About this trial
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
Eligibility criteria
Qualifiers
None
Disqualifiers
None
Trial design
Treatments tested in this trial
- Marstacimab