An Observational Study of Participants With Type 3 Von Willebrand Disease on Prophylactic Standard-of-Care Treatment
A Multicenter Non-Interventional Study Evaluating Bleeds and Health-Related Quality Of Life in Patients With Type 3 Von Willebrand Disease on Prophylactic Standard-of-Care Treatment
Hoffmann-La Roche
40 participants
Apr 29, 2025
OBSERVATIONAL
Conditions
Summary
This non-interventional study (NIS) is designed to collect information on the effectiveness and safety of treatment received in routine clinical care, as well as measure the health-related quality of life (HRQoL) of participants with Type 3 von Willebrand disease (VWD) receiving prophylactic therapy per local standard of care (SOC) over an observation period of at least 24 weeks.
Eligibility
Inclusion Criteria4
- 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 Criteria7
- 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
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Interventions
Used according to local labeling or local treatment guidelines.
Used according to local labeling or local treatment guidelines.
Used according to local labeling or local treatment guidelines.
Used according to local labeling or local treatment guidelines.
Used according to local labeling or local treatment guidelines.
Locations(27)
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NCT06883240