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

April 24, 2026 updated by: Institute of Hematology & Blood Diseases Hospital, China

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

Status

Indicates the current recruitment status or the expanded access status.
Recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Observational

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
100

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Contact Backup

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

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

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

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
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.
after 1 month of marstacimab treatment
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.
after 6 month of marstacimab treatment

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
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.
after 6 month of marstacimab treatment
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).
after 6 month of marstacimab treatment
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.
after 6 month of marstacimab treatment

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Institute of Hematology & Blood Diseases Hospital, China

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
February 15, 2026

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
August 31, 2027

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
December 31, 2028

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
May 19, 2025

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
May 19, 2025

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
May 28, 2025

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
April 29, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
April 24, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
April 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant 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

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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