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Recruiting NCT06883240

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

Conditions: Von Willebrand Disease, Type 3

Sponsor: Hoffmann-La Roche

trial.available_in: БГ
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.
Who can participate
Inclusion 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 Exclusion Criteria: * 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
Interventions
Von Willebrand Factor Concentrates
DRUG
Von Willebrand Factor Concentrates and Factor VIII Concentrates
DRUG
Factor VIII Concentrates
DRUG
Recombinant Activated Factor VII
DRUG
Activated Prothrombin Complex Concentrate
DRUG
Locations 27
Belgium (1)
UZ Leuven Gasthuisberg
Leuven
Canada (2)
McGill University Health Center
Montreal , Quebec
The Hospital for Sick Children
Toronto , Ontario
COMPLETED
Colombia (1)
IPS SURA Industriales Medellín
Medellín
France (2)
Hopital Claude Huriez - CHU Lille
Lille
Groupe Hospitalier Necker Enfants Malades
Paris
Germany (3)
Universitätsklinikum Bonn
Bonn
Gerinnungszentrum Rhein-Ruhr;Gerinnungsambulanz
Duisburg
Hämophiliezentrum Med. Klinik III/Institut für Transfusionsmedizin
Frankfurt/M.
Italy (3)
AOU Careggi
Florence , Tuscany
IRCCS Ca' Granda Ospedale Maggiore Policlinico
Milan , Lombardy
Universita' Degli Studi La Sapienza-Ist.Di Ematologia
Rome , Lazio
Japan (2)
Kurume University Hospital
Fukuoka
COMPLETED
Tokyo Medical University Hospital
Tokyo
Netherlands (1)
Erasmus MC
Rotterdam
Poland (1)
Instytut Hematologii i Transfuzjologii
Warsaw
South Africa (1)
Charlotte Maxeke Johannesburg Academic Hospital
Johannesburg
Spain (2)
Hospital Universitario la Paz
Madrid
Hospital Universtiario Virgen del Rocio
Seville
Sweden (1)
Sahlgrenska Universitetssjukhuset
Gothenburg
United Kingdom (3)
St Thomas' Hospital
London
Great Ormond Street Hospital
London
Manchester Royal Infirmary
Manchester
United States (4)
University of Florida
Gainesville , Florida
University of Minnesota Medical Center
Minneapolis , Minnesota
UC Davis
Sacramento , California
Washington University School of Medicine
St Louis , Missouri
Technical details
Status
Recruiting
Study type
OBSERVATIONAL
Sex
Male and female
Minimum age
2 Years
Healthy volunteers
No
Start date
29.04.2025
Completion date
01.11.2026
Registry ID
NCT06883240
Source
clinicaltrials.gov
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