An Observational Study of Participants With Type 3 Von Willebrand Disease on Prophylactic Standard-of-Care Treatment (WILL-EMI NIS)

A Multicenter Non-Interventional Study Evaluating Bleeds and Health-Related Quality Of Life in Patients With Type 3 Von Willebrand Disease on Prophylactic Standard-of-Care Treatment

This non-interventional study (NIS) is designed to collect information on the effectiveness and safety of treatment received in routine clinical care, as well as measure the health-related quality of life (HRQoL) of participants with Type 3 von Willebrand disease (VWD) receiving prophylactic therapy per local standard of care (SOC) over an observation period of at least 24 weeks.

Study Overview

• Status: Active, not recruiting
• Conditions: Von Willebrand Disease, Type 3
• Intervention / Treatment: Drug: Von Willebrand Factor Concentrates; Drug: Von Willebrand Factor Concentrates and Factor VIII Concentrates; Drug: Factor VIII Concentrates; Drug: Recombinant Activated Factor VII; Drug: Activated Prothrombin Complex Concentrate

• Study Type: Observational
• Enrollment (Actual): 44

Contacts and Locations

Study Locations

• Belgium
  • Leuven, Belgium, 3000
    • UZ Leuven Gasthuisberg
• Canada
  • Ontario
    • Toronto, Ontario, Canada, M5G 1X8
      • The Hospital for Sick Children
  • Quebec
    • Montreal, Quebec, Canada, H4A 3J1
      • McGill University Health Center
• Colombia
  • Medellín, Colombia
    • IPS SURA Industriales Medellín
• France
  • Lille, France, 59037
    • Hopital Claude Huriez - CHU Lille
  • Paris, France, 75015
    • Groupe Hospitalier Necker Enfants Malades
• Germany
  • Bonn, Germany, 53127
    • Universitatsklinikum Bonn
  • Duisburg, Germany, 47051
    • Gerinnungszentrum Rhein-Ruhr;Gerinnungsambulanz
  • Frankfurt/M., Germany, 60590
    • Hämophiliezentrum Med. Klinik III/Institut für Transfusionsmedizin
• Italy
  • Lazio
    • Rome, Lazio, Italy, 00161
      • Universita' Degli Studi La Sapienza-Ist.Di Ematologia
  • Lombardy
    • Milan, Lombardy, Italy, 20122
      • IRCCS Ca' Granda Ospedale Maggiore Policlinico
  • Tuscany
    • Florence, Tuscany, Italy, 50134
      • Aou Careggi
• Japan
  • Fukuoka, Japan, 830-0011
    • Kurume University Hospital
  • Tokyo, Japan, 160-0023
    • Tokyo Medical University Hospital
• Netherlands
  • Rotterdam, Netherlands, 3015 GD
    • Erasmus MC
• Poland
  • Warsaw, Poland, 02-776
    • Instytut Hematologii I Transfuzjologii
• South Africa
  • Johannesburg, South Africa, 2193
    • Charlotte Maxeke Johannesburg Academic Hospital
• Spain
  • Madrid, Spain, 28046
    • Hospital Universitario La Paz
  • Seville, Spain, 41013
    • Hospital Universtiario Virgen del Rocio
• Sweden
  • Gothenburg, Sweden, S-413 45
    • Sahlgrenska Universitetssjukhuset
• United Kingdom
  • London, United Kingdom, WC1N 3JH
    • Great Ormond Street Hospital
  • London, United Kingdom, SE1 7EH
    • St Thomas' Hospital
  • Manchester, United Kingdom, M13 9WL
    • Manchester Royal Infirmary
• United States
  • California
    • Sacramento, California, United States, 95817
      • UC Davis
  • Florida
    • Gainesville, Florida, United States, 32610
      • University of Florida
  • Minnesota
    • Minneapolis, Minnesota, United States, 55455
      • University of Minnesota Medical Center
  • Missouri
    • St Louis, Missouri, United States, 63110
      • Washington University School of Medicine

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

This study will observe approximately 40 participants with Type 3 VWD (aged 2 years and older) that are currently being treated with SOC prophylactic therapy.

Description

Inclusion Criteria:

• Confirmed diagnosis of Type 3 von Willebrand disease (VWD), based on medical records
• Adequate hematologic, hepatic, and renal function
• Documented and confirmed previous use of SOC prophylactic therapy for VWD (1-3 times weekly, as per prescribed dose) and anticipation to remain on the same regimen during the study
• For participants of childbearing potential: agreement to remain abstinent or adhere to the contraception requirements

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

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort

Intervention / Treatment

Participants with Type 3 VWD Treated with Prophylactic SOC; Participants with Type 3 VWD, aged 2 years and above, who are currently on standard of care (SOC) prophylactic therapy are anticipated to remain on the chosen SOC regimen during the study. Dosing and treatment duration of any studied medicinal products collected as part of this study are at the discretion of the treating physician in accordance with local labeling or local treatment guidelines.

Drug: Von Willebrand Factor Concentrates; Used according to local labeling or local treatment guidelines.; Drug: Von Willebrand Factor Concentrates and Factor VIII Concentrates; Used according to local labeling or local treatment guidelines.; Drug: Factor VIII Concentrates; Used according to local labeling or local treatment guidelines.; Drug: Recombinant Activated Factor VII; Used according to local labeling or local treatment guidelines.; Drug: Activated Prothrombin Complex Concentrate; Used according to local labeling or local treatment guidelines.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Annualized Bleed Rate (ABR) for Treated Bleeds	From Baseline to at least 24 weeks

Secondary Outcome Measures

Outcome Measure	Time Frame
ABR for All Bleeds	From Baseline to at least 24 weeks
ABR for Treated Spontaneous Bleeds	From Baseline to at least 24 weeks
ABR for Treated Joint Bleeds	From Baseline to at least 24 weeks
Incidence and Severity of Adverse Events, with Severity Determined According to the World Health Organization (WHO) Toxicity Grading Scale	From Baseline until study completion (at least 24 weeks)

Collaborators and Investigators

• Sponsor: Hoffmann-La Roche
• Investigators: Study Director: Clinical Trials, Hoffmann-La Roche

Publications and helpful links

Helpful Links

• Please use this form to submit your questions for a faster response: https://www.gene.com/contact-us/submit-medical-inquiry. Do not include or attach any medical records when emailing or completing the form. A nurse will respond within 24 business hours.

Study record dates

Study Major Dates

• Study Start (Actual): April 29, 2025
• Primary Completion (Estimated): November 1, 2026
• Study Completion (Estimated): November 1, 2026

Study Registration Dates

• First Submitted: March 13, 2025
• First Submitted That Met QC Criteria: March 13, 2025
• First Posted (Actual): March 19, 2025

Study Record Updates

• Last Update Posted (Actual): June 12, 2026
• Last Update Submitted That Met QC Criteria: June 11, 2026
• Last Verified: June 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Genetic Diseases, Inborn; Hematologic Diseases; Blood Coagulation Disorders; Hemorrhagic Disorders; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Hemic and Lymphatic Diseases; von Willebrand Diseases; von Willebrand Disease, Type 3; Hemostatics; Coagulants; Factor VIII; Thrombin
• Other Study ID Numbers: WP45335

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No