A First-in Human Study of ARD001 in Healthy Volunteers and People With Hemophilia A
Study Details
- Hemophilia A
- Unmapped
- Biological: ARD001
- Phase 1 Inclusion Criteria:
- Healthy adults within the specified age and body-weight ranges
- Able and willing to provide informed consent
- Considered healthy based on medical history, physical examination, vital signs, electrocardiogram and laboratory assessments
- Other essential criteria from the approved protocol
- Phase 1 Exclusion Criteria:
- History of severe drug or food allergy, or known allergy to ARD001 or its components
- Acute infection within 14 days before dosing or fever on the planned dosing day
- Clinically significant cardiovascular, coagulation, hepatic, renal or other medical abnormality
- Recent use of prohibited medications within the protocol-defined washout periods
- Other major exclusions from the approved protocol
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.