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

Running, not enrolling

Conditions studied: Von Willebrand Disease, Type 3

In brief

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.

Key facts

Study ID

NCT06883240

Run by

Hoffmann-La Roche

People needed

44

Starts

2025-04-29

Expected to finish

2026-11-01

Last updated by the study team

2026-07-10

Who can join

Age: 2 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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 may not qualify 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

Where it is running

• UC Davis — Sacramento, California, 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

Full record on ClinicalTrials.gov

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