A Study to Learn How Safe the Study Treatment BAY94-9027 is and How it Affects the Body in Previously Treated Children Aged 7 to Less Than 12 Years With Severe Hemophilia A, a Genetic Bleeding Disorder That is Caused by the Lack of a Protein Called Clotting Factor 8 (FVIII) in the Blood
Study Details
- Ignored
- Hemophilia
- Hemophilia A
- Biological: Damoctocog alfa pegol (Jivi, BAY94-9027)
- Inclusion Criteria:
- Participants with severe hemophilia A (participant's own FVIII activity \[FVIII:C\] \<1%)
- Participants must be previously treated with FVIII concentrate(s) (plasma derived or recombinant) for a minimum of 50 exposure days (EDs) at the time of signing the informed consent
- Participant has understood the study if appropriate for his age, informed consent must be signed by the parent, the participant can only sign the assent
- Willingness and ability of participants and/or parents /caregivers to complete training in the use of the electronic patient diary (EPD) and to document infusions during the study
- Exclusion Criteria:
- History of FVIII inhibitors
- Current evidence of inhibitor to FVIII measured using the Nijmegen-modified Bethesda assay (≥0.6 BU/mL) at the time of screening (central laboratory)
- Any other inherited or acquired bleeding disorder in addition to hemophilia A (e.g. von Willebrand disease, hemophilia B)
- Known hypersensitivity or allergic reaction to drug substance, excipients or mouse or hamster protein
- Any other significant medical condition that the investigator feels would be a risk to the patient or would impede the study
- Requires any pre-medication to tolerate FVIII treatment (e.g. antihistamines)
- Planned major surgery during the study
- Any individual who is receiving chemotherapy, immune modulatory drugs other than anti-retroviral chemotherapy, or chronic use of oral or intravenous (IV) corticosteroids (\> 14 days) within the last 3 months
- Any individual who received commercially available subcutaneous factor substitution therapy (emicizumab) within the last 6 months
- The participant is currently participating in another investigational drug study or has participated in a clinical study involving an investigational drug within 30 days of study entry or previous participation in a clinical study with BAY94-9027
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.