Den här översättningen är inte klar ännu. Den här sidan är just nu på engelska.

Gå till den engelska sidan

Cure My Disease

Don't miss your cure!

 Log in / Sign up

New drug aims to stop bleeds in rare blood disorder

NCT ID NCT06998524

Disease control Sponsor: Hoffmann-La Roche Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Sep 10, 2026 · Updated 5 times

Summary

This study tests a medicine called emicizumab to prevent bleeding episodes in people with Type 3 von Willebrand disease, a severe inherited bleeding disorder. About 75 participants, from infants to adults, will receive either emicizumab or continue their current treatment. The goal is to see if emicizumab reduces the number of bleeds that need medical treatment.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

About 75 people

The number the study aims to enrol. It can still change while the study runs.

Started

Jun 2025

Expected to finish

Apr 2029

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

1 month and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for Von Willebrand disease, type 3 are added.

Vår säkerhetsrekommendation!

Genom att skicka in godkänner du våra Användarvillkor

Conditions

The condition(s) this trial relates to.

von Willebrand disease 3

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Von Willebrand disease, type 3

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    29 sites in 14 countries. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • AOU Careggi

    RECRUITING

    Florence, Tuscany, 50134, Italy

  • Charlotte Maxeke Johannesburg Academic Hospital

    RECRUITING

    Johannesburg, 2193, South Africa

  • Erasmus MC

    RECRUITING

    Rotterdam, 3015 GD, Netherlands

  • Gerinnungszentrum Rhein-Ruhr;Gerinnungsambulanz

    RECRUITING

    Duisburg, 47051, Germany

  • Great Ormond Street Hospital

    RECRUITING

    London, WC1N 3JH, United Kingdom

  • Groupe Hospitalier Necker Enfants Malades

    RECRUITING

    Paris, 75015, France

  • Hopital Claude Huriez - CHU Lille

    RECRUITING

    Lille, 59037, France

  • Hospital Universitario Virgen del Rocio

    RECRUITING

    Seville, 41013, Spain

  • Hospital Universitario la Paz

    RECRUITING

    Madrid, 28046, Spain

  • Hämophiliezentrum Med. Klinik III/Institut für Transfusionsmedizin

    RECRUITING

    Frankfurt/M., 60590, Germany

  • IPS SURA Industriales Medellín

    RECRUITING

    Medellín, Colombia

  • IRCCS Ca' Granda Ospedale Maggiore Policlinico

    RECRUITING

    Milan, Lombardy, 20122, Italy

  • Instytut Hematologii i Transfuzjologii

    RECRUITING

    Warsaw, 02-776, Poland

  • Kurume University Hospital

    RECRUITING

    Fukuoka, 830-0011, Japan

  • Manchester Royal Infirmary

    RECRUITING

    Manchester, M13 9WL, United Kingdom

  • McGill University Health Center

    RECRUITING

    Montreal, Quebec, H4A 3J1, Canada

  • Nagoya University Hospital

    RECRUITING

    Nagoya, 466-8560, Japan

  • Sahlgrenska Universitetssjukhuset

    RECRUITING

    Gothenburg, S-413 45, Sweden

  • St Thomas' Hospital

    RECRUITING

    London, SE1 7EH, United Kingdom

  • The Hospital for Sick Children

    RECRUITING

    Toronto, Ontario, M5G 1X8, Canada

  • UC Davis

    RECRUITING

    Sacramento, California, 95817, United States

  • UZ Leuven Gasthuisberg

    RECRUITING

    Leuven, 3000, Belgium

  • Universita' Degli Studi La Sapienza-Ist.Di Ematologia

    RECRUITING

    Rome, Lazio, 00161, Italy

  • University of Florida

    RECRUITING

    Gainesville, Florida, 32610, United States

  • University of Kentucky Children's Hospital

    RECRUITING

    Lexington, Kentucky, 40536, United States

  • University of Minnesota Medical Center

    RECRUITING

    Minneapolis, Minnesota, 55455, United States

  • Universitätsklinikum Bonn

    RECRUITING

    Bonn, 53127, Germany

  • Virginia Commonwealth University

    RECRUITING

    Richmond, Virginia, 23298-0461, United States

  • Washington University School of Medicine

    RECRUITING

    St Louis, Missouri, 63110, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.