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

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

Study Overview

• Status: Recruiting
• Conditions: Hemophilia
• Intervention / Treatment: Drug: Marstacimab

• Study Type: Observational
• Enrollment (Estimated): 100

Contacts and Locations

• Study Contact: Name: renchi yang, MD; Phone Number: 022-23608174; Email: yangrenchi@ihcams.ac.cn
• Study Contact Backup: Name: feng xue, MD; Phone Number: 022-23608174; Email: xuefeng@ihcams.ac.cn

Study Locations

• China
  • Fujian
    • Xiamen, Fujian, China
      • Recruiting
      • The First Affiliated Hospital of Xiamen University
      • Contact: Rui Su, MD
  • Tianjin Municipality
    • Tianjin, Tianjin Municipality, China
      • Recruiting
      • Institute of haematology and Blood diseases hospital
      • Contact: Feng Xue, MD
      • Principal Investigator: Feng Zhang, MD

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

• 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).

Description

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.

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
To evaluate the annualized bleeding rate (ABR)	To evaluate the annualized bleeding rate (ABR) of different types of bleeds after 6 months of subcutaneous marstacimab injections in hemophilia patients.	after 6 month of marstacimab treatment
To assess changes in joint status scores	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).	after 6 month of marstacimab treatment
patient preferences for subcutaneous	To evaluate patient preferences regarding treatment experience via M-PPQ after 6 months.	after 6 month of marstacimab treatment

Collaborators and Investigators

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

Study record dates

Study Major Dates

• Study Start (Actual): February 15, 2026
• Primary Completion (Estimated): August 31, 2027
• Study Completion (Estimated): December 31, 2028

Study Registration Dates

• First Submitted: May 19, 2025
• First Submitted That Met QC Criteria: May 19, 2025
• First Posted (Actual): May 28, 2025

Study Record Updates

• Last Update Posted (Actual): April 29, 2026
• Last Update Submitted That Met QC Criteria: April 24, 2026
• Last Verified: April 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Genetic Diseases, Inborn; Hematologic Diseases; Blood Coagulation Disorders; Hemorrhagic Disorders; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Hemic and Lymphatic Diseases; Hemophilia A; marstacimab
• Other Study ID Numbers: QT2025001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• IPD Plan Description: Patient-level variables including demographics, clinical characteristics, treatment history will be extracted from medical records. Patient-reported outcomes (PROs), namely the M-PPQ and Hemo-TEM questionnaires, will be reported by patients. Clinician-reported outcomes (ClinROs), namely the HEAD-US-C scale, will be reported by clinicians who have undergone training and certification. Electronic case report forms (eCRFs) will be prepared for all data collection fields.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No