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NCT06998524PHASE3Recruiting

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

Hoffmann-La Roche

Start Date

6/27/2025

Completion Date

4/30/2029

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

Age Range: No minimum to No maximum

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

Interventions

DRUG

Emicizumab

DRUG

von Willebrand Factor (VWF) Concentrates

DRUG

Factor VIII (FVIII) Concentrates

DRUG

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

DRUG

Bypassing Agents

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Conditions

Von Willebrand Disease, Type 3

Locations

UC Davis

Sacramento, California 95817

United States

University of Florida

Gainesville, Florida 32610

United States

University of Minnesota Medical Center

Minneapolis, Minnesota 55455

United States

Washington University School of Medicine

St Louis, Missouri 63110

United States

UZ Leuven Gasthuisberg

Leuven, 3000

Belgium

The Hospital for Sick Children

Toronto, Ontario M5G 1X8

Canada

McGill University Health Center

Montreal, Quebec H4A 3J1

Canada

IPS SURA Industriales Medellín

Medellín,

Colombia

Hopital Claude Huriez - CHU Lille

Lille, 59037

France

Groupe Hospitalier Necker Enfants Malades

Paris, 75015

France

Universitätsklinikum Bonn

Bonn, 53127

Germany

Gerinnungszentrum Rhein-Ruhr;Gerinnungsambulanz

Duisburg, 47051

Germany

Hämophiliezentrum Med. Klinik III/Institut für Transfusionsmedizin

Frankfurt/M., 60590

Germany

Universita' Degli Studi La Sapienza-Ist.Di Ematologia

Rome, Lazio 00161

Italy

IRCCS Ca' Granda Ospedale Maggiore Policlinico

Milan, Lombardy 20122

Italy

AOU Careggi

Florence, Tuscany 50134

Italy

Kurume University Hospital

Fukuoka, 830-0011

Japan

Nagoya University Hospital

Nagoya, 466-8560

Japan

Erasmus MC

Rotterdam, 3015 GD

Netherlands

Instytut Hematologii i Transfuzjologii

Warsaw, 02-776

Poland

Charlotte Maxeke Johannesburg Academic Hospital

Johannesburg, 2193

South Africa

Hospital Universitario la Paz

Madrid, 28046

Spain

Hospital Universitario Virgen del Rocio

Seville, 41013

Spain

Sahlgrenska Universitetssjukhuset

Gothenburg, S-413 45

Sweden

St Thomas' Hospital

London, SE1 7EH

United Kingdom

Great Ormond Street Hospital

London, WC1N 3JH

United Kingdom

Manchester Royal Infirmary

Manchester, M13 9WL

United Kingdom