RECRUITING Phase 3 ClinicalTrials.gov

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

Sponsor: Hoffmann-La Roche • ClinicalTrials.gov ID: NCT06998524

Registry updated: 2026-10-09

Recruiting for Similar Conditions?

Sponsors are looking for patients like you. See if you qualify for compensated research studies.

Description

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).

Interventions

Emicizumab (Drug)
Participants will receive emicizumab 3 milligrams per kilogram (mg/kg) subcutaneous (SC) injections every week (QW) for the first 4 weeks as loading doses, followed by maintenance doses of emicizumab 3 mg/kg SC once every 2 weeks (Q2W). During the extension period, participants may remain on maintenance dose of emicizumab 3 mg/kg Q2W, or change their emicizumab maintenance regimen to 1.5 mg/kg once every week (QW) or 6 mg/kg once every 4 weeks (Q4W), if they prefer and if agreed by the investigators.
von Willebrand Factor (VWF) Concentrates (Drug)
Used according to local labeling or local treatment guidelines.
Factor VIII (FVIII) Concentrates (Drug)
Used according to local labeling or local treatment guidelines.
von Willebrand Factor (VWF) and Factor VIII (FVIII) Concentrates (Drug)
Used according to local labeling or local treatment guidelines.
Bypassing Agents (Drug)
Used according to local labeling or local treatment guidelines.

Eligibility criteria

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

Conditions

Von Willebrand Disease, Type 3

Locations

UC Davis — Sacramento, California, United States
University of Florida — Gainesville, Florida, United States
University of Kentucky Children's Hospital — Lexington, Kentucky, United States
University of Minnesota Medical Center — Minneapolis, Minnesota, United States
Washington University School of Medicine — St Louis, Missouri, United States
Virginia Commonwealth University — Richmond, Virginia, United States
UZ Leuven Gasthuisberg — Leuven, Belgium
The Hospital for Sick Children — Toronto, Ontario, Canada
McGill University Health Center — Montreal, Quebec, Canada
IPS SURA Industriales Medellín — Medellín, Colombia
Hopital Claude Huriez - CHU Lille — Lille, France
Groupe Hospitalier Necker Enfants Malades — Paris, France
Universitätsklinikum Bonn — Bonn, Germany
Gerinnungszentrum Rhein-Ruhr;Gerinnungsambulanz — Duisburg, Germany
Hämophiliezentrum Med. Klinik III/Institut für Transfusionsmedizin — Frankfurt/M., Germany
Universita' Degli Studi La Sapienza-Ist.Di Ematologia — Rome, Lazio, Italy
IRCCS Ca' Granda Ospedale Maggiore Policlinico — Milan, Lombardy, Italy
AOU Careggi — Florence, Tuscany, Italy
Kurume University Hospital — Fukuoka, Japan
Nagoya University Hospital — Nagoya, Japan
Erasmus MC — Rotterdam, Netherlands
Instytut Hematologii i Transfuzjologii — Warsaw, Poland
Charlotte Maxeke Johannesburg Academic Hospital — Johannesburg, South Africa
Hospital Universitario la Paz — Madrid, Spain
Hospital Universitario Virgen del Rocio — Seville, Spain
Sahlgrenska Universitetssjukhuset — Gothenburg, Sweden
St Thomas' Hospital — London, United Kingdom
Great Ormond Street Hospital — London, United Kingdom
Manchester Royal Infirmary — Manchester, United Kingdom

Partner with Nurenyx

Showcase your study to thousands of motivated patients matching your criteria.

Learn More

Official Source

Data sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

View Official Record

Accelerate Your Clinical Trial Recruitment