Version Testing of EnzySystem Version A for Hemophilia A
Study Details
- Hemophilia A
- Inclusion Criteria:
- Healthy volunteers:
- Age between 20 to 70 years old (equally distributed over the age range)
- 7 volunteers 20-40 years old
- 7 volunteers 40-60 years old
- 6 volunteers 60+ years old
- Hemophilia A patients:
- Diagnosed with mild (FVIII activity levels 5-40%), moderate (FVIII activity levels 1-5%) or severe hemophilia A (FVIII activity levels \<1%)
- Medication
- On demand treatment
- Washout of medication of at least 24 hours after treatment with short half life (SHL) replacement therapy
- Washout of medication of at least 72 hours after treatment with extended half life (EHL) replacement therapy
- Age 20-70 years old
- Exclusion Criteria:
- A healthy volunteer who meets any of the following criteria will be excluded from participation in this study:
- use of anticoagulants or platelet antagonists (aspirin or any TAR);
- known allergy to stainless steel;
- trauma or surgery within the last two weeks;
- pregnancy;
- use of:
- NSAIDs;
- antimicrobial medication;
- thyroid inhibitors; or SSRI's. A hemophilia A patient who meets any of the following criteria will be excluded from participation in this study:
- use of anticoagulants or platelet antagonists (aspirin or any TAR);
- known allergy to stainless steel;
- trauma or surgery within the last two weeks;
- a bleeding episode within the last two weeks;
- clinical indication of liver cirrhosis (echographic indication, enlarged spleen, decreased platelet count);
- pregnancy;
- FVIII inhibitors;
- Signs of inflammation or infection
- use of:
- NSAIDs;
- antimicrobial medication;
- thyroid inhibitors or SSRI's;
- Emicizumab.
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.