A Research Study Investigating Mim8 in People With Haemophilia A
Study Details
- Unmapped
- Hemophilia A
- Drug: NNC0365-3769 (Mim8)
- Drug: Placebo (Mim8)
- Inclusion Criteria:
- Single ascending dose part 1:
- Male, aged 18-45 years (both inclusive) at the time of signing informed consent
- Considered to be generally healthy based on the medical history, physical examination, and the results of vital signs, electrocardiogram and clinical laboratory tests performed during the screening visit, as judged by the investigator
- Multiple ascending dose part 2:
- Male, aged 12-64 years (both inclusive) at the time of signing informed consent (Germany and Japan have local requirements)
- Diagnosis of congenital haemophilia A with FVIII activity below 1% based on medical records
- Exploratory biomarker cohort:
- Male, aged equal to or above 12 years at the time of signing informed consent (Germany and Japan have local requirements)
- Diagnosis of congenital haemophilia A with FVIII activity below 1% based on medical recordsv
- Exclusion Criteria:
- Part 1:
- Factor VIII activity equal to or above 150% at screening
- Increased risk of thrombosis, e.g. known history of personal or first degree relative(s) with unprovoked deep vein thrombosis
- Any clinical signs or established diagnosis of venous or arterial thromboembolic disease
- Part 2:
- Known congenital or acquired coagulation disorders other than haemophilia A
- Increased risk of thrombosis as evaluated by the investigator. E.g. known history of personal or first degree relative(s) with unprovoked deep vein thrombosis with exception of previous catheter-associated thrombosis for which anti-thrombotic treatment is not currently ongoing
- Any clinical signs or established diagnosis of venous or arterial thromboembolic disease with exception of previous catheter-associated thrombosis for which anti-thrombotic treatment is not currently ongoing
- Advanced atherosclerotic disease (e.g. known history of ischemic heart disease, ischemic stroke) as evaluated by the investigator
- Any autoimmune disease that may increase the risk of thrombosis
- Receipt of emicizumab or drugs with similar modes of action within 5 half-lives before trial product administration
- Ongoing or planned immune tolerance induction therapy
- Exploratory biomarker cohort:
- Known congenital or acquired coagulation disorders other than haemophilia A
- Increased risk of thrombosis as evaluated by the investigator. E.g. known history of personal or first degree relative(s) with unprovoked deep vein thrombosis with exception of previous catheter-associated thrombosis for which anti-thrombotic treatment is not currently ongoing
- Any clinical signs or established diagnosis of venous or arterial thromboembolic disease with exception of previous catheter-associated thrombosis for which anti-thrombotic treatment is not currently ongoing
- Advanced atherosclerotic disease (e.g. known history of ischemic heart disease, ischemic stroke) as evaluated by the investigator
- Any autoimmune disease that may increase the risk of thrombosis
- Ongoing or planned immune tolerance induction therapy
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.