Efanesoctocog Prophylaxis Or The Optimization Of Treatment: Real-World Experience Of Taylorizing Clotting Therapy In Hemophilia A
Study Details
- Hemophilia A
- Conduct of research: patients followed up at one of the eight investigator centers of the BERHLINGO network
- Pathology: severe HA (FVIII:C \<1 IU/dL) or moderate HA (1 IU/dL \< FVIII:C \< 3 IU/dL), without inhibitor / with history of inhibitor (inhibitor rate \< 0.6 Bethesda unit per milliliter (BU/mL))
- Prophylactic Treatment with FVIII or emicizumab since at least March 1, 2025 -
- Sex: male or female
- Age: patients of all age
- Consent: patient who did not object to their participation in the E-PROTECT study
- Patients under guardianship
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.