Hereditary Angioedema Kininogen Assay: A Multicenter, Epidemiological, Observational Study.
试验速览
- 阶段
- 不适用
- 状态
- 终止
- 发起方
- 入组人数
- 59
- 试验地点
- 7
- 主要终点
- Exploring the cleaved high-molecular weight kininogen (cHMWK) as a biomarker in HAE type 1/2 patients, as well as to study the differences between HAE type 1/2 patients without lanadelumab treatment versus patients on lanadelumab treatment.
研究概览
简要总结
A multicenter epidemiological observational study aiming to explore the cleaved high-molecular weight kininogen (cHMWK) including identification and characterization of other metabolite/biomarkers in HAE type 1/2 patients
详细描述
Hereditary angioedema (HAE) is a rare autosomal dominant genetic disorder characterized by recurrent tissue angioedema episodes, mainly caused by mutations in the SERPING1 gene that encodes C1 inhibitor (C1-INH), a protease involved in limiting bradykinin production. Low levels of C1-INH (HAE type 1) or dysfunctional C1-INH (HAE type 2) lead to bradykinin accumulation, resulting in capillary leakage and tissue swelling.
High Molecular Weight Kininogen (HMWK) proteolysis, by active plasma kallikrein, results in bradykinin and cHMWK generation.
The goal of this study is to explore the cHMWK concentrations in HAE type 1/2 patients, as a biomarker for this disease.
The HAEKA study is performed in collaboration with Shire. Shire is a wholly owned subsidiary of Takeda Pharmaceutical Company Limited.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
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
- •Informed consent is obtained from the participant
- •The patient with diagnosis of Hereditary Angioedema (HAE) type 1/2 based on international guidelines
- •The patient experienced ≥4 HAE attacks within last 12 month before enrolment in the study
- •The participant is older than 18 years old
排除标准
- •Inability to provide informed consent
- •The patient is not diagnosed with Hereditary Angioedema (HAE) type 1/2
- •The patient experienced ˂ 4 HAE attacks within last 12 month before enrolment in the study
- •The participant is younger than 18 years old
结局指标
主要结局
Exploring the cleaved high-molecular weight kininogen (cHMWK) as a biomarker in HAE type 1/2 patients, as well as to study the differences between HAE type 1/2 patients without lanadelumab treatment versus patients on lanadelumab treatment.
时间窗: 30 months
Samples from HAE patients without lanadelumab treatment versus patients on lanadelumab treatment will be analyzed for cHMWK via Liquid Chromatography Multiple Reaction-monitoring Mass Spectrometry (LC/MRM-MS).
次要结局
- Studying cHMWK as a biomarker in HAE type 1/2 patients with edema attack, as well as to study the differences between HAE type 1/2 patients without lanadelumab treatment versus patients on lanadelumab treatment.(30 months)