Phase 3 study of the efficacy, pharmacokinetics, immunogenicity and safety of von Willebrand Factor/Factor VIII concentrate in patients with severe von Willebrand Disease under 6 years of age

Abstract

Introduction Von Willebrand disease (VWD) is a common congenital haemorrhagic disorder affecting around 1 in every 100 individuals. Prophylactic von Willebrand factor (VWF) replacement therapy in patients with severe VWD, including paediatric patients, is associated with reduced mucosal and joint bleeding rates, decreased median annual bleeding rate (ABR), a reduced incidence of major bleeding events (BEs) and good tolerability. WIL-33 will investigate the efficacy, pharmacokinetics (PK) and safety of a plasma-derived, stable, highly purified, double virus inactivated VWF/factor VIII (FVIII) concentrate (Octapharma) in paediatric patients with severe VWD.