SAFE Study: Safety of aPCC Following Emicizumab Prophylaxis

The purpose of the aPCC-emicizumab safety study is to investigate the hemostatic efficacy as measured by thrombin generation, of a low personalized dose of aPCC (FEIBA) in children and adults with hemophilia A and inhibitors on emicizumab prophylaxis.

Inclusion Criteria: * Moderately severe hemophilia A, defined as FVIII level \5 BU/mL) with a 72-hour washout within 2 years of enrollment * Parent/guardian (Legally Authorized Representative) or the patient has provided written informed consent * Adequate hematologic function (Hgb \>8 g/dL and platelet count \>100,000 µL) * Adequate hepatic function (total bilirubin ≤1.5 x ULN and both AST/ALT ≤3x ULN at screening (excluding known Gilbert's) * Adequate renal function (≤2.5 x ULN and CrCl ≥30 mL/min) Exclusion Criteria: * Inherited or acquired bleeding disorder other than hemophilia A excluding low VWF (\>30% VWF:RCo or VWF:GP1bm) * Had an active bleed requiring factor therapy at screening * Previous or current treatment for thromboembolic disease or signs of thromboembolic disease (excluding previously resolved line-associated thrombosis) * Had a surgical procedure 14 days before screening * Conditions that may increase the risk of bleeding or thrombosis * If the patient is treated with rFVIIa or aPCC seven days before screening * History of clinically significant hypersensitivity associated with monoclonal antibody therapies or components of the emicizumab injection * Had current use of any medication other than emicizumab that could affect the coagulation system. * Known HIV infection with CD4 count \