RECRUITING

An Observational Study of Participants With Type 3 Von Willebrand Disease on Prophylactic Standard-of-Care Treatment

Description

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.

Study Overview

Study Details

Study overview

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.

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

An Observational Study of Participants With Type 3 Von Willebrand Disease on Prophylactic Standard-of-Care Treatment

Condition
Von Willebrand Disease, Type 3
Intervention / Treatment

-

Contacts and Locations

Sacramento

UC Davis, Sacramento, California, United States, 95817

St Louis

Washington University School of Medicine, St Louis, Missouri, United States, 63110

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility 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
  • * 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

Ages Eligible for Study

2 Years to

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Hoffmann-La Roche,

Clinical Trials, STUDY_DIRECTOR, Hoffmann-La Roche

Study Record Dates

2026-11-01