Phase
Condition
Von Willebrand Disease
Treatment
von Willebrand Factor (VWF) and Factor VIII (FVIII) Concentrates
Bypassing Agents
Emicizumab
Clinical Study ID
Ages > 2 All Genders
Study Summary
Eligibility Criteria
Inclusion
Inclusion Criteria:
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 adhereto the contraception requirements
Additional Inclusion Criteria for Arms A and B:
Documented previous use of on-demand therapy with intermittent (less than once aweek) on-demand SOC therapy for VWD
Having ≥2 treated bleeds (except menstrual bleeds) with factor concentrate within 24weeks prior to enrollment
Additional Inclusion Criteria for Arm C:
Documented and confirmed previous use of SOC prophylactic therapy for VWD (1-3 timesweekly, as per prescribed dose) as described in the eligibility of Study WP45335
Have completed all study requirements as defined in the WP45335 protocol for atleast 24 weeks
Exclusion
Exclusion Criteria:
Inherited or acquired bleeding disorder other than Congenital Type 3 VWD
History of gastrointestinal bleeding within 18 months prior to enrollment, or anyprevious diagnosis of angiodysplasia
History of intracranial hemorrhage
Previous or current treatment for thromboembolic disease or signs of thromboembolicdisease
Other conditions (e.g., certain autoimmune diseases) that may increase risk ofbleeding or thrombosis
History of clinically significant hypersensitivity associated with monoclonalantibody therapies or components of the emicizumab injection
Use of systemic immunomodulators (e.g., interferon) at enrollment or planned useduring the study, with the exception of anti-retroviral therapy
Study Design
Connect with a study center
UZ Leuven Gasthuisberg
Leuven, 3000
BelgiumActive - Recruiting
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