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
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
- Age range
- 2+ years
- Sex
- All
- Healthy volunteers
- No
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 w…
Interventions
- DrugVon Willebrand Factor Concentrates
Used according to local labeling or local treatment guidelines.
- DrugVon Willebrand Factor Concentrates and Factor VIII Concentrates
Used according to local labeling or local treatment guidelines.
- DrugFactor VIII Concentrates
Used according to local labeling or local treatment guidelines.
- DrugRecombinant Activated Factor VII
Used according to local labeling or local treatment guidelines.
- DrugActivated Prothrombin Complex Concentrate
Used according to local labeling or local treatment guidelines.
Locations (27)
- UC DavisSacramento, California
- University of FloridaGainesville, Florida
- University of Minnesota Medical CenterMinneapolis, Minnesota
- Washington University School of MedicineSt Louis, Missouri
- UZ Leuven GasthuisbergLeuven
- The Hospital for Sick ChildrenToronto, Ontario