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A Study to Assess the Efficacy and Safety of Emicizumab in Participants With Type 3 Von Willebrand Disease
Sponsor: Hoffmann-La Roche
Summary
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).
Official title: 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
Key Details
Gender
All
Age Range
1 Month - Any
Study Type
INTERVENTIONAL
Enrollment
75
Start Date
2025-06-27
Completion Date
2029-03-30
Last Updated
2026-03-19
Healthy Volunteers
No
Conditions
Interventions
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.
Locations (27)
UC Davis
Sacramento, California, United States
University of Florida
Gainesville, Florida, United States
University of Minnesota Medical Center
Minneapolis, Minnesota, United States
Washington University School of Medicine
St Louis, Missouri, 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
Manchester Royal Infirmary
Manchester, United Kingdom