HemophiliaAMBERNCT06992076
Adult and Adolescent Hemophilia Patients Treated With Marstacimab: a Patient Experience Registry
An observational study of Hemophilia, run by Institute of Hematology & Blood Diseases Hospital, China at 2 sites in China.
Trial 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… Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Hemophilia
- What is being tested?
- Marstacimab
- Type of treatment
- Injection or infusion
- How many people
- 100 participants
- Where
- 2 sitesChina
- Study dates
- 15 Feb 2026to 31 Dec 2028
- How long will I take part?
- 6 Months
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
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).
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
2 sites in 1 country. Contact the study team or a site near you to ask about taking part.
China2 sites, 2 recruiting
- Recruiting
Institute of haematology and Blood diseases hospital
Tianjin, Tianjin Municipality
Feng Xue, MD
Feng Zhang, MD
- Recruiting
The First Affiliated Hospital of Xiamen University
Xiamen, Fujian
Rui Su, MD
About this study
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.
Read more
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
- Being tested
- Marstacimab
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MarstacimabDrug
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.
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: after 6 month of marstacimab treatment
Secondary outcomes (3)
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.
Measured: 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).
Measured: after 6 month of marstacimab treatment
patient preferences for subcutaneous
To evaluate patient preferences regarding treatment experience via M-PPQ after 6 months.
Measured: after 6 month of marstacimab treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 May 2025
- Study start15 Feb 2026
- Primary completion31 Aug 2027 (estimated)
- Study completion31 Dec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for HemophiliaStudies of the same condition, open and under way.
- More trials run by Institute of Hematology & Blood Diseases Hospital, ChinaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 100 participants (estimated)
- Target duration
- 6 Months
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06992076
- Sponsor's protocol ID
- QT2025001
- Acronym
- AMBER
- Status last verified
- Apr 2026
- First submitted
- 19 May 2025
- Last updated
- 29 Apr 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: 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.
Study at a glance
From the registry record, last updated by the sponsor on 29 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Adult and Adolescent Hemophilia Patients Treated With Marstacimab: a Patient Experience Registry
- ClinicalTrials.gov ID
- NCT06992076
- Acronym / sponsor ID
- AMBER / QT2025001
- Conditions
- Hemophilia
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 100 participants (estimated)
- Lead sponsor
- Institute of Hematology & Blood Diseases Hospital, ChinaUniversity, hospital or other
- Study start
- 15 Feb 2026 (actual)
- Primary completion
- 31 Aug 2027 (estimated)
- Study completion
- 31 Dec 2028 (estimated)
- First posted
- 28 May 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Case only
- Time perspective
- Prospective
- Target follow-up
- 6 Months
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- 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).
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (4 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Marstacimabthe 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. | Drug | – |
Primary outcome
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.
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.
3 secondary outcomes
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Apr 2026.
- Individual participant dataSharing planned: No. 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hemophilia, run by Institute of Hematology & Blood Diseases Hospital, China at 2 sites in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 29 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact renchi yang, MD (022-23608174, yangrenchi@ihcams.ac.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
patient preferences for subcutaneous (time frame: after 1 month of marstacimab treatment). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Apr 2026.