A Long-Term Follow-Up Study in Severe Hemophilia A Subjects Who Received BMN 270 in a Prior BioMarin Clinical Trial (270-401)
Study Details
- Hemophilia A
- Subjects must have completed their primary treatment study. Subjects may enroll in 270-401 even if they have restarted FVIII prophylaxis or other hemophilia A treatment.
- Subjects must be capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol. If the subject is unable to provide consent, a legally authorized representative may provide written informed consent.
- Subjects who do not directly enroll in 270-401 at the time of the study completion visit in their primary treatment study must enroll in 270-401 within 4 months of the date of that study completion visit.
- Subjects must be overtly healthy and not have any condition that, in the opinion of the Investigator or Medical Monitor, would prevent the subject from fully complying with the requirements of the study and/or would impact or interfere with evaluation and interpretation of the study data (including, if applicable, advanced HIV disease).
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.