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

NCT06998524 · tracked via the Priya Life Science USA tracker

Sponsor

Hoffmann-La Roche

Phase

Phase 3

Started

2025-06-27

Last updated

2026-09-09

Condition(s) studied

Von Willebrand Disease, Type 3

Investigational drug(s) / intervention(s)

Emicizumabvon Willebrand Factor (VWF) ConcentratesFactor VIII (FVIII) Concentratesvon Willebrand Factor (VWF) and Factor VIII (FVIII) ConcentratesBypassing Agents

Emicizumab: 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: Used according to local labeling or local treatment guidelines.

Factor VIII (FVIII) Concentrates: Used according to local labeling or local treatment guidelines.

von Willebrand Factor (VWF) and Factor VIII (FVIII) Concentrates: Used according to local labeling or local treatment guidelines.

Bypassing Agents: Used according to local labeling or local treatment guidelines.

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

Sex

ALL

Min age

1 Month

Max age

—

Healthy volunteers

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

Primary outcome measure(s)

Annualized Bleed Rate (ABR) for Treated Bleeds in the Randomized Arms — From Baseline to at least 24 weeks

Trial sites (29)

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

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Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06998524 on ClinicalTrials.gov ↗ ← All trials in the USA