Safety and Efficacy of STSP-0601 in Adult Patients With Hemophilia A or B With Inhibitor
Study Details
- Hemophilia
- Drug: STSP-0601 for Injection
- Inclusion Criteria:
- 18 ≤age≤70 years of age,male.
- Hemophilia A or B patients. (No less than 3 patients with hemophilia B)
- Peak historical inhibitor titer ≥ 5 BU and apositive inhibitor test when enrolled.
- Establish proper venous access.
- There were at least 3 bleeding events that requiring treatment occurred in the past 6 months before screening (Only applicable to the on-demand treatment stage).
- Agree to use adequate contraception to avoid pregnancy. Agree not to donate sperm or eggs.
- Provide signed informed consent.
- Exclusion Criteria:
- Have any coagulation disorder other than hemophilia.
- Plan to receive prophylactic treatment of coagulation factor during the trail.
- Patients plan to receive Emicizumab during the trial.
- Patients received anticoagulant or antifibrinolytic therapy 7 days before enrollment or plan to receive these drugs during the trial.Patients received anticoagulation therapy (such as coagulation factor replacement therapy, prothrombin complex, plasma, etc.) 7 days before enrollment.
- Have a history of arterial and/or venous thrombotic events.
- Platelet \<100×109/L.
- Hemoglobin\<90g/L.
- Severe liver or kidney disease.
- Severe bleeding event occurred within 4 weeks before enrollment.
- Accepted major operation or blood transfusion within 4 weeks before enrollment.
- Have a known allergy to STSP-0601.
- Pregnant, lactating, or blood pregnancy test positive female subjects
- Participate in other clinical research within 4 weeks before enrollment(except for participating in prothrombin complex, FVII, FVIIa, FVIII, FIX trails).
- Within 1 day prior to enrollment, FVII, FVIIa, tranexamic acid, and aminocaproic acid were used. Within 3 days prior to enrollment, prothrombin complex, FVIII, and FIX were used. Within 4 weeks prior to enrollment, treatment with amisulumab was received.
- Patients not suitable for the trail according to the judgment of the investigators.
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.