A Study to Assess the Efficacy and Safety of Emicizumab in Participants With Type 3 Von Willebrand Disease (NCT06998524)
🩺 Plain-English Summary (8th-Grade Level)
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).
Who can join: To join this study, participants generally need to meet the listed inclusion criteria (10 items) and avoid the listed exclusions (8 items).
Location: UC Davis — Sacramento, California
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistCheck every box below. If you can check all of them, you may meet the study's basic screening requirements (final eligibility is confirmed by the site team).
Usually NOT eligible if any of the following apply:
- 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
🔍 Extracted verbatim from the official NIH eligibility criteria. Always confirm with the study coordinator.
📍 All Participating Trial Locations (28 Sites)
- UC Davis — Sacramento, California
- University of Florida — Gainesville, Florida
- University of Minnesota Medical Center — Minneapolis, Minnesota
- Washington University School of Medicine — St Louis, Missouri
- Virginia Commonwealth University — Richmond, Virginia
- UZ Leuven Gasthuisberg — Leuven,
- The Hospital for Sick Children — Toronto, Ontario
- McGill University Health Center — Montreal, Quebec
- IPS SURA Industriales Medellín — Medellín,
- Hopital Claude Huriez - CHU Lille — Lille,
- Groupe Hospitalier Necker Enfants Malades — Paris,
- Universitätsklinikum Bonn — Bonn,
- Gerinnungszentrum Rhein-Ruhr;Gerinnungsambulanz — Duisburg,
- Hämophiliezentrum Med. Klinik III/Institut für Transfusionsmedizin — Frankfurt/M.,
- 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,
- Nagoya University Hospital — Nagoya,
- Erasmus MC — Rotterdam,
- Instytut Hematologii i Transfuzjologii — Warsaw,
- Charlotte Maxeke Johannesburg Academic Hospital — Johannesburg,
- Hospital Universitario la Paz — Madrid,
- Hospital Universitario Virgen del Rocio — Seville,
- Sahlgrenska Universitetssjukhuset — Gothenburg,
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.