Adult and Adolescent Hemophilia Patients Treated With Marstacimab: a Patient Experience Registry
试验速览
- 阶段
- 不适用
- 状态
- 尚未招募
- 入组人数
- 100
- 主要终点
- patient preferences for subcutaneous
研究概览
简要总结
Research Title：Adult and adolescent hemophilia patients treated with Marstacimab: a patient Experience Registry (AMBER) Protocol Number：94250855 Protocol Version Number：version 0.3 Date：15-November-2024 Leading site：Institution of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences Active Pharmaceutical Ingredient：a human IgG1 monoclonal antibody that targets the tissue factor pathway inhibitor (TFPI) Drug Name Generic name: marstacimab-hncq Purpose of the Study Primary Objectives:To quantify patient preferences for subcutaneous versus intravenous (IV) injection using the Marstacimab-Patient Preference Questionnaire (M-PPQ) after 1 month of marstacimab for routine prophylaxis treatment.
To assess the treatment burden using the Hemophilia Treatment Experience Measure (Hemo-TEM) in hemophilia patients after 6 months of marstacimab for routine prophylaxis treatment.
Exploratory Objectives:
To evaluate the annualized bleeding rate (ABR) of different types of bleeds after 6 months of subcutaneous marstacimab injections in hemophilia patients.
To assess changes in joint status scores between baseline and the final visit after 6 months of subcutaneous marstacimab injections in hemophilia patients, using the Hemophilia Early Arthropathy Detection with Ultrasound in China (HEAD-US-C).
To evaluate patient preferences regarding treatment experience via M-PPQ after 6 months
研究设计
- 研究类型
- Observational
- 观察模型
- Case Only
- 时间视角
- Prospective
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
- •Participants with a diagnosis of hemophilia A or hemophilia B eligible to receive prophylaxis.
- •Clinical physicians prescribe marstacimab for routine prophylaxis treatment of hemophilia patients based on the actual conditions of the patients and in accordance with the approved indications of marstacimab.
- •Adolescent patients(12 to less than 18 years) and adult (18 years and older) patients.
- •Signed consent obtained before the study, participant or legally authorized representative, or participant's caregiver capable of giving signed informed consent.
排除标准
- •Those who are unable to complete at least one month follow-up based on the investigator's judgment.
- •Severe impairment of speech, vision, memory or cognition that affects communication and ability to complete questionnaires and follow-up visits.
- •Women of childbearing age who plan to become pregnant within the next 2 months, as well as women who are pregnant or breastfeeding.
- •Patients are participating in other clinical trials. There are other conditions that the investigator deems unsuitable for participation in this study.
结局指标
主要结局
patient preferences for subcutaneous
时间窗: after 1 month of marstacimab treatment
To quantify patient preferences for subcutaneous versus intravenous (IV) injection using the Marstacimab-Patient Preference Questionnaire (M-PPQ) after 1 month of marstacimab for routine prophylaxis treatment.
To assess the treatment burden
时间窗: after 6 month of marstacimab treatment
To assess the treatment burden using the Hemophilia Treatment Experience Measure (Hemo-TEM) in hemophilia patients after 6 months of marstacimab for routine prophylaxis treatment.
次要结局
- To evaluate the annualized bleeding rate (ABR)(after 6 month of marstacimab treatment)
- To assess changes in joint status scores(after 6 month of marstacimab treatment)
- patient preferences for subcutaneous(after 6 month of marstacimab treatment)