A Prospective, Screening Study of Bleeding and Treatment in Participants with Von Willebrand Disease

This is a non-interventional observational study in participants with confirmed Type 1 VWD according to diagnostic guidelines. The study includes screening, a baseline evaluation, and an approximately 4-month observation period which will include every other week telemedicine check-ins (to monitor bleed diary entries and bleeding event treatments.?

Trial phase

Other

Trial participation type

This trial is for clinical research of something other than a drug or device.

Principal investigator

Prof Huyen Tran

Key inclusion data

Inclusion: Has congenital Type 1 Von Wilebrand’s disease (consistent with British Society for Haematology [BSH]/ United Kingdom Haemophilia Centre Doctors' Organisation [UKHCDO] or International Society of Thrombosis and Hemostasis ISTH diagnostic criteria [ISTH]/ American Society of Hematology [ASH] diagnostic guidelines; Has symptomatic disease as defined by a history of bruising or bleeding events, typically experiencing bleeding symptoms every month, and either; Are expected to experience at least 3 bleeding episodes per year that need treatment Or have recurrent and ongoing heavy menstrual bleeding. Exclusion: Has a congenital or acquired bleeding disorder other than Von Willebrand’s disease; Has any concurrent disease, treatment, condition, medication, or abnormality in clinical laboratory tests which may impact on the participant’s bleeding symptoms or affect their ability to complete the study.