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

  • Blood Disorder
  • Blood platelet disorder
  • Von Willebrand disease (VWD)
Please note that the recruitment status of the study at your site may differ from the overall study status because some study sites may recruit earlier than others.
Study Status:

Not yet recruiting

This study runs in
N/A
Study Identifier:

NCT06998524 2024-515622-80-00 WP45338

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      The source of the below information is public registry websites such as ClinicalTrials.gov, EuClinicalTrials.eu, ISRCTN.com, etc.. It has been summarised and edited into simpler language. For more information about this clinical study see the For Expert tab on the specific ForPatients page or follow these links to https://clinicaltrials.gov and/or https://euclinicaltrials.eu and/or https://www.isrctn.com.

      The below information is taken directly from public registry websites such as ClinicalTrials.gov, EuClinicalTrials.eu, ISRCTN.com, etc., and has not been edited.

      Results Disclaimer

      Study 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).

      Hoffmann-La Roche Sponsor
      Phase 3 Phase
      NCT06998524, WP45338, 2024-515622-80-00 Study Identifier
      Emicizumab, von Willebrand Factor (VWF) Concentrates, Factor VIII (FVIII) Concentrates, von Willebrand Factor (VWF) and Factor VIII (FVIII) Concentrates, Bypassing Agents Treatments
      Von Willebrand Disease, Type 3 Condition
      Official Title

      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

      Eligibility Criteria

      All Gender
      ≥2 Years Age
      No Healthy Volunteers
      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:

      • 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
      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

      For the latest version of this information please go to www.forpatients.roche.com

       

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