Tracks patient experience of marstacimab treatment for hemophilia in adults and adolescents
Follows people with hemophilia treated with marstacimab, recording how they experience the injections.
Plain-language title generated from the registry record
Registry title: Adult and Adolescent Hemophilia Patients Treated With Marstacimab: a Patient Experience Registry
Key facts
- Where
- Tianjin and Xiamen, ChinaSee the 2 sites
Where in China
- Institute of haematology and Blood diseases hospitalTianjin, ChinaTaking people
- The First Affiliated hospital of Xiamen UniversityXiamen, ChinaTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Institute of Hematology & Blood Diseases Hospital, China
- Trial code
- NCT06992076
- Record updated
- 29 April 2026
- Original description
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.Read full original descriptionShow less
Adult and Adolescent Hemophilia Patients Treated With Marstacimab: a Patient Experience Registry
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
Inclusion: 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.
Exclusion: 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.