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An Observational Study of Participants With Type 3 Von Willebrand Disease on Prophylactic Standard-of-Care Treatment
Sponsor: Hoffmann-La Roche
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
Key Details
Gender
All
Age Range
2 Years - Any
Study Type
OBSERVATIONAL
Enrollment
40
Start Date
2025-04-29
Completion Date
2026-11-01
Last Updated
2026-03-18
Healthy Volunteers
No
Conditions
Interventions
Von Willebrand Factor Concentrates
Used according to local labeling or local treatment guidelines.
Von Willebrand Factor Concentrates and Factor VIII Concentrates
Used according to local labeling or local treatment guidelines.
Factor VIII Concentrates
Used according to local labeling or local treatment guidelines.
Recombinant Activated Factor VII
Used according to local labeling or local treatment guidelines.
Activated Prothrombin Complex Concentrate
Used according to local labeling or local treatment guidelines.
Locations (27)
UC Davis
Sacramento, California, United States
University of Florida
Gainesville, Florida, United States
University of Minnesota Medical Center
Minneapolis, Minnesota, United States
Washington University School of Medicine
St Louis, Missouri, United States
UZ Leuven Gasthuisberg
Leuven, Belgium
The Hospital for Sick Children
Toronto, Ontario, Canada
McGill University Health Center
Montreal, Quebec, Canada
IPS SURA Industriales Medellín
Medellín, Colombia
Hopital Claude Huriez - CHU Lille
Lille, France
Groupe Hospitalier Necker Enfants Malades
Paris, France
Universitätsklinikum Bonn
Bonn, Germany
Gerinnungszentrum Rhein-Ruhr;Gerinnungsambulanz
Duisburg, Germany
Hämophiliezentrum Med. Klinik III/Institut für Transfusionsmedizin
Frankfurt/M., Germany
Universita' Degli Studi La Sapienza-Ist.Di Ematologia
Rome, Lazio, Italy
IRCCS Ca' Granda Ospedale Maggiore Policlinico
Milan, Lombardy, Italy
AOU Careggi
Florence, Tuscany, Italy
Kurume University Hospital
Fukuoka, Japan
Tokyo Medical University Hospital
Tokyo, Japan
Erasmus MC
Rotterdam, Netherlands
Instytut Hematologii i Transfuzjologii
Warsaw, Poland
Charlotte Maxeke Johannesburg Academic Hospital
Johannesburg, South Africa
Hospital Universitario la Paz
Madrid, Spain
Hospital Universtiario Virgen del Rocio
Seville, Spain
Sahlgrenska Universitetssjukhuset
Gothenburg, Sweden
St Thomas' Hospital
London, United Kingdom
Great Ormond Street Hospital
London, United Kingdom
Manchester Royal Infirmary
Manchester, United Kingdom