Actively Recruiting
A Study to Assess the Efficacy and Safety of Emicizumab in Participants With Type 3 Von Willebrand Disease
Led by Hoffmann-La Roche · Updated on 2026-04-20
75
Participants Needed
27
Research Sites
196 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
This is a Phase III, multicenter, open-label clinical study designed to evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of emicizumab prophylaxis in participants aged 1 month and above, who have been diagnosed with Type 3 von Willebrand disease (VWD). Participants on prior standard of care (SOC) on-demand therapy will be assessed via a randomized comparison (Arm A - emicizumab prophylaxis and Arm B - continuation of SOC on-demand therapy), while participants on prior SOC prophylactic therapy (Arm C - emicizumab prophylaxis) will be assessed via intra-participant analysis with data obtained from the preceding non-interventional study (NIS), WP45335 (NCT06883240).
CONDITIONS
Official Title
A Study to Assess the Efficacy and Safety of Emicizumab in Participants With Type 3 Von Willebrand Disease
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Confirmed diagnosis of Type 3 von Willebrand disease based on medical records
- Medical record verifying von Willebrand factor inhibitor status (positive or negative, including titer if available)
- Adequate blood, liver, and kidney function
- For participants who can become pregnant: agree to abstain or follow contraception requirements
- For Arms A and B: Age 1 month or older at consent
- For Arms A and B: Previous use of on-demand treatment less than once a week for VWD
- For Arms A and B: At least 2 treated bleeds (excluding menstrual bleeds) with factor concentrate in the 24 weeks before enrollment
- For Arm C: Age 2 years or older at consent
- For Arm C: Documented use of prophylactic therapy for VWD (1-3 times weekly) as per prior study
- For Arm C: Completed all requirements of the previous study (WP45335) for at least 24 weeks
You will not qualify if you...
- Any bleeding disorder other than congenital Type 3 von Willebrand disease
- History of gastrointestinal bleeding within 18 months before enrollment or angiodysplasia diagnosis
- History of intracranial hemorrhage
- Previous or current treatment for blood clots or signs of clotting disease
- Other conditions that increase risk of bleeding or clotting, such as certain autoimmune diseases
- History of significant allergic reactions to monoclonal antibody treatments or components of emicizumab
- Use of systemic immune-modulating drugs (e.g., interferon) at enrollment or planned during the study, except for anti-retroviral therapy
AI-Screening
AI-Powered Screening
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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
Actively Recruiting
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
Actively Recruiting
18
Nagoya University Hospital
Nagoya, Japan, 466-8560
Actively Recruiting
19
Erasmus MC
Rotterdam, Netherlands, 3015 GD
Actively Recruiting
20
Instytut Hematologii i Transfuzjologii
Warsaw, Poland, 02-776
Actively Recruiting
21
Charlotte Maxeke Johannesburg Academic Hospital
Johannesburg, South Africa, 2193
Actively Recruiting
22
Hospital Universitario la Paz
Madrid, Spain, 28046
Actively Recruiting
23
Hospital Universitario Virgen del Rocio
Seville, Spain, 41013
Actively Recruiting
24
Sahlgrenska Universitetssjukhuset
Gothenburg, Sweden, S-413 45
Actively Recruiting
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: WP45338 https://forpatients.roche.com/
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
4
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