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NCT Number: NCT06992076

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

Recruiting

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Key information

Conditions

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

Age range

12 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

The First Affiliated Hospital of Xiamen University, Xiamen, Fujian, China

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

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.

Primary outcomes

  1. patient preferences for subcutaneous

    Time frame: after 1 month of marstacimab treatment

    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.

  2. To assess the treatment burden

    Time frame: after 6 month of marstacimab 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.

Secondary outcomes

  1. To evaluate the annualized bleeding rate (ABR)

    Time frame: after 6 month of marstacimab treatment

    To evaluate the annualized bleeding rate (ABR) of different types of bleeds after 6 months of subcutaneous marstacimab injections in hemophilia patients.

  2. To assess changes in joint status scores

    Time frame: after 6 month of marstacimab treatment

    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).

  3. patient preferences for subcutaneous

    Time frame: after 6 month of marstacimab treatment

    To evaluate patient preferences regarding treatment experience via M-PPQ after 6 months.

Study contacts

Contact information is provided by the study sponsor or research team.

feng xue, MD

CONTACT

[email protected]

022-23608174

renchi yang, MD

CONTACT

[email protected]

022-23608174

Sponsors and collaborators

Lead sponsor

Institute of Hematology & Blood Diseases Hospital, China

Other

Registry information

Acronym: AMBER

Important dates

Study start
2026
Primary completion
2027
Study completion
2028
First posted
May 28, 2025
Registry last updated
Apr 29, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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