Von Willebrand Disease, Type 3
Conditions
Brief 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.
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.
Used according to local labeling or local treatment guidelines.
Used according to local labeling or local treatment guidelines.
DRUGActivated Prothrombin Complex Concentrate
Used according to local labeling or local treatment guidelines.
Sponsors
Hoffmann-La Roche
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
2 Years to No maximum
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 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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Annualized Bleed Rate (ABR) for Treated Bleeds | From Baseline to at least 24 weeks |
Secondary
| Measure | Time frame |
|---|---|
| ABR for All Bleeds | From Baseline to at least 24 weeks |
| ABR for Treated Spontaneous Bleeds | From Baseline to at least 24 weeks |
| ABR for Treated Joint Bleeds | From Baseline to at least 24 weeks |
| Incidence and Severity of Adverse Events, with Severity Determined According to the World Health Organization (WHO) Toxicity Grading Scale | From Baseline until study completion (at least 24 weeks) |
Countries
Belgium, Canada, Colombia, France, Germany, Italy, Japan, Netherlands, Poland, South Africa, Spain, Sweden, United Kingdom, United States
Contacts
CONTACTReference Study ID Number: WP45335 https://forpatients.roche.com/
STUDY_DIRECTORClinical Trials
Hoffmann-La Roche
Outcome results
None listed