A Study to Learn About How Changing Therapy From Emicizumab to Marstacimab Affects People With the Severe Hemophilia A.
Study Details
- Hemophilia A, Severe
- Drug: MARSTACIMAB
- Inclusion Criteria:
- Male and 12 to \<75 years of age with a minimum body weight of 35 kg at the time of signing the informed consent.
- Diagnosis of severe hemophilia A (FVIII activity \<1%) without inhibitors.
- On emicizumab therapy at a standard clinical dose for ≥6 months.
- Exclusion Criteria:
- Previous or current treatment for or history of coronary artery diseases, venous or arterial thrombosis, or ischemic disease.
- Any medical or psychiatric condition including recent (within the past year) or active suicidal ideation/behavior or laboratory abnormality that may increase the risk of study participation or, in the investigator's judgment, make the participant inappropriate for the study.
- Known hemostatic defect other than hemophilia A.
- Current use of any prohibited concomitant medication(s) or unwillingness or inability to use a required concomitant medication(s).
- Previous administration of an investigational product (drug or vaccine) within 30 days or 5 half-lives preceding the first dose of study intervention used in this study (whichever is longer). Participation in studies of other investigational products (drug or vaccine) at any time during participation in this study.
- Platelet count \<100,000/μl or hemoglobin \<10 g/dL.
- Clinically significant renal or hepatic function abnormality based on laboratory results at screening, or known kidney or liver disease.
- CD4 cell count ≤200/μl if HIV positive.
- Screening 12-lead ECG that demonstrates clinically significant abnormalities that, in the opinion of the investigator, may affect participant safety or interpretation of study results.
- Known planned surgical procedure.
- Hypersensitivity or allergic reaction to hamster protein or other components of the study intervention.
- Investigator site staff directly involved in the conduct of the study and their family members, site staff otherwise supervised by the investigator, and sponsor and sponsor-delegate employees directly involved in the conduct of the study and their family members.
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.