Emicizumab for Severe Von Willebrand Disease (VWD) and VWD/Hemophilia A
Emicizumab for Severe VON Willebrand Disease (VWD) and VWD/Hemophilia A
Bleeding and Clotting Disorders Institute Peoria, Illinois
40 participants
Nov 1, 2022
INTERVENTIONAL
Conditions
Summary
Von Willebrand Disease (VWD) is the most common inherited bleeding disorder affecting up to 0.1% of the population, is usually characterized by mucocutaneous bleeding, HMB, surgical bleeding or other hemostatic challenges. Severe bleeding events require VWF concentrates administered solely through intravenous access. Emicizumab (Hemlibra) is a monoclonal bispecific antibody developed to bind activated FIX and FX and mimic FVIII cofactor functionality. Hemlibra is administered via subcutaneous injection rather than intravenous infusion. The hypothesis of this study is that Emicizumab is safe and efficacious for prophylaxis in severe VWD and concomitant VWD/hemophilia patients.
Eligibility
Plain Language Summary
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Interventions
Subcutaneous injection of emicizumab for prophylaxis
Locations(12)
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NCT05500807