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
Status: Recruiting
Location: See all (13) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 3
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 2 years 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
Participation Requirements
Sex: All
Minimum Age: 2
Healthy Volunteers: f
View:
• 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:
• 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:
• 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
Locations
United States
California
UC Davis
RECRUITING
Sacramento
Other Locations
Belgium
UZ Leuven Gasthuisberg
RECRUITING
Leuven
Canada
McGill University Health Center
RECRUITING
Montreal
France
Hopital Claude Huriez - CHU Lille
RECRUITING
Lille
Germany
Universitätsklinikum Bonn
RECRUITING
Bonn
Gerinnungszentrum Rhein-Ruhr;Gerinnungsambulanz
RECRUITING
Duisburg
Hämophiliezentrum Med. Klinik III/Institut für Transfusionsmedizin
RECRUITING
Frankfurt/m.
Italy
AOU Careggi
RECRUITING
Florence
Japan
Nagoya University Hospital
RECRUITING
Nagoya
Netherlands
Erasmus MC
RECRUITING
Rotterdam
Poland
Instytut Hematologii i Transfuzjologii
RECRUITING
Warsaw
Spain
Hospital Universitario la Paz
RECRUITING
Madrid
Hospital Universitario Virgen del Rocio
RECRUITING
Seville
Contact Information
Primary
Reference Study ID Number: WP45338 https://forpatients.roche.com/
global-roche-genentech-trials@gene.com
888-662-6728 (U.S. Only)
Time Frame
Start Date:2025-06-27
Estimated Completion Date:2028-12-31
Participants
Target number of participants:66
Treatments
Experimental: Arm A (Prior On-Demand SOC): Emicizumab Prophylaxis for 24 Weeks
Participants who are randomized to Arm A, taking on-demand standard of care (SOC) treatment at the time of study entry (and for at least 24 weeks prior to enrollment), will receive emicizumab SC prophylaxis.
Active_comparator: Arm B (Prior On-Demand SOC): On-Demand SOC for 24 Weeks
Participants who are randomized to Arm B, taking on-demand standard of care (SOC) treatment at the time of study entry (and for at least 24 weeks prior to enrollment), will continue to receive their current SOC on-demand treatment until Week 24.
Experimental: Arm C (Prior Prophylaxis SOC): Emicizumab Prophylaxis for 24 Weeks
Participants who enroll in Arm C, taking SOC prophylactic treatment at the time of study entry and for at least 24 weeks of observation during the preceding NIS WP45335, will receive emicizumab SC prophylaxis.
Experimental: Treatment Extension Period for All Arms: Emicizumab Prophylaxis
Participants in Arms A and C who have completed 24 weeks of emicizumab prophylaxis and who derive benefit from emicizumab will have the opportunity to continue to receive emicizumab prophylaxis in the extension period. Participants in Arm B who have completed 24 weeks of SOC on-demand treatment will have the opportunity to receive emicizumab prophylaxis in the extension period.