Von Willebrand Disease, Type 3WILL-EMI NISNCT06883240
An Observational Study of Participants With Type 3 Von Willebrand Disease on Prophylactic Standard-of-Care Treatment
An observational study of Von Willebrand Disease, Type 3, run by Hoffmann-La Roche at 26 sites in 14 countries.
Trial summary
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… Read more about this study
- Who is it for?
- Ages 2 years and older
- Conditions the study is for
- Von Willebrand Disease, Type 3
- What is being tested?
- Von Willebrand Factor Concentrates, Von Willebrand Factor Concentrates and Factor VIII Concentrates and Factor VIII Concentrates
- How many people
- 44 participants
- Where
- 26 sitesin 14 countries
- Study dates
- 29 Apr 2025to 1 Nov 2026
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
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.
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
26 sites in 14 countries.
26 sites in 14 countries
United States3 sites
University of Minnesota Medical Center
Minneapolis, Minnesota, 55455
UC Davis
Sacramento, California, 95817
Washington University School of Medicine
St Louis, Missouri, 63110
Germany3 sites
Universitätsklinikum Bonn
Bonn, 53127
Gerinnungszentrum Rhein-Ruhr;Gerinnungsambulanz
Duisburg, 47051
Hämophiliezentrum Med. Klinik III/Institut für Transfusionsmedizin
Frankfurt/M., 60590
Italy3 sites
AOU Careggi
Florence, Tuscany, 50134
IRCCS Ca' Granda Ospedale Maggiore Policlinico
Milan, Lombardy, 20122
Universita' Degli Studi La Sapienza-Ist.Di Ematologia
Rome, Lazio, 00161
United Kingdom3 sites
St Thomas' Hospital
London, SE1 7EH
Great Ormond Street Hospital
London, WC1N 3JH
Manchester Royal Infirmary
Manchester, M13 9WL
Canada2 sites
McGill University Health Center
Montreal, Quebec, H4A 3J1
The Hospital for Sick Children
Toronto, Ontario, M5G 1X8
France2 sites
Hopital Claude Huriez - CHU Lille
Lille, 59037
Groupe Hospitalier Necker Enfants Malades
Paris, 75015
Japan2 sites
Kurume University Hospital
Fukuoka, 830-0011
Tokyo Medical University Hospital
Tokyo, 160-0023
Spain2 sites
Hospital Universitario la Paz
Madrid, 28046
Hospital Universtiario Virgen del Rocio
Seville, 41013
South Africa1 site
Charlotte Maxeke Johannesburg Academic Hospital
Johannesburg, 2193
About this study
Official title: 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.
- Conditions
- Von Willebrand Disease, Type 3
- Being tested
- Von Willebrand Factor Concentrates, Von Willebrand Factor Concentrates and Factor VIII Concentrates, Factor VIII Concentrates, Recombinant Activated Factor VII, Activated Prothrombin Complex Concentrate
- Medical subject headings
- von Willebrand Disease, Type 3
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Von Willebrand Factor ConcentratesDrug
Used according to local labeling or local treatment guidelines.
Von Willebrand Factor Concentrates and Factor VIII ConcentratesDrug
Used according to local labeling or local treatment guidelines.
Factor VIII ConcentratesDrug
Used according to local labeling or local treatment guidelines.
Recombinant Activated Factor VIIDrug
Used according to local labeling or local treatment guidelines.
Activated Prothrombin Complex ConcentrateDrug
Used according to local labeling or local treatment guidelines.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: 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 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Annualized Bleed Rate (ABR) for Treated Bleeds
Measured: From Baseline to at least 24 weeks
Secondary outcomes (4)
ABR for All Bleeds
Measured: From Baseline to at least 24 weeks
ABR for Treated Spontaneous Bleeds
Measured: From Baseline to at least 24 weeks
ABR for Treated Joint Bleeds
Measured: 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
Measured: From Baseline until study completion (at least 24 weeks)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Mar 2025
- Study start29 Apr 2025
- Primary completion1 Nov 2026 (estimated)
- Study completion1 Nov 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Von Willebrand Disease, Type 3Studies of the same condition, open and under way.
- More trials run by Hoffmann-La RocheThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 44 participants (actual)
Sponsor and investigators
- Lead sponsor
- Hoffmann-La Roche
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Trials, Study director, Hoffmann-La Roche
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06883240
- Sponsor's protocol ID
- WP45335
- Acronym
- WILL-EMI NIS
- Status last verified
- Aug 2026
- First submitted
- 13 Mar 2025
- Last updated
- 10 Aug 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT06883240
- Acronym / sponsor ID
- WILL-EMI NIS / WP45335
- Conditions
- Von Willebrand Disease, Type 3
- Study type
- Observational
- Recruitment status
- Active, not recruiting
- Enrolment
- 44 participants (actual)
- Lead sponsor
- Hoffmann-La RocheIndustry (pharma, biotech, devices)
- Study start
- 29 Apr 2025 (actual)
- Primary completion
- 1 Nov 2026 (estimated)
- Study completion
- 1 Nov 2026 (estimated)
- First posted
- 19 Mar 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Participants with Type 3 VWD Treated with Prophylactic SOC | – | 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 ConcentrateParticipants 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. |
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- 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.
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (7 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Von Willebrand Factor ConcentratesUsed according to local labeling or local treatment guidelines. | Drug | Participants with Type 3 VWD Treated with Prophylactic SOC |
| Von Willebrand Factor Concentrates and Factor VIII ConcentratesUsed according to local labeling or local treatment guidelines. | Drug | Participants with Type 3 VWD Treated with Prophylactic SOC |
| Factor VIII ConcentratesUsed according to local labeling or local treatment guidelines. | Drug | Participants with Type 3 VWD Treated with Prophylactic SOC |
| Recombinant Activated Factor VIIUsed according to local labeling or local treatment guidelines. | Drug | Participants with Type 3 VWD Treated with Prophylactic SOC |
| Activated Prothrombin Complex ConcentrateUsed according to local labeling or local treatment guidelines. | Drug | Participants with Type 3 VWD Treated with Prophylactic SOC |
Primary outcome
Annualized Bleed Rate (ABR) for Treated Bleeds
Time frame: From Baseline to at least 24 weeks
4 secondary outcomes
ABR for All Bleeds
Time frame: From Baseline to at least 24 weeks
ABR for Treated Spontaneous Bleeds
Time frame: From Baseline to at least 24 weeks
ABR for Treated Joint Bleeds
Time frame: 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)
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Aug 2026.
- 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.Link provided by the sponsor.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Clinical Trials study director
Hoffmann-La Roche
26 sites in 14 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Von Willebrand Disease, Type 3, run by Hoffmann-La Roche at 26 sites in 14 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 10 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 26 sites in United States, Germany, Italy, United Kingdom, Canada and 9 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Annualized Bleed Rate (ABR) for Treated Bleeds (time frame: From Baseline to at least 24 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Aug 2026.