A Double-blind, Randomized, Placebo-controlled, Cross-over Study to Evaluate the Clinical Efficacy and Safety of Subcutaneous Administration of Human Plasma-derived C1-esterase Inhibitor in the Prophylactic Treatment of Hereditary Angioedema
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- CSL Behring
- 入组人数
- 90
- 试验地点
- 40
- 主要终点
- The Time-normalized Number of Hereditary Angioedema Attacks
研究概览
简要总结
The aim of this study is to assess the efficacy of C1-esterase inhibitor in preventing hereditary angioedema attacks when it is administered under the skin of subjects with hereditary angioedema. The safety of C1-esterase inhibitor will also be assessed. Each subject will enter a run-in period of up to 8-weeks. Subjects who complete the run-in period and who are eligible will then enter the treatment phase which comprises two sequential treatment periods. In the treatment phase, subjects will be randomized to one of four arms consisting of treatment with low- or higher-volume C1-esterase inhibitor in one treatment period and treatment with low- or higher-volume placebo in the other treatment period. The study will measure the number of hereditary angioedema attacks that subjects experience while receiving each treatment.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Prevention
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
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
- •Run-In Period Inclusion Criteria:
- •Males or females aged 12 years or older.
- •A clinical diagnosis of hereditary angioedema type I or II.
- •Hereditary angioedema attacks over a consecutive 2-month period that required acute treatment, medical attention, or caused significant functional impairment.
- •For subjects who have used oral therapy for prophylaxis against HAE attacks within 3 months of Screening: use of a stable regimen within 3 months of Screening, with no plans to change.
- •Eligibility Criteria for Entering Treatment Period 1:
- •Laboratory confirmation of type I or type II hereditary angioedema, including C1-esterase inhibitor functional activity less than 50% AND C4 antigen level below the laboratory reference range.
- •No clinically significant abnormalities as assessed using laboratory parameters.
- •During participation in the run-in period, subjects must have experienced hereditary angioedema attacks that required acute treatment, required medical attention, or caused significant functional impairment.
排除标准
- •Run-In Period Exclusion Criteria:
- •History of clinical significant arterial or venous thrombosis, or current history of a clinically significant prothrombotic risk.
- •Incurable malignancies at screening.
- •Any clinical condition that will interfere with the evaluation of C1-esterase inhibitor therapy.
- •Clinically significant history of poor response to C1-esterase therapy for the management of hereditary angioedema.
- •Receiving therapy prohibited by the protocol, including medications for hereditary angioedema prophylaxis.
- •Female subjects who started taking or changed dose of any hormonal contraceptive regimen or hormone replacement therapy (i.e., estrogen/progesterone-containing products) within 3 months prior to the screening visit.
结局指标
主要结局
The Time-normalized Number of Hereditary Angioedema Attacks
时间窗: During the treatment phase, up to 28 weeks.
The time normalized number of HAE attacks as reported by the investigator per subject was calculated as: The total number of HAE attacks per subject and per treatment period / length of stay of subject in treatment period (days), Where length of stay of subject in treatment period was calculated as: Date of last day of subject in treatment period - date of first day of Week 3 of subject in treatment period + 1.
次要结局
- Time-Normalized Number of Uses of Rescue Medication(During the treatment phase, up to 28 weeks.)
- Percentage of Subjects With Adverse Events (AEs) Within 24 Hours of C1-esterase Inhibitor or Placebo Administration(Within 24 hours of C1-esterase inhibitor or placebo administration.)
- Percentage of Subjects Experiencing Solicited AEs (Injection Site Reactions)(During the treatment phase, up to 32 weeks.)
- Injections Resulting in Solicited AEs (Injection Site Reactions)(During the treatment phase, up to 32 weeks.)
- Percentage of Subjects With AEs or Other Specified Safety Events.(During the treatment phase, up to 32 weeks.)
- Percentage of Subjects With a ≥ 50% Reduction in the Number of Hereditary Angioedema Attacks by CSL830 Treatment(During the treatment phase, up to 28 weeks.)