A Study to Assess the Efficacy and Safety of Emicizumab in Participants With Type 3 Von Willebrand Disease (NCT06998524)
A Phase III, Multicenter, Open-Label Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Emicizumab Prophylaxis in Patients With Type 3 Von Willebrand Disease
🩺 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: Review the eligibility criteria below.
Location: UC Davis — Sacramento, California
Age range: 1 Month
🧪 Interventions in This Study
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (28 Sites)
Showing the first 25 of 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,
📞 Contact the Study Coordinator
Phone: (888) 662-6728
Email: global-roche-genentech-trials@gene.com
Coordinator details come directly from the registered NIH protocol. Confirm the study is still recruiting before traveling.
💰 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.
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💡 How to Participate
- Check the checklist above to see if you may qualify.
- Contact the study coordinator using the phone/email above.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.