A Study Evaluating Physical Activity and Joint Health in Severe Haemophilia A Patients ≥12 Years Treated Once Weekly With Efanesoctocog Alfa
Study Details
- Hemophilia A, Severe
- Drug: Efanesoctocog alfa
- Previous treatment for haemophilia A with any marketed recombinant and/or plasma-derived FVIII for at least 150 EDs.
- Having received prophylactic treatment with any marketed recombinant and/or plasma FVIII or emicizumab per local label for at least 12 months preceding enrolment.
- Having 12 months documented pre-study treatment data regarding prophylactic treatment prescriptions and 6 months data on bleeding episodes prior to baseline visit.
- Willingness and ability to complete training in the use of the study ePD and to use the ePD in their own smartphone throughout the study.
- Willingness and ability to use the activity tracker provided by the sponsor to measure physical activity and heart rate.
- Be able and willing to administer efanesoctocog alfa intravenously at home.
- Serious musculoskeletal and/or neurological impairment limiting the mobility and the physical ability to a degree that makes the patient unsuitable for the study as judged by the investigator.
- Other known coagulation disorder(s) in addition to haemophilia A.
- History and/or current positive inhibitor test defined as ≥0.6 BU/mL.
- Treatment with NSAIDs above the maximum dose specified in the prescribing information within 2 weeks prior to screening.
- Systematic treatment within 12 weeks prior to screening with chemotherapy and/or other immunosuppressive drugs.
- Treatment with an investigational product within 30 days or 5.5 half-lives prior to screening, whichever is longer.
- Major surgery within 12 weeks prior to screening or planned major orthopaedic surgery to occur during the study.
- At baseline visit, patients who have not been compliant in using the activity tracker.
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.