PCC Treatment for Hemophilia Patients With Inhibitor（2022PCC-A）
Study Details
- Hemophilia
- Hemophilia A With Inhibitor(s)
- Drug: PCC
- Diagnosed with hemophilia A with inhibitors. For high-responding patients (those who have previously had an inhibitor titer \>5 BU), the inhibitor titer at enrollment must be \>0.6 BU;
- Age between 12 and 65 years;
- At least three joint bleeding episodes within the past six months;
- Signed informed consent form.
- Presence of other congenital or acquired bleeding disorders;
- Liver function tests (ALT, AST) \>2.5 times the upper limit of normal, or renal function tests (BUN, Cr) \>1.5 times the upper limit of normal;
- Currently receiving immune tolerance induction (ITI) therapy with an inhibitor titer \<5 BU;
- History of thrombotic events;
- Known history of drug allergy, asthma, urticaria, or other allergic conditions;
- Deemed unsuitable for study participation by the investigator.
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.