A Phase 3 safety study of BAY 94-9027 in children 7 to <12 years of age with severe hemophilia A

Prior treatment

• Planned major surgery during the study
• Any individual who is currently receiving or received 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 BAY 94-9027.
• Requires any pre-medication to tolerate FVIII treatment (e.g. antihistamines)

Other illness

• "History of FVIII inhibitors. Inhibitor to FVIII is defined as a titer >0.6 BU/mL or clinical history suggestive of inhibitor requiring modification of treatment. Participants with a maximum historical titer of <1.0 BU on no more than 1 occasion with the classical Bethesda assay but at least 3 subsequent negative results [<0.6 BU] are eligible."
• 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 participant or would impede the study

Lab values

• Current evidence of inhibitor to FVIII measured using the Nijmegen modified Bethesda assay (>0.6 BU/mL) at the time of screening (central laboratory). Participants should not receive FVIII within 48 h prior to the collection of screening samples and should have FVIII administered within the prior 2-3 weeks
• Platelet count <100,000 cells/μL
• Serum creatinine > 2x upper limit of normal
• Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) > 5x upper limit of normal

• Participant must be 7 to <12 years of age at the time of parent / guardian signing the informed consent.
• Participants with known medical history of severe hemophilia A (participant's own FVIII activity [FVIII:C] <1%. FVIII:C) based on reliable prior documentation in clinical records of the participants. If no reliable documentation is available, FVIII activity must be measured at the time of screening after a 48-72 hours wash-out period (depending on his previous product).
• Male
• 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 and if appropriate for his age, has signed an informed assent. The parent(s) or guardian(s) is capable of giving signed informed consent and are able to comply with the requirements and restrictions listed in the informed consent form (ICF) and the protocol.
• 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.