Summary

Eligibility
for people ages 2 years and up (full criteria)
Location
at UC Davis
Dates
study started
study ends around

Description

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.

Official Title

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

Keywords

Von Willebrand Disease, Type 3, Type 3 von Willebrand Disease, Von Willebrand Factor Concentrates, Von Willebrand Factor Concentrates and Factor VIII Concentrates, Factor VIII Concentrates, Recombinant Activated Factor VII, Activated Prothrombin Complex Concentrate

Eligibility

You can join if…

Open to people ages 2 years and up

  • 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

You CAN'T join if...

  • 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

Locations

  • UC Davis accepting new patients
    Sacramento 5389489 California 5332921 95817 United States
  • Washington University School of Medicine accepting new patients
    St Louis 4407066 Missouri 4398678 63110 United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Hoffmann-La Roche
ID
NCT06883240
Study Type
Observational
Participants
Expecting 40 study participants
Last Updated