A Trial of the Safety and Efficacy of Single-Dose Administration of ANB-010 in Subjects With Hemophilia A
Study Details
- Hemophilia A
- Inclusion Criteria:
- 1\. Male subjects aged ≥18 years at the time of signing the informed consent form.
- 3\. Established diagnosis of hemophilia A with a documented history of endogenous FVIII activity ≤1% AND ≤2% at screening.
- 4\. Therapy with FVIII concentrates for at least 150 exposure days.
- Exclusion Criteria:
- History of use of any gene therapy product.
- Use of emicizumab within less than 6 months before the date of signing the ICF.
- The presence of other blood or hematopoietic disorders other than hemophilia A.
- Presence of AAV6 antibodies detected by ELISA.
- BMI \<16 kg/m² or ≥35 kg/m².
- Diagnosis of HIV infection.
- HBV infection.
- HCV infection.
- Any active systemic infections or recurrent infections requiring systemic therapy at screening.
- Any other disorders associated with severe immunodeficiency.
- Relevant hepatic disorders or conditions that can be a symptom of existing liver disorder.
- Malignancies with remission duration of less than 5 years at the time of signing the ICF, except for cured basal cell carcinoma.
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.