An Observational Study of Participants With Type 3 Von Willebrand Disease on Prophylactic Standard-of-Care Treatment

To join this study, you must have a confirmed diagnosis of severe Type 3 Von Willebrand Disease. You also need to be receiving your usual, regular treatment (called 'prophylactic therapy') for your condition, typically taking it 1 to 3 times a week, and expect to continue this treatment during the study period. Your blood, liver, and kidney functions should be generally healthy.

There are also some important reasons why you might not be able to join. For example, if you have another bleeding disorder, a recent history of stomach or bowel bleeding, or a history of bleeding in the brain, you wouldn't be suitable. People who have had blood clots or are being treated for them, or those with certain other medical conditions affecting bleeding or clotting, would also not be able to participate.

Additionally, if you are pregnant, planning to become pregnant, or are of childbearing age, you would need to agree to use effective contraception throughout the study. If you're taking certain strong immune-system affecting medications (like systemic immunomodulators), you also wouldn't be able to join, though some specific medications like HIV treatments are exceptions.

If you decide to take part, you will continue with your regular medical appointments and treatments as prescribed by your doctor. The study will mainly involve collecting information from your medical records and asking you about your health and quality of life through questionnaires. This will happen over at least 24 weeks (about 6 months).

You won't receive any new medications or treatments specially for the study. Instead, the researchers will observe your usual care. You'll likely have regular check-ups with the study team to record information, but these are typically planned around your existing appointments. There's no strict number of visits mentioned, as it follows your standard care.