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Clinical Trials in the UK / NCT06883240
Active, not recruiting Observational

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

NCT06883240 · tracked via the Priya Life Science UK tracker
Sponsor
Hoffmann-La Roche
Phase
Observational
Started
2025-04-29
Last updated
2026-08-10

Condition(s) studied

Von Willebrand Disease, Type 3

Investigational drug(s) / intervention(s)

Von Willebrand Factor ConcentratesVon Willebrand Factor Concentrates and Factor VIII ConcentratesFactor VIII ConcentratesRecombinant Activated Factor VIIActivated Prothrombin Complex Concentrate

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.

Study 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.

Eligibility

Sex
ALL
Min age
2 Years
Max age
Healthy volunteers
No
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

Primary outcome measure(s)

Trial sites (26)

FacilityCityRegionStatus
UC Davis Sacramento California
University of Minnesota Medical Center Minneapolis Minnesota
Washington University School of Medicine St Louis Missouri
UZ Leuven Gasthuisberg Leuven Belgium
The Hospital for Sick Children Toronto Ontario
McGill University Health Center Montreal Quebec
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
IRCCS Ca' Granda Ospedale Maggiore Policlinico Milan Lombardy
AOU Careggi Florence Tuscany
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
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06883240 on ClinicalTrials.gov ↗ ← All trials in the UK