An Open-label Exploratory Phase II Study of the Safety and Immunogenicity of Repeated "rhC1INH" Administration of 50 U/Kg in Patients With Hereditary C1 Inhibitor Deficiency ("HAE")
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 25
- 试验地点
- 2
- 主要终点
- HAE Attacks/Week
研究概览
简要总结
Hereditary angioedema ("HAE") is a disease characterized by recurrent tissue swelling affecting various body locations. Recent literature shows that patients with frequent attacks may benefit from long-term prophylaxis. This study aims to evaluate the safety and prophylactic effect of weekly administrations of 50 IU/kg recombinant C1 Inhibitor ("rhC1INH").
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
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Aged at least 18 years
- •Signed informed consent
- •Comfirmed diagnosis of HAE with baseline plasma level of functional C1INH activity of less than 50% of normal, and/or proven HAE ,mutation in C1INH gene.
排除标准
- •A history of anaphylaxis or severe allergy (i.e. requiring medication) to food, proteins and/or drugs.
- •A history of allergic reactions to C1INH products or rabbit protein.
- •Any reported SAE related to study drug administration (withdrawal criterium)
- •Elevated IgE against rabbit dander (>0.35 kU/L; ImmunoCap assay; Phadia)
- •A diagnosis of acquired C1INH deficiency.
- •Woman of child bearing potential, pregnancy or breast-feeding
- •previous treatment within the last 3 months with plasma-derived C1INH
- •Any clinically significant abnormality in the routine haematology, biochemistry and urinalysis
- •Any condition or treatment that in the opinion of the investigator might interfere with the evaluation of the study objectives.
- •Any changes since screening that would exclude subject based on above exclusion criteria.
研究组 & 干预措施
Recombinant Human C1 Inhibitor
Weekly administration of 50 IU/kg Recombinant Human C1 Inhibitor
干预措施: Recombinant Human C1 Inhibitor (Drug)
结局指标
主要结局
HAE Attacks/Week
时间窗: 8 weeks
Prior to the treatment period, patients enrolled in the study, were asked about the amount of "HAE" attacks in the past 2 years, (calculated to attacks/week), this number is defined as "Historical". During the treatment period, patients received a dose of 50 IU/kg of "rhC1INH" administered by slow "IV" injection over 4 to 5 minutes, once a week during an eight week period. The amount of attacks during this period is defined as "Prophylaxis" (calculated to attacks/week).
次要结局
- The Evaluation of Pharmacokinetic/ Pharmacodynamic ("PK/PD")Parameters.(8 weeks)