Observational Study for Type 3 Von Willebrand Disease

This study is looking at people with Type 3 Von Willebrand Disease (VWD), a bleeding disorder, who are already receiving their usual care to prevent bleeding. It's an observational study, meaning researchers will collect information about how well your current treatment works and your quality of life. The treatments being observed include Von Willebrand Factor Concentrates, Von Willebrand Factor Concentrates and Factor VIII Concentrates, Factor VIII Concentrates, Recombinant Activated Factor VII, and Activated Prothrombin Complex Concentrate. To join, you must have a confirmed diagnosis of Type 3 VWD, be at least 2 years old, and already be on a regular preventative treatment for VWD. The main goal is to see how often bleeding events occur over at least 24 weeks.

Study design
This is an observational study, not a treatment study, designed to gather information from 44 participants already receiving standard care. It is not specified if it is randomized or blinded.
What's involved
Participants will be observed for at least 24 weeks while continuing their usual preventative treatment for Type 3 VWD. The study aims to collect information on treatment effectiveness, safety, and health-related quality of life.
Compensation
Not stated in the trial record.
Follow-up
Participants will be observed for at least 24 weeks from the start of the study.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT06883240

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

Active, Not Recruiting
Not specifiedAges 2+Observational
Hoffmann-La Roche
~44 participants
Updated 2026-08-10 on ClinicalTrials.gov
What's tested:Von Willebrand Factor ConcentratesVon Willebrand Factor Concentrates and Factor VIII ConcentratesFactor VIII ConcentratesRecombinant Activated Factor VIIActivated Prothrombin Complex Concentrate

At a glance

Recruiting sites
0 of 26 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Annualized Bleed Rate (ABR) for Treated Bleeds
Measured over From Baseline to at least 24 weeks
Von Willebrand Disease, Type 3

NCT06883240

Where you'd take part

This study runs at 26 sites. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.

  • AOU Careggi

    Florence, Tuscany, Italyno site contact published

  • Charlotte Maxeke Johannesburg Academic Hospital

    Johannesburg, South Africano site contact published

  • Erasmus MC

    Rotterdam, Netherlandsno site contact published

  • Gerinnungszentrum Rhein-Ruhr;Gerinnungsambulanz

    Duisburg, Germanyno site contact published

  • Great Ormond Street Hospital

    London, United Kingdomno site contact published

  • Groupe Hospitalier Necker Enfants Malades

    Paris, Franceno site contact published

  • Hämophiliezentrum Med. Klinik III/Institut für Transfusionsmedizin

    Frankfurt/M., Germanyno site contact published

  • Hopital Claude Huriez - CHU Lille

    Lille, Franceno site contact published

Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.

  • Clinical Trials · STUDY_DIRECTOR · Hoffmann-La Roche

This trial hasn't published a contact. View it on ClinicalTrials.gov

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Eligibility criteria

Inclusion

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

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
  • Annualized Bleed Rate (ABR) for Treated BleedsFrom Baseline to at least 24 weeks