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

Participation Requirements

• Sex:

  ALL

• Eligible Ages:

  2 and up

Participation Criteria

Inclusion Criteria:

* Confirmed diagnosis of Type 3 von Willebrand disease (VWD), based on medical records
* Adequate hematologic, hepatic, and renal function
* Documented and confirmed previous use of SOC prophylactic therapy for VWD (1-3 times weekly, as per prescribed dose) and anticipation to remain on the same regimen during the study
* For participants of childbearing potential: agreement to remain abstinent or adhere to the contraception requirements

Exclusion Criteria:

* Inherited or acquired bleeding disorder other than Congenital Type 3 VWD
* History of gastrointestinal bleeding within 18 months prior to enrollment, or any previous diagnosis of angiodysplasia
* History of intracranial hemorrhage
* Previous or current treatment for thromboembolic disease or signs of thromboembolic disease
* Other conditions (e.g., certain autoimmune diseases) that may increase risk of bleeding or thrombosis
* History of clinically significant hypersensitivity associated with monoclonal antibody therapies or components of the emicizumab injection
* Use of systemic immunomodulators (e.g., interferon) at enrollment or planned use during the study, with the exception of anti-retroviral therapy