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

NCT06992076 | Recruiting

• 1 other identifier: QT2025001
• Study Type: observational
• Target: 100
• Locations: 1 country
• Sites: 2

Brief Summary

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

Conditions

Hemophilia

Outcome Measures

Primary Outcomes (2)

• patient preferences for subcutaneous

  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.

  after 1 month of marstacimab treatment

• To assess the treatment burden

  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.

  after 6 month of marstacimab treatment

Secondary Outcomes (3)

• 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

Interventions

Marstacimab DRUG

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 adherence rates. Visit 2 (V2) and visit 3 (V3) are defined as 30 days (±7 days) and 180 days (±30 days) after the initiation of marstacimab treatment, respectively.

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

patients with hemophilia who will receive marstacimab for routine prophylactic treatment in a real-world setting. Each patient will receive treatment and care according to the standard clinical practice. The total recruitment period will be 18 months. Patients will be prescribed marstacimab in routine practice and initiate marstacimab treatment on the same day (or as soon as possible after prescribing). The observation period for individual patients is defined as ending on the 180th day from the index date (the date of the first dose of marstacimab).

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.

Sponsors & Collaborators

• Institute of Hematology & Blood Diseases Hospital, China: lead

Study Sites (2)

The First Affiliated Hospital of Xiamen University

Xiamen, Fujian, China

RECRUITING

Institute of haematology and Blood diseases hospital

Tianjin, Tianjin Municipality, China

RECRUITING

MeSH Terms

Conditions

Hemophilia A

Interventions

marstacimab

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Central Study Contacts

renchi yang, MD

CONTACT

022-23608174; yangrenchi@ihcams.ac.cn

feng xue, MD

CONTACT

022-23608174; xuefeng@ihcams.ac.cn

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: PROSPECTIVE
• Target Duration: 6 Months
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 19, 2025
• First Posted: May 28, 2025
• Study Start: February 15, 2026
• Primary Completion (Estimated): August 31, 2027
• Study Completion (Estimated): December 31, 2028
• Last Updated: April 29, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

CN (2)