An Observational Study of Participants With Type 3 Von Willebrand Disease on Prophylactic Standard-of-Care Treatment

Launched by HOFFMANN-LA ROCHE · Mar 13, 2025

Keywords

ClinConnect Summary

This clinical trial is looking at how effective and safe the standard treatment is for people with Type 3 von Willebrand Disease (VWD), a bleeding disorder, when they receive regular preventive care. The study will last for at least 24 weeks and aims to gather information on participants' health and quality of life while they continue their usual treatment routine.

To be eligible for the study, participants must have a confirmed diagnosis of Type 3 VWD and have been using a standard preventive treatment for this condition at least once a week. They also need to have good overall health in terms of their blood, liver, and kidney functions. The study is open to all genders and includes individuals aged 7 and older. Participants can expect to share their experiences during the study, which will help researchers understand better how this treatment works in everyday life. It’s important to note that certain medical conditions or recent health issues may exclude someone from participating in this trial.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • Confirmed diagnosis of Type 3 von Willebrand disease (VWD), based on medical records
• • Adequate hematologic, hepatic, and renal function
• • Documented and confirmed previous use of SOC prophylactic therapy for VWD (1-3 times weekly, as per prescribed dose) and anticipation to remain on the same regimen during the study
• • For participants of childbearing potential: agreement to remain abstinent or adhere to the contraception requirements
• 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