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

Last updated: June 9, 2025
Sponsor: Hoffmann-La Roche
Overall Status: Active - Recruiting

Phase

3

Condition

Von Willebrand Disease

Treatment

von Willebrand Factor (VWF) and Factor VIII (FVIII) Concentrates

Bypassing Agents

Emicizumab

Clinical Study ID

NCT06998524
WP45338
2024-515622-80-00
  • Ages > 2
  • All Genders

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).

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

Total Participants: 66
Treatment Group(s): 5
Primary Treatment: von Willebrand Factor (VWF) and Factor VIII (FVIII) Concentrates
Phase: 3
Study Start date:
June 15, 2025
Estimated Completion Date:
May 15, 2029

Connect with a study center

  • UZ Leuven Gasthuisberg

    Leuven, 3000
    Belgium

    Active - Recruiting

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