A Study to Assess the Efficacy and Safety of Emicizumab in Participants With Type 3 Von Willebrand Disease
Start Date
6/27/2025
Completion Date
4/30/2029
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).
Eligibility Criteria
Age Range: No minimum to No maximum
Interventions
Emicizumab
von Willebrand Factor (VWF) Concentrates
Factor VIII (FVIII) Concentrates
von Willebrand Factor (VWF) and Factor VIII (FVIII) Concentrates
Bypassing Agents
Conditions
Locations
UC Davis
Sacramento, California 95817
United States
University of Florida
Gainesville, Florida 32610
United States
University of Minnesota Medical Center
Minneapolis, Minnesota 55455
United States
Washington University School of Medicine
St Louis, Missouri 63110
United States
UZ Leuven Gasthuisberg
Leuven, 3000
Belgium
The Hospital for Sick Children
Toronto, Ontario M5G 1X8
Canada
McGill University Health Center
Montreal, Quebec H4A 3J1
Canada
IPS SURA Industriales Medellín
Medellín,
Colombia
Hopital Claude Huriez - CHU Lille
Lille, 59037
France
Groupe Hospitalier Necker Enfants Malades
Paris, 75015
France
Universitätsklinikum Bonn
Bonn, 53127
Germany
Gerinnungszentrum Rhein-Ruhr;Gerinnungsambulanz
Duisburg, 47051
Germany
Hämophiliezentrum Med. Klinik III/Institut für Transfusionsmedizin
Frankfurt/M., 60590
Germany
Universita' Degli Studi La Sapienza-Ist.Di Ematologia
Rome, Lazio 00161
Italy
IRCCS Ca' Granda Ospedale Maggiore Policlinico
Milan, Lombardy 20122
Italy
AOU Careggi
Florence, Tuscany 50134
Italy
Kurume University Hospital
Fukuoka, 830-0011
Japan
Nagoya University Hospital
Nagoya, 466-8560
Japan
Erasmus MC
Rotterdam, 3015 GD
Netherlands
Instytut Hematologii i Transfuzjologii
Warsaw, 02-776
Poland
Charlotte Maxeke Johannesburg Academic Hospital
Johannesburg, 2193
South Africa
Hospital Universitario la Paz
Madrid, 28046
Spain
Hospital Universitario Virgen del Rocio
Seville, 41013
Spain
Sahlgrenska Universitetssjukhuset
Gothenburg, S-413 45
Sweden
St Thomas' Hospital
London, SE1 7EH
United Kingdom
Great Ormond Street Hospital
London, WC1N 3JH
United Kingdom
Manchester Royal Infirmary
Manchester, M13 9WL
United Kingdom