A Phase 3 Open-label Study to Evaluate the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of CSL312 (Garadacimab) in the Prophylactic Treatment of Hereditary Angioedema in Pediatric Subjects 2 to 11 Years of Age
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- CSL Behring
- 入组人数
- 22
- 试验地点
- 13
- 主要终点
- Number of Participants With Treatment Emergent Adverse Events (TEAE)
研究概览
简要总结
The purpose of this study is to investigate the safety, PK / PD, and efficacy of SC CSL312 for prophylactic treatment of pediatric subjects with HAE.
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
- 2 Years 至 11 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female
- •Aged 2 to 11 years, inclusive, with body weight ≥ 10th percentile based on age
- •Diagnosed with clinically confirmed C1-INH HAE
- •Experienced ≥ 2 HAE attacks during the 6 months before Screening
排除标准
- •Concomitant diagnosis of another form of angioedema, such as idiopathic or acquired angioedema, recurrent angioedema associated with urticaria, or HAE type 3
- •Use of C1-INH products, androgens, antifibrinolytics, approved or future approved medications, or other small molecule medications for routine prophylaxis against HAE attacks within a minimum of 2 weeks before the Treatment Period
- •Participation in another interventional clinical study during the 30 days before the Treatment Period or within 5 half-lives of the final dose of the investigational product administered during the previous interventional study, whichever is longer
- •Having laboratory clinical abnormalities assessed as clinically significant by the investigator in results of hematology or chemistry assessments performed during Screening
- •Currently receiving a therapy not permitted during the study
- •Being pregnant or breastfeeding.
研究组 & 干预措施
CSL312
Ages 2-5 years and 6-11 years will have specific subcutaneous dosing schedules
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: Up to Month 12
Time to Maximum Concentration (Tmax) of CSL312 at Steady-State
时间窗: Up to Month 12
Percentage of Participants With TEAE
时间窗: Up to Month 12
The percentage of participants was rounded to one place of decimal.
Number of TEAE
时间窗: Up to Month 12
TEAE Rates Per Injection
时间窗: Up to Month 12
The TEAE rate per injection was calculated as the number of TEAE/ number of injections. The number of injections was defined as the total injections a participant received during the Safety Evaluation Period under the dosing regimen to which the TEAE was assigned.
TEAE Rates Per Participant-Year
时间窗: Up to Month 12
The TEAE rate per participant year was calculated as number of TEAEs/ participant years. Participant-years of exposure were calculated as the sum of each participant's exposure duration (in years) under the specified dosing regimen or overall. For the time assigned to a dosing regimen, each study day was counted under the corresponding regimen.
Maximum Concentration (Cmax) of CSL312 at Steady-state
时间窗: Up to Month 12
Trough Concentration (Ctrough) of CSL312 at Steady-state
时间窗: At Months 3, 4, 6, 9, 10, and 12
次要结局
- Time-normalized Number of HAE Attacks Per Month(Up to Month 12)
- Time-normalized Number of HAE Attacks Per Year(Up to Month 12)
- Time-normalized Number of HAE Attacks Treated With On-demand Treatment Per Month(Up to Month 12)
- Time-normalized Number of HAE Attacks Treated With On-demand Treatment Per Year(Up to Month 12)
- Time-normalized Number of Moderate and/or Severe HAE Attacks Per Month(Up to Month 12)
- Time-normalized Number of Moderate and/or Severe HAE Attacks Per Year(Up to Month 12)
- Percentage Reduction in the Time-normalized Number of HAE Attacks(Up to Month 12)
- Number of Participants Experiencing at Least Greater Than or Equal to (>=) 50 Percent (%), >= 70%, >= 90%, or Equal to 100% (Attack-free) Reduction in the Time-normalized Number of HAE Attacks(Up to Month 12)
- Number of Participants Experiencing Serious Adverse Events (SAE), Experiencing Death, Related TEAE, TEAE Leading to Study Discontinuation(Up to Month 12)
- Percentage of Participants Experiencing SAE, Experiencing Death, Related TEAE, TEAE Leading to Study Discontinuation(Up to Month 12)
- Number of Participants With TEAE by Severity(Up to Month 12)
- Percentage of Participants With TEAE by Severity(Up to Month 12)
- Number of Participants With Anti-CSL312 Antibodies(At Day 1, Months 6 and 12)
- Percentage of Participants With Anti-CSL312 Antibodies(At Day 1, Months 6 and 12)
- Number of Participants With Adverse Events of Special Interest (AESI)(Up to Month 12)
- Percentage of Participants With AESI(Up to Month 12)
- FXIIa-mediated Kallikrein Activity(At Months 3, 4, and 12 and pre-dose and post dose at Months 6, 9, and 10)
- Percent of Baseline FXIIa-mediated Kallikrein Activity(At Months 3, 4, and 12 and pre-dose and post dose at Months 6, 9, and 10)
- Number of Participants With Laboratory Findings Reported as AE(Up to Month 12)
- Percentage of Participants With Laboratory Findings Reported as AE(Up to Month 12)