SAFE Study: Safety of aPCC Following Emicizumab Prophylaxis

• Status: Not yet recruiting
• Trial end date: 2022-12-01
• Target enrollment: 0
• Participant gender: All

Summary

The purpose of the aPCC-emicizumab safety study is to prospectively investigate the safety and hemostatic efficacy of a personalized dose of aPCC in children and adults with hemophilia A and inhibitors on emicizumab prophylaxis during acute bleeding events or prior to procedures.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Emory University
• Collaborator: Takeda Pharmaceuticals North America, Inc.
• Treatments: Anti-inhibitor coagulant complex; Coagulants

Criteria

Inclusion Criteria:

• Moderately severe hemophilia A, defined as FVIII level <0.02 IU/mL in the central laboratory prior to development of an inhibitor

• Age ≥6 years of age at time of informed consent

• Documented on 2 occasions a high titer inhibitor (>5 BU/mL) with a 72-hour washout within 2 years of enrollment

• Parent/guardian (caregiver henceforth) or 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)

• Previous or current treatment for thromboembolic disease or signs of thromboembolic disease (excluding previous resolved line associated thrombosis)

• Conditions that may increase risk of bleeding or thrombosis

• History of clinically significant hypersensitivity associated with monoclonal antibody therapies or components of the emicizumab injection

• Known HIV infection with CD4 count <200 cells/µL within 24 weeks prior to screening. Testing not required if <35 years of age.

• Use of systemic immunomodulators at enrollment or planned use during the study

• Participants who are at high risk for TMA (for example, have a previous medical/family history of TMA), in the investigator's judgment

• Concurrent disease, treatment, or abnormality in clinical laboratory tests that could interfere with the conduct of the study, may pose additional risk, or would, in the opinion of the investigator, preclude the participant's safe participation in and completion of the study

• Every effort will be made to include participants that are considering minor and major procedures over the next 2 years to capture this important data with the goal of 10 procedures.