Incidence of Ultrasonographically Detected Articular Damage in Initially Healthy Joints of Patients With Hemophilia A Receiving Prophylactic Treatment With Emicizumab
Study Details
- Hemophilia
- Confirmed diagnosis of hemophilia A;
- Receiving prophylactic treatment with emicizumab (Hemlibra®) according to routine clinical practice;
- Age ≥10 years at the time of study enrollment;
- Absence of ultrasonographic joint damage in at least one of the evaluated joints (elbows, knees, or ankles), defined as HEAD-US = 0 at baseline assessment;
- No documented history of clinically evident hemarthrosis in the corresponding joints from initiation of emicizumab prophylaxis to the study baseline evaluation;
- Ability to understand and complete study procedures (interviews, questionnaires, and clinical assessments), in accordance with the participant's age; and
- Provision of written informed consent; for minors, written informed consent from parents or legal guardians and assent from the minor participant, in accordance with applicable regulations.
- Presence of ultrasonographic joint damage (HEAD-US ≥1) in all evaluated joints at the baseline visit;
- Documented history of clinically evident hemarthrosis in the joints under study;
- Prior major orthopedic surgery or arthroplasty in the evaluated joints;
- Presence of concomitant musculoskeletal pathology unrelated to hemophilia that could interfere with joint assessment (e.g., inflammatory arthritis, recent severe trauma);
- Inability to complete the planned 24-month follow-up or to undergo study assessments;
- Concurrent participation in another interventional study that could interfere with the joint health variables under evaluation; and
- Any clinical or social condition that, in the investigator's judgment, could compromise participant safety or the validity of the study data.
Protocol Summary
This study is looking at how safe it is to switch from emicizumab to Mim8, in people with haemophilia A. Mim8 is a new medicine that is used to prevent bleeding episodes in people with haemophilia A. Mim8 works by replacing the function of the missing clotting factor VIII (FVIII). Mim8 will be injected under the skin using a pen-injector either once every week, once every two weeks or once every month. The participants will be trained in using the pen injector. The participants can choose themselves, in collaboration with the study doctor how often they get Mim8 in this study. When the participant will get their first Mim8 injection depends on their current treatment with emicizumab. The participants will get their first Mim8 injection at Visit 2. Participants will have between 6 and 27 Mim8 injections. The total number of injections participants will have depends on their dosing frequency. The study will last for about 6-12 months. While taking part in this study, there are some restrictions about what medicine participant can use. The study doctor will tell the participants more about this. In case the participants experience bleeds, these can be treated with additional haemostatic medicine as agreed with the study doctor. Female participants cannot take part if they are pregnant, breast-feeding or plan to get pregnant during the study period.