Status:
RECRUITING
A Study to Assess the Efficacy and Safety of Emicizumab in Participants With Type 3 Von Willebrand Disease
Lead Sponsor:
Hoffmann-La Roche
Conditions:
Von Willebrand Disease, Type 3
Eligibility:
All Genders
1+ years
Phase:
PHASE3
Brief 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...
Eligibility Criteria
Inclusion
- 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:
- Age ≥1 month at the time of signing Informed Consent/Assent Form
- 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:
- Age ≥2 years at the time of signing Informed Consent/Assent Form
- 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
Exclusion
- 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
Key Trial Info
Start Date :
June 27 2025
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
March 30 2029
Estimated Enrollment :
75 Patients enrolled
Trial Details
Trial ID
NCT06998524
Start Date
June 27 2025
End Date
March 30 2029
Last Update
December 24 2025
Active Locations (24)
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1
UC Davis
Sacramento, California, United States, 95817
2
University of Florida
Gainesville, Florida, United States, 32610
3
UZ Leuven Gasthuisberg
Leuven, Belgium, 3000
4
The Hospital for Sick Children
Toronto, Ontario, Canada, M5G 1X8