A Multicenter, Randomized, Placebo-controlled, Parallel-arm Study to Investigate the Efficacy, Pharmacokinetics, and Safety of CSL312 in Subjects With Hereditary Angioedema
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- CSL Behring
- 入组人数
- 44
- 试验地点
- 16
- 主要终点
- The Mean Time Normalized Number of HAE Attacks Per Month in Subjects With C1-INH HAE During Treatment Period 1
研究概览
简要总结
This is a multicenter, randomized, placebo-controlled, parallel-arm, phase 2 study to investigate the clinical efficacy, pharmacokinetics, and safety of CSL312 as prophylaxis to prevent attacks in subjects with HAE.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Prevention
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
盲法说明
Double blind
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
- •Male or female
- •Aged ≥ 18 to ≤ 65 years
- •A diagnosis of C1-INH HAE or FXII/PLG HAE;
- •For subjects with C1-INH HAE: ≥ 4 HAE attacks over a consecutive 2-month period during the 3 months before Screening, as documented in the subject's medical record.
排除标准
- •History of clinically significant arterial or venous thrombosis, or current clinically significant prothrombotic risk
- •History of an uncontrolled, abnormal bleeding event due to a coagulopathy, or a current clinically significant coagulopathy or clinically significant risks for bleeding events
- •Known incurable malignancies
研究组 & 干预措施
Placebo
Subjects with C1-INH HAE receiving buffer only
干预措施: Placebo (Drug)
CSL312 (low)
Subjects with C1-INH HAE receiving low dose CSL312
干预措施: Factor XIIa antagonist monoclonal antibody (Biological)
CSL312 (med)
Subjects with C1-INH HAE receiving medium dose CSL312
干预措施: Factor XIIa antagonist monoclonal antibody (Biological)
CSL312 (high)
Subjects with C1-INH HAE receiving high dose CSL312
干预措施: Factor XIIa antagonist monoclonal antibody (Biological)
CSL312 (med/high)
Subjects with C1-INH HAE receiving medium/high dose CSL312
干预措施: Factor XIIa antagonist monoclonal antibody (Biological)
结局指标
主要结局
The Mean Time Normalized Number of HAE Attacks Per Month in Subjects With C1-INH HAE During Treatment Period 1
时间窗: 13 weeks
The time-normalized number of HAE attacks per month during Treatment Period 1 for a subject was calculated as the (number of HAE attacks / length of subject's evaluation period in days) \* 30.4375
次要结局
- The Number of Responder Subjects With C1-INH HAE During Treatment Period 1(13 weeks)
- The Percentage of Responder Subjects With C1-INH HAE During Treatment Period 1(13 weeks)
- The Number of HAE Attack-free Subjects With C1-INH HAE During Treatment Period 1(13 weeks)
- The Percentage of HAE Attack-free Subjects With C1-INH HAE During Treatment Period 1(13 weeks)
- Terminal Elimination Half-life (T1/2) of CSL312 in Subjects With C1-INH HAE During Treatment Period 1(13 weeks)
- Area Under the Concentration-time Curve in 1 Dosing Interval (AUC0-tau) of CSL312 in Subjects With C1-INH HAE During Treatment Period 1(13 weeks)
- The Percentage of Mild, Moderate or Severe HAE Attacks in Subjects With C1-INH HAE During Treatment Period 1(13 weeks)
- The Number of Subjects With at Least One (1) HAE Attack Treated With On-demand HAE Medication, in Subjects With C1-INH HAE During Treatment Period 1(13 weeks)
- The Percentage of Subjects With at Least One (1) HAE Attack Treated With On-demand HAE Medication, in Subjects With C1-INH HAE During Treatment Period 1(13 weeks)
- Time of Maximum Concentration (Tmax) of CSL312 in Subjects With C1-INH HAE During Treatment Period 1(13 weeks)
- Volume of Distribution During the Elimination Phase (Vz/F) of CSL312 in Subjects With C1-INH HAE During Treatment Period 1(13 weeks)
- Clearance (CL/F) of CSL312 in Subjects With C1-INH HAE During Treatment Period 1(13 weeks)
- The Number of Mild, Moderate or Severe HAE Attacks in Subjects With C1-INH HAE During Treatment Period 1(13 weeks)
- The Mean Time-normalized Number of Mild, Moderate or Severe HAE Attacks Per Month in Subjects With C1-INH HAE During Treatment Period 1(13 weeks)
- Maximum Concentration (Cmax) of CSL312 in Subjects With C1-INH HAE During Treatment Period 1(13 weeks)
- The Number of Subjects With C1-INH HAE With Adverse Events (AEs), Serious Adverse Events (SAEs), Adverse Events of Special Interest (AESI), Injection Site Reactions (ISRs), Binding Antibodies to CSL312 During Treatment Period 1(13 weeks)