A Study to Assess the Efficacy and Safety of Emicizumab in Participants With Type 3 Von Willebrand Disease

Recruiting now · Phase 3

Conditions studied: Von Willebrand Disease, Type 3

In brief

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).

Key facts

Study ID

NCT06998524

Run by

Hoffmann-La Roche

People needed

75

Starts

2025-06-27

Expected to finish

2029-04-30

Last updated by the study team

2026-07-10

Who can join

Age: 0 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Confirmed diagnosis of Type 3 von Willebrand disease (VWD), based on medical records
• 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
• Additional Inclusion Criteria for Arms A and B:
• 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
• Additional Inclusion Criteria for Arm C:
• 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

You may not qualify if…

• 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

Where it is running

• UC Davis — Sacramento, California, United States (enrolling)
• University of Florida — Gainesville, Florida, United States (enrolling)
• University of Minnesota Medical Center — Minneapolis, Minnesota, United States (enrolling)
• Washington University School of Medicine — St Louis, Missouri, United States (enrolling)
• Virginia Commonwealth University — Richmond, Virginia, United States (enrolling)
• UZ Leuven Gasthuisberg — Leuven, Belgium (enrolling)
• The Hospital for Sick Children — Toronto, Ontario, Canada (enrolling)
• McGill University Health Center — Montreal, Quebec, Canada (enrolling)
• IPS SURA Industriales Medellín — Medellín, Colombia (enrolling)
• Hopital Claude Huriez - CHU Lille — Lille, France (enrolling)
• Groupe Hospitalier Necker Enfants Malades — Paris, France (enrolling)
• Universitätsklinikum Bonn — Bonn, Germany (enrolling)
• Gerinnungszentrum Rhein-Ruhr;Gerinnungsambulanz — Duisburg, Germany (enrolling)
• Hämophiliezentrum Med. Klinik III/Institut für Transfusionsmedizin — Frankfurt/M., Germany (enrolling)
• Universita' Degli Studi La Sapienza-Ist.Di Ematologia — Rome, Lazio, Italy (enrolling)
• IRCCS Ca' Granda Ospedale Maggiore Policlinico — Milan, Lombardy, Italy (enrolling)
• AOU Careggi — Florence, Tuscany, Italy (enrolling)
• Kurume University Hospital — Fukuoka, Japan (enrolling)
• Nagoya University Hospital — Nagoya, Japan (enrolling)
• Erasmus MC — Rotterdam, Netherlands (enrolling)
• Instytut Hematologii i Transfuzjologii — Warsaw, Poland (enrolling)
• Charlotte Maxeke Johannesburg Academic Hospital — Johannesburg, South Africa (enrolling)
• Hospital Universitario la Paz — Madrid, Spain (enrolling)
• Hospital Universitario Virgen del Rocio — Seville, Spain (enrolling)
• Sahlgrenska Universitetssjukhuset — Gothenburg, Sweden (enrolling)

Full record on ClinicalTrials.gov

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