Back to search results

Study of Emicizumab for Type 3 Von Willebrand Disease

This study is testing a drug called emicizumab for people with Type 3 von Willebrand disease (VWD), a bleeding disorder. Emicizumab is a monoclonal antibody, a type of protein that helps the blood clot. The study aims to see how well emicizumab works to prevent bleeding and if it is safe. Participants will receive emicizumab injections under the skin, either weekly for the first month then every two weeks, or they may continue their usual treatment. You could be eligible if you have a confirmed diagnosis of Type 3 VWD and meet certain health requirements. The main goal is to measure how many bleeding episodes occur over at least 24 weeks. The current status of this study is unclear, and it plans to enroll 75 participants.

Study design
This is a Phase III, open-label study with 75 planned participants. Some participants will be randomly assigned to receive emicizumab or continue their current treatment, while others will receive emicizumab and be compared to their past treatment data.
What's involved
Participants will receive emicizumab injections under the skin, either weekly for the first 4 weeks, then every two weeks, or continue their usual treatment. The study will measure bleeding episodes for at least 24 weeks.
Compensation
Not stated in the trial record.
Follow-up
The primary endpoint measures bleeding rates from baseline to at least 24 weeks.

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

NCT06998524

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

Recruiting
PHASE3Ages 1+InterventionalTreatment
Hoffmann-La Roche
~75 participants
Updated 2026-09-09 on ClinicalTrials.gov
What's tested:Emicizumabvon Willebrand Factor (VWF) ConcentratesFactor VIII (FVIII) Concentratesvon Willebrand Factor (VWF) and Factor VIII (FVIII) ConcentratesBypassing Agents

At a glance

Recruiting sites
29 of 29 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Annualized Bleed Rate (ABR) for Treated Bleeds in the Randomized Arms
Measured over From Baseline to at least 24 weeks

Conditions

Von Willebrand Disease, Type 3

Explore related trials

von Willebrand Disease, Type 3VWF inhibitor

NCT06998524

Where you'd take part

This study runs at 29 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

    Recruiting

  • Charlotte Maxeke Johannesburg Academic Hospital

    Johannesburg, South Africano site contact published

    Recruiting

  • Erasmus MC

    Rotterdam, Netherlandsno site contact published

    Recruiting

  • Gerinnungszentrum Rhein-Ruhr;Gerinnungsambulanz

    Duisburg, Germanyno site contact published

    Recruiting

  • Great Ormond Street Hospital

    London, United Kingdomno site contact published

    Recruiting

  • Groupe Hospitalier Necker Enfants Malades

    Paris, Franceno site contact published

    Recruiting

  • Hämophiliezentrum Med. Klinik III/Institut für Transfusionsmedizin

    Frankfurt/M., Germanyno site contact published

    Recruiting

  • Hopital Claude Huriez - CHU Lille

    Lille, Franceno site contact published

    Recruiting

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

Study leadership

  • Clinical Trials · STUDY_DIRECTOR · Hoffmann-La Roche

Who to contact

Reference Study ID Number: WP45338 https://forpatients.roche.com/
[email protected]
888-662-6728 (U.S. Only)
Email the study team

Opens a ready-to-send draft in your own email app — review before sending.

Want this trial checked against your situation?

Add a private profile and we'll compare every criterion below against your situation — and tell you which ones are met, uncertain, or excluding.

Check eligibility for this trial ~2 min · HIPAA-protected · delete anytime
Eligibility criteria

Inclusion

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
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
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

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

What this trial measures

  • Annualized Bleed Rate (ABR) for Treated Bleeds in the Randomized ArmsFrom Baseline to at least 24 weeks