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

August 7, 2026 updated by: Hoffmann-La Roche

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

Status

Indicates the current recruitment status or the expanded access status.
Active, not recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Observational

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
44

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.
  • Name: Fastest response: use the inquiry form. https://www.gene.com/contact-us/submit-medical-inquiry

Study Contact Backup

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
        • Universitätsklinikum 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
    • 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

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

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

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort

Group / Cohort

A group or subgroup of participants in an observational study that is assessed for biomedical or health outcomes.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Time Frame
Annualized Bleed Rate (ABR) for Treated Bleeds
Time Frame: From Baseline to at least 24 weeks
From Baseline to at least 24 weeks

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Time Frame
ABR for All Bleeds
Time Frame: From Baseline to at least 24 weeks
From Baseline to at least 24 weeks
ABR for Treated Spontaneous Bleeds
Time Frame: From Baseline to at least 24 weeks
From Baseline to at least 24 weeks
ABR for Treated Joint Bleeds
Time Frame: From Baseline to at least 24 weeks
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
Time Frame: From Baseline until study completion (at least 24 weeks)
From Baseline until study completion (at least 24 weeks)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Hoffmann-La Roche

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Clinical Trials, Hoffmann-La Roche

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
April 29, 2025

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
November 1, 2026

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
November 1, 2026

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
March 13, 2025

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
March 13, 2025

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
March 19, 2025

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
August 10, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
August 7, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
August 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant 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

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Cookie Settings

We use cookies to ensure that we give you the best experience on our website. For more details carefully read our Privacy and Cookies Policy. ...>>>You can change your preferences or withdraw your consent at any time by deleting the cookies from your website or computer as described in the policy. Please accept it and choose your preferences by ticking the corresponding boxes in the "Manage Settings" section below. Please carefully read our Terms of Service before you proceed with using any part of this website.
«Manage Settings