SAFE Study: Safety of aPCC Following Emicizumab Prophylaxis
Study Details
- Hemophilia A
- Drug: Emicizumab
- Drug: FEIBA
- Drug: rFVIIa
- Moderately severe hemophilia A, defined as FVIII level \<0.05 IU/mL before development of an inhibitor
- Age ≥6 years of age at time of informed consent
- Documented on 2 occasions a high titer inhibitor (\>5 BU/mL) with a 72-hour washout within 2 years of enrollment
- Parent/guardian (Legally Authorized Representative) or the patient has provided written informed consent
- Adequate hematologic function (Hgb \>8 g/dL and platelet count \>100,000 µL)
- Adequate hepatic function (total bilirubin ≤1.5 x ULN and both AST/ALT ≤3x ULN at screening (excluding known Gilbert's)
- Adequate renal function (≤2.5 x ULN and CrCl ≥30 mL/min)
- Inherited or acquired bleeding disorder other than hemophilia A excluding low VWF (\>30% VWF:RCo or VWF:GP1bm)
- Had an active bleed requiring factor therapy at screening
- Previous or current treatment for thromboembolic disease or signs of thromboembolic disease (excluding previously resolved line-associated thrombosis)
- Had a surgical procedure 14 days before screening
- Conditions that may increase the risk of bleeding or thrombosis
- If the patient is treated with rFVIIa or aPCC seven days before screening
- History of clinically significant hypersensitivity associated with monoclonal antibody therapies or components of the emicizumab injection
- Had current use of any medication other than emicizumab that could affect the coagulation system.
- Known HIV infection with CD4 count \<200 cells/µL within 24 weeks before screening. Testing is not required if \<35 years of age.
- Use of systemic immunomodulators at enrollment or planned use during the study
- Participants who are at high risk for TMA (for example, have a previous medical/family history of TMA), in the investigator's judgment
- Concurrent disease, treatment, or abnormality in clinical laboratory tests that could interfere with the conduct of the study, may pose an additional risk, or would, in the opinion of the investigator, preclude the participant's safe participation in and completion of 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.