Open-Label Extension Study of Marstacimab in Hemophilia Participants With or Without Inhibitors
Study Details
- Hemophilia A
- Hemophilia B
- Drug: PF-06741086
- Inclusion Criteria:
- All participants will have a minimum body weight as defined by parent studies
- Participants who are willing and able to comply with all scheduled visits, treatment plan, laboratory tests, and other study procedures.
- Participants have successfully completed participation in parent studies, defined as did not require "Early Termination"
- Exclusion Criteria:
- Previous or current treatment for or history of coronary artery disease, venous or arterial thrombosis (CTCAE Grade \>3), or ischemic disease (except catheter-associated thrombosis)
- Abnormal renal function as defined by eGFR \<30 mL.min/1.73 m(2)
- Known planned surgical procedure during the planned study period
- Unstable hepatic function as determined by the Investigator clinical assessment and review of the participant's most recent laboratory results, which would make the participant inappropriate for the study
- For participants known to be HIV+, worsening disease status as determined by the Investigator clinical assessment and review of participant's most recent laboratory results, to include recent locally available CD4 count (if available), which would make the participant inappropriate for the study
- Regular, concomitant therapy with immunomodulatory drugs (eg, IVIG, and routine systemic corticosteroids, rituximab)
- Ongoing or planned use of immune tolerance induction or prophylaxis with FVIII or FIX replacement during the study
- Participation in other study involving investigational drug(s) or investigational vaccine(s) within 30 days or 5 half-lives prior to or during study participation, with the exception of participation in parent studies
- Investigator site staff or Pfizer employees directly involved in the conduct of the study, site staff otherwise supervised by the Investigator, and their respective family members
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.