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

Launched by HOFFMANN-LA ROCHE · May 22, 2025

Keywords

ClinConnect Summary

This clinical trial is investigating the effectiveness and safety of a medication called emicizumab for patients with Type 3 von Willebrand disease (VWD), a condition that affects blood clotting. The study will include participants aged 2 years and older who have been diagnosed with this condition. It will compare two approaches: one group will receive emicizumab as a preventive treatment, while another group will continue with their usual on-demand treatment. There’s also a group that has been receiving preventive care that will switch to emicizumab. The goal is to see how well emicizumab works in preventing bleeding and how safe it is for patients.

To qualify for the trial, participants must have a confirmed diagnosis of Type 3 VWD and a medical record showing their treatment history. They should have had at least two treated bleeding episodes within the past six months if they are on on-demand therapy. There are some health conditions that could disqualify individuals from participating, such as other bleeding disorders or certain serious medical histories. Participants can expect to be closely monitored throughout the study to track their health and response to the treatment. It’s worth noting that the trial is not yet recruiting participants, so interested individuals will need to wait for the study to begin.

Gender

ALL

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:
• • 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