Actively Recruiting
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
Led by Hoffmann-La Roche · Updated on 2026-05-15
40
Participants Needed
27
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are observing people with Type 3 von Willebrand disease (VWD) who are receiving preventive treatment as part of their regular care. This study aims to gather information on how well these treatments work and how safe they are, while also measuring participants' quality of life related to their health. The study is non-interventional, meaning it does not change the treatments participants receive but monitors outcomes over time. Participants aged 2 years and older who are already on standard preventive therapy for Type 3 VWD will continue their usual treatment as decided by their doctors. The study collects details about the doses and duration of Von Willebrand Factor concentrates, Factor VIII concentrates, recombinant activated Factor VII, and activated prothrombin complex concentrate used, following local treatment guidelines. Participants are expected to maintain their current treatment regimen throughout the observation period of at least 24 weeks. During the study, researchers will track bleeding rates, including treated and spontaneous bleeds, especially joint bleeds, and any adverse events according to World Health Organization standards. Health-related quality of life will also be assessed. Data collection will occur over a minimum of 24 weeks, allowing for comprehensive monitoring of treatment effectiveness and safety. Participants remain under their usual care, and no changes to their treatment are required by the study.
CONDITIONS
Brief Title
An Observational Study of Participants With Type 3 Von Willebrand Disease on Prophylactic Standard-of-Care Treatment
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Confirmed diagnosis of Type 3 von Willebrand disease based on medical records
- Adequate blood, liver, and kidney function
- Documented previous use of standard-of-care prophylactic therapy for VWD (1-3 times weekly) and plans to remain on the same regimen during the study
- For participants able to have children: agreement to either remain abstinent or follow contraception requirements
You will not qualify if you...
- Having any inherited or acquired bleeding disorder other than congenital Type 3 VWD
- History of gastrointestinal bleeding within 18 months before enrollment or diagnosis of angiodysplasia
- History of intracranial hemorrhage
- Previous or current treatment for thromboembolic disease or signs of thromboembolic disease
- Other conditions that may increase bleeding or clotting risk, such as certain autoimmune diseases
- History of significant allergic reactions to monoclonal antibody treatments or components of emicizumab injection
- Use of systemic immune-modulating drugs (e.g., interferon) at enrollment or planned during the study, except antiretroviral therapy
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - At least 24 weeks
Participants who undergo routine prophylactic treatment for Type 3 von Willebrand disease are observed to evaluate bleeding rates and health-related quality of life.
Visits as per routine clinical care during the study period
Trial Site Locations
Total: 27 locations
1
UC Davis
Sacramento, California, United States, 95817
Actively Recruiting
2
University of Florida
Gainesville, Florida, United States, 32610
Actively Recruiting
3
University of Minnesota Medical Center
Minneapolis, Minnesota, United States, 55455
Actively Recruiting
4
Washington University School of Medicine
St Louis, Missouri, United States, 63110
Actively Recruiting
5
UZ Leuven Gasthuisberg
Leuven, Belgium, 3000
Actively Recruiting
6
The Hospital for Sick Children
Toronto, Ontario, Canada, M5G 1X8
Completed
7
McGill University Health Center
Montreal, Quebec, Canada, H4A 3J1
Actively Recruiting
8
IPS SURA Industriales Medellín
Medellín, Colombia
Actively Recruiting
9
Hopital Claude Huriez - CHU Lille
Lille, France, 59037
Actively Recruiting
10
Groupe Hospitalier Necker Enfants Malades
Paris, France, 75015
Actively Recruiting
11
Universitätsklinikum Bonn
Bonn, Germany, 53127
Actively Recruiting
12
Gerinnungszentrum Rhein-Ruhr;Gerinnungsambulanz
Duisburg, Germany, 47051
Actively Recruiting
13
Hämophiliezentrum Med. Klinik III/Institut für Transfusionsmedizin
Frankfurt/M., Germany, 60590
Actively Recruiting
14
Universita' Degli Studi La Sapienza-Ist.Di Ematologia
Rome, Lazio, Italy, 00161
Actively Recruiting
15
IRCCS Ca' Granda Ospedale Maggiore Policlinico
Milan, Lombardy, Italy, 20122
Actively Recruiting
16
AOU Careggi
Florence, Tuscany, Italy, 50134
Actively Recruiting
17
Kurume University Hospital
Fukuoka, Japan, 830-0011
Completed
18
Tokyo Medical University Hospital
Tokyo, Japan, 160-0023
Completed
19
Erasmus MC
Rotterdam, Netherlands, 3015 GD
Actively Recruiting
20
Instytut Hematologii i Transfuzjologii
Warsaw, Poland, 02-776
Completed
21
Charlotte Maxeke Johannesburg Academic Hospital
Johannesburg, South Africa, 2193
Actively Recruiting
22
Hospital Universitario la Paz
Madrid, Spain, 28046
Actively Recruiting
23
Hospital Universtiario Virgen del Rocio
Seville, Spain, 41013
Actively Recruiting
24
Sahlgrenska Universitetssjukhuset
Gothenburg, Sweden, S-413 45
Completed
25
St Thomas' Hospital
London, United Kingdom, SE1 7EH
Actively Recruiting
26
Great Ormond Street Hospital
London, United Kingdom, WC1N 3JH
Actively Recruiting
27
Manchester Royal Infirmary
Manchester, United Kingdom, M13 9WL
Actively Recruiting
Research Team
R
Reference Study ID Number: WP45335 https://forpatients.roche.com/
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
1
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