A Study of Bleeding and Treatment in Participants With Von Willebrand Disease
Start Date
8/30/2024
Completion Date
12/1/2026
Summary
The purpose of this screening study is to accumulate information regarding bleeding events, quality of life, and the social and clinical impact of bleeds in participants with Von Willebrand Disease (VWD). Data from this study will be used to establish baseline bleeding and treatment rates in a population of participants with VWD and act as comparator data for future clinical study outcomes.(e.g. Velora Pioneer)
Detailed Description
This is a prospective, screening study in participants with confirmed Type 1 VWD according to diagnostic guidelines. Participants with confirmed Type 1 VWD and associated bleeding symptoms will be enrolled. The study may also be opened to participants with Type 2 and Type 3 VWD with Sponsor approval. Up to a total of 200 participants may be enrolled in the study. The study includes screening, a baseline evaluation, and an approximately 4 month observation period which will include every other week telemedicine check-ins (to monitor bleed diary entries and bleeding event treatments. There will be an optional extension to the observation period of up to a total of 12 months for participants wishing to continue.
Eligibility Criteria
Age Range: 16 years to No maximum
Interventions
Clinical outcomes of patients with VWD, Type 1
Clinical outcomes of patients with VWD, Type 2A, Type 2M, Type 2N, or Type 3
Conditions
Locations
Phoenix Children's Hospital
Phoenix, Arizona 85016
United States
Arkansas Children's Hospital
Little Rock, Arkansas 72202-3591
United States
Children's Hospital of Los Angeles
Los Angeles, California 90027
United States
University of Miami Hospital and Clinics, Sylvester Comprehensive Cancer Center
Miami, Florida 33136
United States
Emory Children's Center
Atlanta, Georgia 30329
United States
Innovative Hematology, Inc./Indiana Hemophilia and Thrombosis Center
Indianapolis, Indiana 46260
United States
Tulane University School of Medicine
New Orleans, Louisiana 70112-2699
United States
University of Michigan Hospitals, Department of Hemophilia and Coagulation Disorders
Ann Arbor, Michigan 48109
United States
Mayo Clinic - Rochester
Rochester, Minnesota 55905
United States
Oregon Health & Science University
Portland, Oregon 97239-3098
United States
Hemophilia Center of Western Pennsylvania
Pittsburgh, Pennsylvania 15213
United States
The University of Texas Southwestern Medical Center
Dallas, Texas 75390
United States
Washington Institute For Coagulation (WIC)
Seattle, Washington 98101
United States
Fiona Stanley Hospital
Murdoch, Perth WA 6150
Australia
Royal Prince Alfred Hospital
Camperdown, Sydney NSW 2050
Australia
The Alfred Hospital
Melbourne, Victoria VIC 3004
Australia
Richmond Pharmacology
London, SE1 1YR
United Kingdom