Emicizumab for Severe Von Willebrand Disease (VWD) and VWD/Hemophilia A
Status:
Not yet recruiting
Trial end date:
2026-03-01
Target enrollment:
Participant gender:
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.
Phase:
Phase 1
Details
Lead Sponsor:
Bleeding and Clotting Disorders Institute Peoria, Illinois