Efficacy and Safety of KN057 Prophylaxis in Patients With Haemophilia A or B With Inhibitors
Study Details
- Hemophilia A With Inhibitor(s)
- Hemophilia B With Inhibitor(s)
- Drug: KN057
- Inclusion Criteria:
- Male, 12 to 70 years old at the time of signing informed consent (including the cut-off value), body weight ≥25 kg and BMI \<28 kg/m\^2 at screening;
- The FVIII or FIX inhibitor test is positive at a high titer (≥5 BU/ml) during the screening period; or the FVIII or FIX inhibitor is detected at a low titer (0.6 BU/ml or the upper limit of normal value \< inhibitor titer \< 5 BU/ml) during the screening period and treatment with bypass agents (rFVIIa or PCC) has been started;
- ≥6 treated bleeding episodes within 26 weeks before screening;
- Have not used TFPI antibody drugs before;
- Be able and agree to elute prior drugs for the treatment of hemophilia.
- Exclusion Criteria:
- Have serious or poorly controlled chronic diseases or obvious systemic diseases;
- Have a history of thromboembolic disease, or currently have symptoms or signs related to thromboembolic disease or being treated with thrombolytic/antithrombotic therapy;
- Have high-risk factors for thrombosis: such as a history of coronary atherosclerotic disease, ischemic disease of important organs, vascular occlusive disease, autoimmune diseases with a high risk of thrombosis, or indwelling central venous catheter;
- The presence of other inherited or acquired bleeding disorders other than hemophilia A and hemophilia B;
- Being on standard prophylaxis and maintaining it for more than 12 weeks (standard prophylaxis is defined as at least 80% compliance with a predetermined prophylaxis regimen);
- Ongoing or planned Immune Tolerance Induction treatment;
- When bleeding occurred in the past, rFVIIa was ineffective and PCC treatment must be used;
- Known or suspected hypersensitivity to any constituent of the trial product or related products;
- Have undergone major surgery (as determined by the investigator) within 3 months before screening, or have elective surgery planned during the study.
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.