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A Study to Assess the Efficacy and Safety of Emicizumab in Participants With Type 3 Von Willebrand Disease

RecruitingPhase 3Ages 1 Month - anyAllHoffmann-La Roche
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In plain words

What the trial is, and whether it could suit you

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).

Sign in to get the eligibility rules as simple questions to take to your doctor.

📋 Eligibility rules (as published)
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 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 Criteria: * 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
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What it tests

  • Emicizumab
  • von Willebrand Factor (VWF) Concentrates
  • Factor VIII (FVIII) Concentrates
  • von Willebrand Factor (VWF) and Factor VIII (FVIII) Concentrates
  • Bypassing Agents
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Where

29 sites
UC Davis
Sacramento, United States
University of Florida
Gainesville, United States
University of Kentucky Children's Hospital
Lexington, United States
University of Minnesota Medical Center
Minneapolis, United States
Washington University School of Medicine
St Louis, United States
Virginia Commonwealth University
Richmond, United States
UZ Leuven Gasthuisberg
Leuven, Belgium
The Hospital for Sick Children
Toronto, Canada

…and 21 more on ClinicalTrials.gov.

🤝 Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible — always decide together with your own doctor.
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