Von Willebrand Disease (VWD)NCT06433778
A Study on the Outcomes of Recombinant Von Willebrand Factor on Demand Treatment and Prevention and Treatment of Bleeding During and After Surgery in Adults With Inherited Von Willebrand Disease in the United Kingdom (UK)
An observational study of Von Willebrand Disease (VWD), run by Takeda at 7 sites in United Kingdom.
Trial summary
This study is a retrospective chart review study and will collect data on real world use of vonicog alfa (Recombinant Von Willebrand Factor [rVWF]). Von Willebrand disease (VWD) is the most common inherited bleeding disorder. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Von Willebrand Disease (VWD)
- How many people
- 34 participants
- Where
- 7 sitesUnited Kingdom
- Study dates
- 16 Dec 2021to 30 Oct 2023
- Phase
- Observational
- Are there results?
- None posted in the registry
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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Participants who have been diagnosed with congenital VWD and were prescribed rVWF in UK.
Who may be able to join
- Adults (aged 18 or over at time of first administration of rVWF) who have provided informed consent and used rVWF within its licensed indication.
- Participants who have been diagnosed with congenital von Willebrand disease.
- Confirmed instance of
- at least one bleed (either a new bleed or ongoing bleed treated under a treatment switch) treated on-demand with rVWF between 01-Oct-2020 and 30-Jun- 2022 and/or
- treatment to prevent and treat surgical bleeds with rVWF between 01-Oct-2020 and 30-Jun-2022
Who cannot join
- Participants who were aged 17 years or less at the time of the first administration of rVWF.
- Participants who have been diagnosed with any other bleeding disorders or factor deficiencies including acquired von Willebrand disease.
- Participants with neutralising antibodies/inhibitors to VWF.
- Participants participation in a clinical trial of an investigational medical product during the study period.
Trial locations and contacts
7 sites in 1 country.
7 sites in 1 country
United Kingdom7 sites
University Hospitals Birmingham
Birmingham, B15 2GW
Leeds Teaching Hospital
Leeds, LS9 7TF
Liverpool University Hospital
Liverpool, L7 8XP
Royal Free London
London, NW3 2QG
Imperial College Healthcare
London, W2 1NY
Manchester University
Manchester, M13 9WL
Oxford University Hospital
Oxford, OX3 9DU
About this study
Official title: Vonicog Alfa (Recombinant Von Willebrand Factor) Treatment Outcomes in Von Willebrand Disease in the UK: a Retrospective Chart Review Study
This study is a retrospective chart review study and will collect data on real world use of vonicog alfa (Recombinant Von Willebrand Factor [rVWF]). Von Willebrand disease (VWD) is the most common inherited bleeding disorder. rVWF is approved in Europe and UK to treat bleeding and to treat and prevent bleeding during surgeries in adults in 2018.
This study will review and collect information on the treatment and bleed prevention of adult persons with inherited VWD with rVWF in UK. These data were already collected as a part of the routine care.
The main aims of this study are to describe the use of rVWF in on-demand treatment of bleeding and the prevention of treatment and treatment of bleeding during surgeries. Other aims are to describe bleedings and their treatment as well as any surgeries before and after first treatment with rVWF and to gather information on the use of healthcare resources (such as hospital visits, emergency room visits, etc.).
- Conditions
- Von Willebrand Disease (VWD)
- Being tested
- No Intervention
- Keywords
- Drug Therapy
- Medical subject headings
- von Willebrand Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
No InterventionOther
This is a non-interventional study.
| Study group | Role | What they receive |
|---|---|---|
| Participants diagnosed With Congenital VWD | Participants who have been diagnosed with congenital VWD and prescribed rVWF within the index date range (defined as the first administration of rVWF and must fall between 1st October 2020 and 30th June 2022) will be assessed using data obtained from medical records to evaluate the treatment outcome of rVWF in real-world clinical practice. Receives: Other: No Intervention |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Bleeding Episodes Treated with rVWF Between the Index Date and Following 12 Months
Bleeding episodes will be assessed based on categories of overall and annualized, type, severity, location, bleed frequency, bleed type/location and bleed severity.
Measured: From index date up to 12 months
Number of Surgical Procedures Between the Index Date and Following 12 Months
Surgical procedures included surgery type, severity, category (emergency or elective). Data on the participants use of rVWF in the pre-, intra- and post-operative setting during the period between the index date and the following 12 months, will be stratified by VWD type, surgery type, severity, location, or treatment rationale.
Measured: From index date up to 12 months
Number of Participants with Surgery Outcomes (Success, Failure, Complications) Between the Index Date and Following 12 Months
Surgery outcomes (success, failure, complications) will be reported.
Measured: From index date up to 12 months
Secondary outcomes (5)
Number of Participants With VWD-Related Healthcare Resource Utilization (HRU)
HRU will include bleeding-related hospitalization rates, outpatient visits, accident and emergency visits.
Measured: Up to 24 months
Number of Participants With Surgery Related Costs By Type
Surgery related costs by type will include length of stay in intensive care unit (ICU), rVWF consumption, factor VIII (FVIII) consumption, VWD treatment consumption, laboratory tests and examinations.
Measured: Up to 24 months
Number of Participants With Bleeding Episodes Treated With rVWF
Bleeding episodes will be reported based on type, severity, location, treatment, and outcomes, of all recorded bleeds within 12 months prior to and 12 months following the first administration of rVWF.
Measured: Up to 24 months
Number of Surgical Procedures
Surgical procedures will be reported based on surgery type (orthopaedic, gastro-intestinal, dental, etc), severity (major, minor), category (emergency or elective) treatment duration and outcomes, of all recorded surgeries within 12 months prior to and 12 months following the first administration of rVWF.
Measured: Up to 24 months
Number of Participants with Surgery Outcomes (Success, Failure, Complications)
Surgery outcomes (success, failure, complications) based on average number of post-operative bleeds will be reported.
Measured: Up to 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 May 2024
- Study start16 Dec 2021
- Primary completion30 Oct 2023
- Study completion30 Oct 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Von Willebrand Disease (VWD)Studies of the same condition, open and under way.
- More trials run by TakedaThe 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
- Retrospective
- Enrolment
- 34 participants (actual)
Sponsor and investigators
- Lead sponsor
- Takeda
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, Takeda
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06433778
- Sponsor's protocol ID
- TAK-577-5001
- Status last verified
- Mar 2026
- First submitted
- 23 May 2024
- Last updated
- 11 Mar 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: Takeda does not provide access to Individual Participant Data when a study is in a very limited (small) study population due to participant privacy concerns such as potential reidentification of study participants.
Study at a glance
From the registry record, last updated by the sponsor on 11 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Vonicog Alfa (Recombinant Von Willebrand Factor) Treatment Outcomes in Von Willebrand Disease in the UK: a Retrospective Chart Review Study
- ClinicalTrials.gov ID
- NCT06433778
- Acronym / sponsor ID
- TAK-577-5001
- Conditions
- Von Willebrand Disease (VWD)
- Keywords
- Drug Therapy
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 34 participants (actual)
- Lead sponsor
- TakedaIndustry (pharma, biotech, devices)
- Study start
- 16 Dec 2021 (actual)
- Primary completion
- 30 Oct 2023 (actual)
- Study completion
- 30 Oct 2023 (actual)
- First posted
- 30 May 2024 (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 study is a retrospective chart review study and will collect data on real world use of vonicog alfa (Recombinant Von Willebrand Factor [rVWF]). Von Willebrand disease (VWD) is the most common inherited bleeding disorder. rVWF is approved in Europe and UK to treat bleeding and to treat and prevent bleeding during surgeries in adults in 2018.
This study will review and collect information on the treatment and bleed prevention of adult persons with inherited VWD with rVWF in UK. These data were already collected as a part of the routine care.
The main aims of this study are to describe the use of rVWF in on-demand treatment of bleeding and the prevention of treatment and treatment of bleeding during surgeries. Other aims are to describe bleedings and their treatment as well as any surgeries before and after first treatment with rVWF and to gather information on the use of healthcare resources (such as hospital visits, emergency room visits, etc.).
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Participants diagnosed With Congenital VWD | – | Other: No InterventionParticipants who have been diagnosed with congenital VWD and prescribed rVWF within the index date range (defined as the first administration of rVWF and must fall between 1st October 2020 and 30th June 2022) will be assessed using data obtained from medical records to evaluate the treatment outcome of rVWF in real-world clinical practice. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Participants who have been diagnosed with congenital VWD and were prescribed rVWF in UK.
Key inclusion criteria (5 listed)
- Adults (aged 18 or over at time of first administration of rVWF) who have provided informed consent and used rVWF within its licensed indication.
- Participants who have been diagnosed with congenital von Willebrand disease.
- Confirmed instance of
Key exclusion criteria (4 listed)
- Participants who were aged 17 years or less at the time of the first administration of rVWF.
- Participants who have been diagnosed with any other bleeding disorders or factor deficiencies including acquired von Willebrand disease.
- Participants with neutralising antibodies/inhibitors to VWF.
- Participants participation in a clinical trial of an investigational medical product during the study period.
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 |
|---|---|---|
| No InterventionThis is a non-interventional study. | Other | Participants diagnosed With Congenital VWD |
Primary outcome
Number of Participants With Bleeding Episodes Treated with rVWF Between the Index Date and Following 12 Months
Time frame: From index date up to 12 months
Bleeding episodes will be assessed based on categories of overall and annualized, type, severity, location, bleed frequency, bleed type/location and bleed severity.
Number of Surgical Procedures Between the Index Date and Following 12 Months
Time frame: From index date up to 12 months
Surgical procedures included surgery type, severity, category (emergency or elective). Data on the participants use of rVWF in the pre-, intra- and post-operative setting during the period between the index date and the following 12 months, will be stratified by VWD type, surgery type, severity, location, or treatment rationale.
Number of Participants with Surgery Outcomes (Success, Failure, Complications) Between the Index Date and Following 12 Months
Time frame: From index date up to 12 months
Surgery outcomes (success, failure, complications) will be reported.
5 secondary outcomes
Number of Participants With VWD-Related Healthcare Resource Utilization (HRU)
Time frame: Up to 24 months
Number of Participants With Surgery Related Costs By Type
Time frame: Up to 24 months
Number of Participants With Bleeding Episodes Treated With rVWF
Time frame: Up to 24 months
Number of Surgical Procedures
Time frame: Up to 24 months
Number of Participants with Surgery Outcomes (Success, Failure, Complications)
Time frame: Up to 24 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Mar 2026.
- To obtain more information on the study, click here/on this link.Link provided by the sponsor.
- Individual participant dataSharing planned: No. Takeda does not provide access to Individual Participant Data when a study is in a very limited (small) study population due to participant privacy concerns such as potential reidentification of study participants.
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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.
Study Director study director
Takeda
7 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Von Willebrand Disease (VWD), run by Takeda at 7 sites in United Kingdom.
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 5 inclusion and 4 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: finished, as of the sponsor's last update on 11 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in United Kingdom. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Number of Participants With Bleeding Episodes Treated with rVWF Between the Index Date and Following 12 Months (time frame: From index date up to 12 months). The study lists 3 primary outcomes in all. 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 11 Mar 2026.