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 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

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

March 19 2026

Active Locations (27)

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Page 1 of 7 (27 locations)

1

UC Davis

Sacramento, California, United States, 95817

2

University of Florida

Gainesville, Florida, United States, 32610

3

University of Minnesota Medical Center

Minneapolis, Minnesota, United States, 55455

4

Washington University School of Medicine

St Louis, Missouri, United States, 63110