This is a phase 3 study that will evaluate subcutaneous (SC) VGA039 in patients with von Willebrand Disease (VWD)
12 to 75 years of age, inclusive
No clinically significant laboratory, ECG, or vital signs results
Documented diagnosis consistent with VWD of any type
Historical annualized bleeding rate (ABR; excluding menstrual bleeds and bleeds under the skin) of both untreated and treated bleeds ≥12 per year
Use of routine prophylaxis of VWF-containing concentrates defined as at least 1 VWF-containing concentrate infusion to prevent or reduce bleeding per week during the previous 6 months prior to screening
Planning to initiate routine prophylaxis with VWF-containing concentrates or any other hemostatic treatment during the study
Patients with pro-thrombotic disorders or abnormal findings on laboratory thrombophilia evaluation performed at screening or previously documented
History of arterial or venous thrombosis, including superficial thrombophlebitis, or embolism
Sequential
VGA039
DrugVGA039 SC administered for 49-weeks during the interventional phase following a 24-week observational period.
Incidence of bleeding events [Efficacy]
Incidence of treated bleeding events [Efficacy]
Incidence, nature and severity of adverse events (AEs) and serious adverse events (SAEs), including hypersensitivity, anaphylaxis, or anaphylactoid reactions
Plasma concentrations of SC doses of VGA039
Incidence of Anti-drug antibodies to VGA039
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