Active Comparator
干预措施: givosiran (ALN-AS1) (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 givosiran 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 42; Part B (MAD) phase: through Day 70; Part C (MD) phase: through Day 168
次要结局
- Profile of Pharmacokinetics (PK) of givosiran(Part A (SAD) phase: predose - 42 days post-dose; Part B (MAD) phase: predose - 70 days post-dose; Part C (MD) phase: predose - 168 days post-dose)
- The change in delta-aminolevulinic acid (ALA) from baseline(Part A (SAD) phase: screening - 42 days post-dose; Part B (MAD) phase: screening - 70 days post-dose; Part C (MD) phase: screening - 168 days post-dose)
- The change in Porphobilinogen (PBG) from baseline(Part A (SAD) phase: screening - 42 days post-dose; Part B (MAD) phase: screening - 70 days post-dose; Part C (MD) phase: screening - 168 days post-dose)
研究者
研究点 (1)
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