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

Phase 3RecruitingNCT06998524

Hoffmann-La Roche75 enrolled

Overview

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).

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Von Willebrand Disease, Type 3

Interventions

Eligibility

Sex: ALLMin age: 1 Month

Medical Language ↔ Plain English

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

Locations (26)

UC Davis

Sacramento, California, United States

RECRUITING

University of Florida

Gainesville, Florida, United States

RECRUITING

Washington University School of Medicine

St Louis, Missouri, United States

RECRUITING

UZ Leuven Gasthuisberg

Leuven, Belgium

RECRUITING

The Hospital for Sick Children

Toronto, Ontario, Canada

RECRUITING

McGill University Health Center

Montreal, Quebec, Canada

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

...and 16 more locations