A Study to Assess the Efficacy and Safety of Emicizumab in Participants With Type 3 Von Willebrand Disease
NCT ID: NCT06998524
Last Updated: 2025-12-24
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE3
75 participants
INTERVENTIONAL
2025-06-27
2029-03-30
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
NONE
Study Groups
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Arm A (Prior On-Demand SOC): Emicizumab Prophylaxis for 24 Weeks
Participants who are randomized to Arm A, taking on-demand standard of care (SOC) treatment at the time of study entry (and for at least 24 weeks prior to enrollment), will receive emicizumab SC prophylaxis.
Emicizumab
Participants will receive emicizumab 3 milligrams per kilogram (mg/kg) subcutaneous (SC) injections every week (QW) for the first 4 weeks as loading doses, followed by maintenance doses of emicizumab 3 mg/kg SC once every 2 weeks (Q2W).
During the extension period, participants may remain on maintenance dose of emicizumab 3 mg/kg Q2W, or change their emicizumab maintenance regimen to 1.5 mg/kg once every week (QW) or 6 mg/kg once every 4 weeks (Q4W), if they prefer and if agreed by the investigators.
Arm B (Prior On-Demand SOC): On-Demand SOC for 24 Weeks
Participants who are randomized to Arm B, taking on-demand standard of care (SOC) treatment at the time of study entry (and for at least 24 weeks prior to enrollment), will continue to receive their current SOC on-demand treatment until Week 24.
von Willebrand Factor (VWF) Concentrates
Used according to local labeling or local treatment guidelines.
Factor VIII (FVIII) Concentrates
Used according to local labeling or local treatment guidelines.
von Willebrand Factor (VWF) and Factor VIII (FVIII) Concentrates
Used according to local labeling or local treatment guidelines.
Bypassing Agents
Used according to local labeling or local treatment guidelines.
Arm C (Prior Prophylaxis SOC): Emicizumab Prophylaxis for 24 Weeks
Participants who enroll in Arm C, taking SOC prophylactic treatment at the time of study entry and for at least 24 weeks of observation during the preceding NIS WP45335, will receive emicizumab SC prophylaxis.
Emicizumab
Participants will receive emicizumab 3 milligrams per kilogram (mg/kg) subcutaneous (SC) injections every week (QW) for the first 4 weeks as loading doses, followed by maintenance doses of emicizumab 3 mg/kg SC once every 2 weeks (Q2W).
During the extension period, participants may remain on maintenance dose of emicizumab 3 mg/kg Q2W, or change their emicizumab maintenance regimen to 1.5 mg/kg once every week (QW) or 6 mg/kg once every 4 weeks (Q4W), if they prefer and if agreed by the investigators.
Treatment Extension Period for All Arms: Emicizumab Prophylaxis
Participants in Arms A and C who have completed 24 weeks of emicizumab prophylaxis and who derive benefit from emicizumab will have the opportunity to continue to receive emicizumab prophylaxis in the extension period. Participants in Arm B who have completed 24 weeks of SOC on-demand treatment will have the opportunity to receive emicizumab prophylaxis in the extension period.
Emicizumab
Participants will receive emicizumab 3 milligrams per kilogram (mg/kg) subcutaneous (SC) injections every week (QW) for the first 4 weeks as loading doses, followed by maintenance doses of emicizumab 3 mg/kg SC once every 2 weeks (Q2W).
During the extension period, participants may remain on maintenance dose of emicizumab 3 mg/kg Q2W, or change their emicizumab maintenance regimen to 1.5 mg/kg once every week (QW) or 6 mg/kg once every 4 weeks (Q4W), if they prefer and if agreed by the investigators.
Interventions
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Emicizumab
Participants will receive emicizumab 3 milligrams per kilogram (mg/kg) subcutaneous (SC) injections every week (QW) for the first 4 weeks as loading doses, followed by maintenance doses of emicizumab 3 mg/kg SC once every 2 weeks (Q2W).
During the extension period, participants may remain on maintenance dose of emicizumab 3 mg/kg Q2W, or change their emicizumab maintenance regimen to 1.5 mg/kg once every week (QW) or 6 mg/kg once every 4 weeks (Q4W), if they prefer and if agreed by the investigators.
von Willebrand Factor (VWF) Concentrates
Used according to local labeling or local treatment guidelines.
Factor VIII (FVIII) Concentrates
Used according to local labeling or local treatment guidelines.
von Willebrand Factor (VWF) and Factor VIII (FVIII) Concentrates
Used according to local labeling or local treatment guidelines.
Bypassing Agents
Used according to local labeling or local treatment guidelines.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Preexisting medical record verifying the status of von Willebrand factor (VWF) inhibitor (positive or negative, including titer if available)
* Adequate hematologic, hepatic, and renal function
* For participants of childbearing potential: agreement to remain abstinent or adhere to the contraception requirements
* Age ≥1 month at the time of signing Informed Consent/Assent Form
* Documented previous use of on-demand therapy with intermittent (less than once a week) on-demand SOC therapy for VWD
* Having ≥2 treated bleeds (except menstrual bleeds) with factor concentrate within 24 weeks prior to enrollment
* Age ≥2 years at the time of signing Informed Consent/Assent Form
* Documented and confirmed previous use of SOC prophylactic therapy for VWD (1-3 times weekly, as per prescribed dose) as described in the eligibility of Study WP45335
* Have completed all study requirements as defined in the WP45335 protocol for at least 24 weeks
Exclusion Criteria
* 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
1 Month
ALL
No
Sponsors
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Hoffmann-La Roche
INDUSTRY
Responsible Party
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Principal Investigators
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Clinical Trials
Role: STUDY_DIRECTOR
Hoffmann-La Roche
Locations
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UC Davis
Sacramento, California, United States
University of Florida
Gainesville, Florida, 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
Nagoya University Hospital
Nagoya, , 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 Universitario Virgen del Rocio
Seville, , Spain
Sahlgrenska Universitetssjukhuset
Gothenburg, , Sweden
St Thomas' Hospital
London, , United Kingdom
Great Ormond Street Hospital
London, , United Kingdom
Countries
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Central Contacts
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Reference Study ID Number: WP45338 https://forpatients.roche.com/
Role: CONTACT
Phone: 888-662-6728 (U.S. Only)
Email: [email protected]
Other Identifiers
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2024-515622-80-00
Identifier Type: CTIS
Identifier Source: secondary_id
WP45338
Identifier Type: -
Identifier Source: org_study_id