Marstacimab experiences in adolescents and adults with hemophilia

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

This registry will collect experiences from adolescents and adults with hemophilia who are receiving marstacimab to see if they prefer subcutaneous injections and to track treatment burden and bleeding rates.

Quick facts

• Study type: Observational
• Enrollment: 100 (estimated)
• Ages: 12 Years and up
• Sex: All
• Sponsor: Institute of Hematology & Blood Diseases Hospital, China (Academic / other)
• Drugs / interventions: marstacimab
• Locations: 2 sites (Xiamen, Fujian and 1 other locations)
• Trial ID: NCT06992076 on ClinicalTrials.gov

What this trial studies

This observational patient registry enrolls adolescents (12–17) and adults (≥18) with hemophilia A or B who are prescribed marstacimab for routine prophylaxis. Participants complete the Marstacimab-Patient Preference Questionnaire (M-PPQ) after one month to record injection-route preferences and the Hemophilia Treatment Experience Measure (Hemo-TEM) after six months to measure treatment burden. The registry will also collect clinical data, including annualized bleeding rates, to explore real-world bleeding outcomes during marstacimab prophylaxis. Data are collected at participating hospitals in China during routine clinical visits without altering prescribed care.

Who should consider this trial

Good fit: Adolescents (12 to <18) and adults (≥18) with a diagnosis of hemophilia A or B who are prescribed marstacimab for routine prophylaxis and can provide informed consent and complete follow-up are ideal candidates.

Not a fit: Patients who are pregnant or breastfeeding, planning pregnancy within two months, have severe speech/vision/memory or cognitive impairment that prevents questionnaire completion, or cannot complete at least one month of follow-up are unlikely to benefit from participation.

Why it matters

Potential benefit: If successful, the registry could show whether subcutaneous marstacimab improves patient preference and reduces treatment burden, supporting more patient-friendly prophylaxis choices.

How similar studies have performed: Previous clinical trials of marstacimab and other TFPI-targeting agents have reported reductions in bleeding, but patient-preference and treatment-burden registries for marstacimab are relatively novel.

Eligibility criteria

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.

Where this trial is running

Xiamen, Fujian and 1 other locations

• The First Affiliated Hospital of Xiamen University — Xiamen, Fujian, China (Recruiting)
• Institute of haematology and Blood diseases hospital — Tianjin, Tianjin Municipality, China (Recruiting)

Study contacts

• Study coordinator: renchi yang, MD

Conditions Hemophilia