The purpose of this screening study is to accumulate information regarding bleeding events, quality of life, and the social and clinical impact of bleeds in participants with Von Willebrand Disease (VWD). Data from this study will be used to establish baseline bleeding and treatment rates in a population of participants with VWD and act as comparator data for future clinical study outcomes.(e.g. Velora Pioneer)
Has the ability to provide informed consent to participate in the study, in accordance with applicable regulations.
Has an understanding, ability, and willingness to comply with Study procedures and restrictions.
Is 16 years and < 70 years at the time of screening.
Weight 50 to 120 kg (±10%) at Screening and body mass index (BMI) <38.5 kg/m*2.
Has a history of clinically significant hypersensitivity associated with monoclonal antibody therapies.
Has a personal history of venous or arterial thrombosis or thromboembolic disease, except for catheter-associated, superficial vein thrombosis events.
Has a high-risk thrombophilia: Homozygous Factor V Leiden (FVL), compound heterozygous FVL/prothrombin gene mutation, antithrombin <50%, congenital protein C and protein S deficiency with levels <50%.
Requires ongoing hemostatic (bleed-prophylaxis) treatment to prevent bleeding
Hemab ApS
Lead sponsor