An Observational Study of Participants With Type 3 Von Willebrand Disease on Prophylactic Standard-of-Care Treatment (WILL-EMI NIS)

Roche

Status: Enrolling

Conditions: Von Willebrand Disease, Type 3

Treatments: Drug: Von Willebrand Factor Concentrates and Factor VIII Concentrates; Drug: Activated Prothrombin Complex Concentrate; Drug: Factor VIII Concentrates; Drug: Von Willebrand Factor Concentrates; Drug: Recombinant Activated Factor VII

Study type: Observational

Funder types: Industry

Identifiers

NCT06883240

WP45335

Details and patient eligibility

About

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.

Enrollment: 40 estimated patients

Sex: All

Ages: 2+ years old

Volunteers: No Healthy Volunteers

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

Trial design

40 participants in 1 patient group

Participants with Type 3 VWD Treated with Prophylactic SOC

Description:

Participants with Type 3 VWD, aged 2 years and above, who are currently on standard of care (SOC) prophylactic therapy are anticipated to remain on the chosen SOC regimen during the study. Dosing and treatment duration of any studied medicinal products collected as part of this study are at the discretion of the treating physician in accordance with local labeling or local treatment guidelines.

Treatment:

Drug: Recombinant Activated Factor VII

Drug: Von Willebrand Factor Concentrates

Drug: Factor VIII Concentrates

Drug: Activated Prothrombin Complex Concentrate

Drug: Von Willebrand Factor Concentrates and Factor VIII Concentrates