Adult and Adolescent Hemophilia Patients Treated With Marstacimab: a Patient Experience Registry

Recruiting now

Conditions studied: Hemophilia

In brief

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

Key facts

Study ID

NCT06992076

Run by

Institute of Hematology & Blood Diseases Hospital, China

People needed

100

Starts

2026-02-15

Expected to finish

2028-12-31

Last updated by the study team

2026-04-29

Who can join

Age: 12 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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.

Where it is running

• The First Affiliated Hospital of Xiamen University — Xiamen, Fujian, China (enrolling)
• Institute of haematology and Blood diseases hospital — Tianjin, Tianjin Municipality, China (enrolling)

Full record on ClinicalTrials.gov

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