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

Last updated: March 18, 2026
Sponsor: Hoffmann-La Roche
Overall Status: Active - Recruiting

Phase

3

Condition

Von Willebrand Disease

Treatment

Factor VIII (FVIII) Concentrates

von Willebrand Factor (VWF) and Factor VIII (FVIII) Concentrates

Bypassing Agents

Clinical Study ID

NCT06998524
WP45338
2024-515622-80-00
  • Ages > 1
  • All Genders

Study Summary

This is a Phase III, multicenter, open-label clinical study designed to evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of emicizumab prophylaxis in participants aged 1 month and above, who have been diagnosed with Type 3 von Willebrand disease (VWD). Participants on prior standard of care (SOC) on-demand therapy will be assessed via a randomized comparison (Arm A - emicizumab prophylaxis and Arm B - continuation of SOC on-demand therapy), while participants on prior SOC prophylactic therapy (Arm C - emicizumab prophylaxis) will be assessed via intra-participant analysis with data obtained from the preceding non-interventional study (NIS), WP45335 (NCT06883240).

Eligibility Criteria

Inclusion

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 adhereto 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 aweek) on-demand SOC therapy for VWD

  • Having ≥2 treated bleeds (except menstrual bleeds) with factor concentrate within 24weeks 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 timesweekly, as per prescribed dose) as described in the eligibility of Study WP45335

  • Have completed all study requirements as defined in the WP45335 protocol for atleast 24 weeks

Exclusion

Exclusion Criteria:

  • Inherited or acquired bleeding disorder other than Congenital Type 3 VWD

  • History of gastrointestinal bleeding within 18 months prior to enrollment, or anyprevious diagnosis of angiodysplasia

  • History of intracranial hemorrhage

  • Previous or current treatment for thromboembolic disease or signs of thromboembolicdisease

  • Other conditions (e.g., certain autoimmune diseases) that may increase risk ofbleeding or thrombosis

  • History of clinically significant hypersensitivity associated with monoclonalantibody therapies or components of the emicizumab injection

  • Use of systemic immunomodulators (e.g., interferon) at enrollment or planned useduring the study, with the exception of anti-retroviral therapy

Study Design

Total Participants: 75
Treatment Group(s): 5
Primary Treatment: Factor VIII (FVIII) Concentrates
Phase: 3
Study Start date:
June 27, 2025
Estimated Completion Date:
March 30, 2029

Connect with a study center

  • UZ Leuven Gasthuisberg

    Leuven, 3000
    Belgium

    Active - Recruiting

  • UZ Leuven Gasthuisberg

    Leuven 2792482, 3000
    Belgium

    Site Not Available

  • The Hospital for Sick Children

    Toronto, Ontario M5G 1X8
    Canada

    Active - Recruiting

  • The Hospital for Sick Children

    Toronto 6167865, Ontario 6093943 M5G 1X8
    Canada

    Site Not Available

  • McGill University Health Center

    Montreal, Quebec H4A 3J1
    Canada

    Active - Recruiting

  • McGill University Health Center

    Montreal 6077243, Quebec 6115047 H4A 3J1
    Canada

    Site Not Available

  • IPS SURA Industriales Medellín

    Medellín,
    Colombia

    Active - Recruiting

  • IPS SURA Industriales Medellín

    Medellín 3674962,
    Colombia

    Site Not Available

  • Hopital Claude Huriez - CHU Lille

    Lille, 59037
    France

    Active - Recruiting

  • Hopital Claude Huriez - CHU Lille

    Lille 2998324, 59037
    France

    Site Not Available

  • Groupe Hospitalier Necker Enfants Malades

    Paris, 75015
    France

    Active - Recruiting

  • Groupe Hospitalier Necker Enfants Malades

    Paris 2988507, 75015
    France

    Site Not Available

  • Universitätsklinikum Bonn

    Bonn, 53127
    Germany

    Active - Recruiting

  • Universitätsklinikum Bonn

    Bonn 2946447, 53127
    Germany

    Site Not Available

  • Gerinnungszentrum Rhein-Ruhr;Gerinnungsambulanz

    Duisburg, 47051
    Germany

    Active - Recruiting

  • Gerinnungszentrum Rhein-Ruhr;Gerinnungsambulanz

    Duisburg 2934691, 47051
    Germany

    Site Not Available

  • Hämophiliezentrum Med. Klinik III/Institut für Transfusionsmedizin

    Frankfurt/M., 60590
    Germany

    Active - Recruiting

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

    Rome, Lazio 00161
    Italy

    Active - Recruiting

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

    Rome 3169070, Lazio 3174976 00161
    Italy

    Site Not Available

  • IRCCS Ca' Granda Ospedale Maggiore Policlinico

    Milan, Lombardy 20122
    Italy

    Active - Recruiting

  • IRCCS Ca' Granda Ospedale Maggiore Policlinico

    Milan 3173435, Lombardy 3174618 20122
    Italy

    Site Not Available

  • AOU Careggi

    Firenze, Toscana 50134
    Italy

    Site Not Available

  • AOU Careggi

    Florence, Tuscany 50134
    Italy

    Active - Recruiting

  • AOU Careggi

    Florence 3176959, Tuscany 3165361 50134
    Italy

    Site Not Available

  • Nagoya University Hospital

    Aichi, 466-8560
    Japan

    Site Not Available

  • Nagoya University Hospital

    Aichi 11192139, 466-8560
    Japan

    Site Not Available

  • Kurume University Hospital

    Fukuoka, 830-0011
    Japan

    Active - Recruiting

  • Kurume University Hospital

    Fukuoka 1863967, 830-0011
    Japan

    Site Not Available

  • Nagoya University Hospital

    Nagoya, 466-8560
    Japan

    Active - Recruiting

  • Nagoya University Hospital

    Nagoya 1856057, 466-8560
    Japan

    Site Not Available

  • Erasmus MC

    Rotterdam, 3015 GD
    Netherlands

    Active - Recruiting

  • Erasmus MC

    Rotterdam 2747891, 3015 GD
    Netherlands

    Site Not Available

  • Instytut Hematologii i Transfuzjologii

    Warsaw, 02-776
    Poland

    Active - Recruiting

  • Instytut Hematologii i Transfuzjologii

    Warsaw 756135, 02-776
    Poland

    Site Not Available

  • Charlotte Maxeke Johannesburg Academic Hospital

    Johannesburg, 2193
    South Africa

    Active - Recruiting

  • Charlotte Maxeke Johannesburg Academic Hospital

    Johannesburg 993800, 2193
    South Africa

    Site Not Available

  • Hospital Universitario la Paz

    Madrid, 28046
    Spain

    Active - Recruiting

  • Hospital Universitario la Paz

    Madrid 3117735, 28046
    Spain

    Site Not Available

  • Hospital Universitario Virgen del Rocio

    Sevilla, 41013
    Spain

    Site Not Available

  • Hospital Universitario Virgen del Rocio

    Seville, 41013
    Spain

    Active - Recruiting

  • Hospital Universitario Virgen del Rocio

    Seville 2510911, 41013
    Spain

    Site Not Available

  • Sahlgrenska Universitetssjukhuset

    Gothenburg, S-413 45
    Sweden

    Active - Recruiting

  • Sahlgrenska Universitetssjukhuset

    Gothenburg 2711537, S-413 45
    Sweden

    Site Not Available

  • Great Ormond Street Hospital

    London, WC1N 3JH
    United Kingdom

    Active - Recruiting

  • St Thomas' Hospital

    London, SE1 7EH
    United Kingdom

    Active - Recruiting

  • Great Ormond Street Hospital

    London 2643743, WC1N 3JH
    United Kingdom

    Site Not Available

  • St Thomas' Hospital

    London 2643743, SE1 7EH
    United Kingdom

    Active - Recruiting

  • Manchester Royal Infirmary

    Manchester, M13 9WL
    United Kingdom

    Active - Recruiting

  • Manchester Royal Infirmary

    Manchester 2643123, M13 9WL
    United Kingdom

    Site Not Available

  • UC Davis

    Sacramento, California 95817
    United States

    Active - Recruiting

  • UC Davis

    Sacramento 5389489, California 5332921 95817
    United States

    Site Not Available

  • University of Florida

    Gainesville, Florida 32610
    United States

    Active - Recruiting

  • University of Florida

    Gainesville 4156404, Florida 4155751 32610
    United States

    Site Not Available

  • University of Minnesota Medical Center

    Minneapolis, Minnesota 55455
    United States

    Active - Recruiting

  • University of Minnesota Medical Center

    Minneapolis 5037649, Minnesota 5037779 55455
    United States

    Site Not Available

  • Washington University School of Medicine

    St Louis, Missouri 63110
    United States

    Active - Recruiting

  • Washington University School of Medicine

    St Louis 4407066, Missouri 4398678 63110
    United States

    Site Not Available

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