Prevention of Bleeding in Patients With Moderate and Severe Hemophilia A Playing Sports: A Comparison Between Factor VIII and Emicizumab Prophylaxis
Study Details
- Hemophilia A
- Drug: Emicizumab
- Drug: FVIII
- Participant (if 18 years of age or older) or parent/LAR is willing and able to provide written informed consent; minor participant is willing and able to provide assent, if applicable based on site and local regulations
- Males and females between 6 to ≤ 19 years of age at time of enrollment with moderate to severe Hemophilia A (FVIII activity ≤ 5%) without inhibitors are eligible for participation in this study
- Participants must be on Emicizumab or standard FVIII prophylaxis per institutional/primary hematologist recommendations
- Participants must be engaging in or registered to start participating in one or more sports activities with moderate to high risk of bleeding as defined by the NHF- Playing it Safe guidelines (numerical rating \>/= 2).
- Participant must be willing to keep activity, bleed, and treatment logs for the duration of the study
- Participant/parent/LAR unwilling to provide informed consent/assent
- Unwilling to log or document bleeds and treatment information as per study guidelines
- Participants with any other bleeding disorders will be excluded
- Patients who are pregnant, planning to become pregnant, or breastfeeding should not be enrolled in the study
- Participants on concomittent FVIII replacement and emicizumab for sports participation
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.