NCT04739059已完成3 期
An Open-label Study to Evaluate the Long-term Safety and Efficacy of CSL312 (Garadacimab) in the Prophylactic Treatment of Hereditary Angioedema
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- CSL Behring
- 入组人数
- 171
- 试验地点
- 44
- 主要终点
- Number of Participants With Treatment-emergent Adverse Events (TEAE)
研究概览
简要总结
This phase 3b study will evaluate long-term safety and efficacy of CSL312 (also known as garadacimab) when administered subcutaneously (SC)
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Prevention
- 盲法
- None
入排标准
- 年龄范围
- 12 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Males and females aged ≥ 12 years
- •Diagnosed with clinically confirmed C1-INH HAE
- •Experienced ≥ 3 HAE attacks during the 3 months before Screening
- •Participated in the Run-in Period for at least 1 month (CSL312-naïve subjects only)
- •Experienced at least an average of 1 HAE attack per month during the Run-in Period
排除标准
- •Concomitant diagnosis of another form of angioedema, such as idiopathic or acquired angioedema or recurrent angioedema associated with urticaria
- •Use of C1-INH products, androgens, antifibrinolytics or other small molecule medications for routine prophylaxis against HAE attacks at least 2 weeks before the first day of the Run-in Period
- •Use of monoclonal antibodies such as lanadelumab (Takhzyro®) 3 months before the first day of the Run-in Period.
- •Female subjects use estrogen-containing oral contraceptives or hormone replacement therapy within 4 weeks prior to screening
- •Female or male subjects who are fertile and sexually active not using or not willing to use an acceptable method of contraception to avoid pregnancy during the study and for 30 days after receipt of the last dose of CSL312
- •Pregnant, breastfeeding, or not willing to cease breastfeeding
研究组 & 干预措施
CSL312
Experimental
Fully human immunoglobulin G subclass 4/lambda recombinant inhibitor monoclonal antibody administered subcutaneously
干预措施: CSL312 (Biological)
结局指标
主要结局
Number of Participants With Treatment-emergent Adverse Events (TEAE)
时间窗: Approximately up to 54 months
Percentage of Participants With TEAE
时间窗: Approximately up to 54 months
The percentage of participants was rounded to one place of decimal.
Number of TEAE
时间窗: Approximately up to 54 months
TEAE Rates Per Injection
时间窗: Approximately up to 54 months
The TEAE rate per injection was calculated as the number of TEAE/ total number of injections. The number of injections was defined as the total injections received by participants during the respective safety evaluation period.
TEAE Rates Per Participant Year
时间窗: Approximately up to 54 months
The TEAE rate per participant year was calculated as the total number of TEAE/ participant years. Participant years was defined as the sum of the time (in years) that participant were exposed to study treatment during the respective safety evaluation period.
次要结局
- Number of Participants With Anti-CSL312 Antibodies(At Day 1, Months 6, 12, 36 and 43 (end of treatment [EOT]))
- The Time-normalized Number (Per Month) of Hereditary Angioedema (HAE) Attacks During the Run-in Period and Treatment Period(Run-in Period: Up to Day 60 and Treatment Period: Approximately up to 52 months)
- The Time-normalized Number (Per Year) of HAE Attacks During Treatment Period(Approximately up to 52 months)
- Percentage Reduction in the Attack Rate During the Treatment Period Compared to the Run-in Period(Run-in Period: Up to 60 days and Treatment Period: Approximately up to 52 months)
- Number of Participants With Percentage Reduction (of >=50%, >=70%, >=90%, and 100%) in HAE Attacks(Run-in Period: Up to Day 60 and Treatment Period: Approximately up to 52 months)
- The Time-normalized Number (Per Month) of HAE Attacks Requiring On-demand Treatment(Approximately up to 52 months)
- Percentage of Participants With Anti-CSL312 Antibodies(At Day 1, Months 6, 12, 36 and 43 (EOT))
- The Time-normalized Number (Per Year) of HAE Attacks Requiring On-demand Treatment(Approximately up to 52 months)
- The Time-normalized Number (Per Month) of Moderate and/or Severe HAE Attacks(Approximately up to 52 months)
- The Time-normalized Number (Per Year) of Moderate and/or Severe HAE Attacks(Approximately up to 52 months)
- Number of Participants Rating Their Response to Therapy as Good or Excellent(At Months 12, 24, and 36)
- Percentage of Participants Rating Their Response to Therapy as Good or Excellent(At Months 12, 24, and 36)
- Number of Participants Experiencing Serious Adverse Events (SAE), Experiencing Death, Related TEAE, TEAE Leading to Study Discontinuation(Approximately up to 54 months)
- Percentage of Participants Experiencing SAE, Experiencing Death, Related TEAE, TEAE Leading to Study Discontinuation(Approximately up to 54 months)
- Number of Participants Experiencing TEAE by Severity(Approximately up to 54 months)
- Percentage of Participants Experiencing TEAE by Severity(Approximately up to 54 months)
- Number of Participants Experiencing Adverse Events of Special Interest (AESI)(Approximately up to 54 months)
- Percentage of Participants Experiencing AESI(Approximately up to 54 months)
- Number of Participants With Laboratory Findings Reported as TEAE(Approximately up to 54 months)
- Percentage of Participants With Laboratory Findings Reported as TEAE(Approximately up to 54 months)
- Number of Participants With Normal C1-esterase Inhibitor (nC1-INH) Experiencing TEAE(Approximately up to 54 months)
- Percentage of Participants With nC1-INH Experiencing TEAE(Approximately up to 54 months)
研究者
研究点 (44)
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