An Open-Label, Comparative Study of the Efficacy, Safety and Pharmacodynamics of Single Dose of ANB-002 in Patients With Hemophilia B
Study Details
- Hemophilia B
- Inclusion Criteria:
- Men diagnosed with hemophilia B aged 18 or older
- FIX activity ≤2%
- Absense of FIX inhibitor
- ≥150 previous exposure days of treatment with FIX concentrates
- Exclusion Criteria:
- Any diseases of blood and hematopoietic organs other than hemophilia B
- A history of any gene therapy, including ANB-002
- Diagnosed HIV-infection, not controlled with anti-viral therapy
- Active HBV or HCV infection
- Anti-AAV5 antibodies
- Any active systemic infections or recurrent infections requiring systemic therapy
- Any other disorders associated with severe immunodeficiency
- Relevant hepatic disorders or conditions that can be a symptom of existing liver disorder
- Malignancies with less than 5 years of remission
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.