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

Launched by INSTITUTE OF HEMATOLOGY & BLOOD DISEASES HOSPITAL, CHINA · May 19, 2025

Keywords

ClinConnect Summary

This clinical trial, called the AMBER study, is researching a new treatment for hemophilia using a medication called Marstacimab. The study aims to understand how patients feel about receiving this treatment through an injection under the skin (subcutaneous) compared to an injection into a vein (intravenous). It will also look at how patients manage their treatment over time and how often they experience bleeding episodes. The trial will include both adults and adolescents aged 12 and older who have hemophilia A or B and are eligible for regular treatment.

Participants in this study can expect to fill out questionnaires about their treatment experience and preferences after one month and again after six months of using Marstacimab. They will also undergo some assessments to monitor their bleeding rates and joint health. To join, participants must be able to follow the study's requirements and provide informed consent. However, those who are pregnant, breastfeeding, or unable to complete the follow-up will not be eligible. This trial is not yet recruiting, but it aims to improve the quality of life for those living with hemophilia by exploring their treatment options.

Gender

ALL

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.