Adult and Adolescent Hemophilia Patients Treated With Marstacimab: a Patient Experience Registry
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Marstacimab.
- Who it may be relevant to
- Registry conditions: Hemophilia. Basic parameters: from 12 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- China
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Overview
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
Interventions
- Drug Marstacimab
the first visit (V1) is scheduled on the day of the first administration of marstacimab. The recommended dosing frequency for the patient should follow the instructions provided, which are as follows: On Day 1, administer 300 mg subcutaneously (divided into two injections, each 150 mg) (loading dose); starting from Day 8, administer 150 mg subcutaneously once a week. Record the patient's prescription recommendations, actual treatment records (administration time, dosage, frequency), and adherenc
Primary outcome measures
- patient preferences for subcutaneous [Time frame: after 1 month of marstacimab treatment]
- To assess the treatment burden [Time frame: after 6 month of marstacimab treatment]
Secondary outcome measures (3)
- To evaluate the annualized bleeding rate (ABR) [Time frame: after 6 month of marstacimab treatment]
- To assess changes in joint status scores [Time frame: after 6 month of marstacimab treatment]
- patient preferences for subcutaneous [Time frame: after 6 month of marstacimab treatment]
Eligibility criteria
Inclusion criteria
\- 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 criteria
\- 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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Case-only
Study locations
China · 2 centers
- The First Affiliated Hospital of Xiamen University — Xiamen
- Institute of haematology and Blood diseases hospital — Tianjin
Identifiers
NCT: NCT06992076 · QT2025001