Long-term Study Evaluating Joint Health in People With Haemophilia A Receiving Real-world Prophylactic Treatment With Efanesoctocog Alfa
Study Details
- Hemophilia A
- Drug: Efanesoctocog alfa
- Male or female patient with a diagnosis of haemophilia A.
- 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.
- Prescribed with efanesoctocog alfa prophylactic treatment within 6 months prior to study enrolment or at the latest at the enrolment visit, in accordance with local regulations.
- Must have received prophylactic treatment(s) with any haemophilia product(s) for at least 12 months prior to being prescribed with efanesoctocog alfa treatment.
- Have documented pre-study treatment data on haemophilia prophylaxis prescriptions and on treated bleeding episodes from the 12-months prior to being prescribed efanesoctocog alfa treatment and until enrolment into this study.
- Willing to adhere to the visit schedule and to undergo mandatory study assessment (HJHS, PROs).
- Willingness and the ability of the patient or their legally designated representative to document all bleeding episodes, including any treatments for those episodes.
- For HEAD-US subgroup: Accept to undergo annual ultrasound assessment on the index joints (ankles, elbows, knees).
- Acquired haemophilia A and other blood clotting disorders than hereditary haemophilia A.
- Any positive FVIII inhibitor result (defined as inhibitor titre ≥0.6 Bethesda unit \[BU\]/mL) from the medical records in connection to the switch to efanesoctocog alfa until the enrolment visit.
- Enrolment in a concurrent clinical interventional study, or intake of an investigational medicinal product (IMP), including for haemophilia prophylaxis, within 3 months prior to enrolment in this study.
- Patient not suitable for participation, whatever the reason, as judged by the Investigator, e.g., patient is not able or willing to perform the study assessments.
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.