Von Willebrand Disease (VWD)NCT05265078
A Study on Estimated Risks of Certain Adverse Events in Adults With Von Willebrand Disease (VWD) Treated With Veyvondi
An observational study of Von Willebrand Disease (VWD), run by Takeda at 1 site in Germany.
Trial summary
The main aim of this study is to estimate the risks of certain adverse events in adults with Von Willebrand Disease treated with VEYVONDI. No study medicines will be provided to participants in this study. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Von Willebrand Disease (VWD)
- Type of treatment
- Injection or infusion
- How many people
- 102 participants
- Where
- 1 siteGermany
- Study dates
- 8 Jun 2022to 6 Apr 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 VWD and were prescribed VEYVONDI in Europe.
Who may be able to join
- Participants with 18 years of age or older
- Participants with diagnosis of congenital VWD.
- Participants received VEYVONDI with or without a FVIII concentrate.
- Participants must have at least 7 days of observation time after the index dose.
- Provides informed consent, if required, in accordance with local ethical and institutional requirements. All medical record data will be abstracted retrospectively; therefore, an exemption or waiver of informed consent will be sought from all associated independent ethics committees (IECs) and institutional review boards (IRBs).
Who cannot join
- History of any other coagulation or platelet disorder (including acquired VWD).
- History of neutralizing antibodies / inhibitors to VWF or FVIII.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Estimating Risk of Selected Adverse Events in Patients With Von Willebrand Disease Treated With VEYVONDI® (Vonicog Alfa; Recombinant Von Willebrand Factor)
The main aim of this study is to estimate the risks of certain adverse events in adults with Von Willebrand Disease treated with VEYVONDI. No study medicines will be provided to participants in this study.
Data from medical records of participants diagnosed with Von Willebrand Disease and treated with VEYVONDI will be evaluated during this study.
- Conditions
- Von Willebrand Disease (VWD)
- 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.
| Study group | Role | What they receive |
|---|---|---|
| Participants Diagnosed With VWD | Participants who have been diagnosed with VWD and prescribed VEYVONDI for the approved indications for the index infusion (first eligible VEYVONDI infusion) will be assessed using secondary data obtained from medical records to evaluate the safety of VEYVONDI in real-world clinical practice. All study data will be retrospectively abstracted from medical records by dedicated clinical research staff in partnership with the treating physician. The data window for this study will begin on 01 January 2019 and end one day before site activation at each site. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of Participants With Hypersensitivity Reactions
Hypersensitivity reactions are acute events and are considered an adverse events of special interest (AESI) in this study if they occur within 7 days of the infusion (including the day of the initial VEYVONDI infusion).
Measured: From 01 January 2019 up to one day before site activation at each site (up to approximately 4 years)
Percentage of Participants With Thromboembolic Events
Thrombotic events considered as an AESIs if diagnosed during the 30 days after a VEYVONDI infusion. Thromboembolic events include venous thrombosis, arterial thrombosis, pulmonary embolism, and cerebral artery thrombosis.
Measured: From 01 January 2019 up to one day before site activation at each site (up to approximately 4 years)
Percentage of Participants With VWF or Factor VIII (FVIII) Inhibitor Formation
Percentage of participants with VWF or FVIII inhibitor formation will be reported.
Measured: From 01 January 2019 up to one day before site activation at each site (up to approximately 4 years)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Mar 2022
- Study start8 Jun 2022
- Primary completion6 Apr 2023
- Study completion6 Apr 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
- 102 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
- NCT05265078
- Sponsor's protocol ID
- TAK-577-4005
- Other IDs
- EUPAS45617
- Status last verified
- Aug 2023
- First submitted
- 2 Mar 2022
- Last updated
- 31 Aug 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
Study at a glance
From the registry record, last updated by the sponsor on 31 Aug 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Estimating Risk of Selected Adverse Events in Patients With Von Willebrand Disease Treated With VEYVONDI® (Vonicog Alfa; Recombinant Von Willebrand Factor)
- ClinicalTrials.gov ID
- NCT05265078
- Acronym / sponsor ID
- TAK-577-4005
- Conditions
- Von Willebrand Disease (VWD)
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 102 participants (actual)
- Lead sponsor
- TakedaIndustry (pharma, biotech, devices)
- Study start
- 8 Jun 2022 (actual)
- Primary completion
- 6 Apr 2023 (actual)
- Study completion
- 6 Apr 2023 (actual)
- First posted
- 3 Mar 2022 (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.
The main aim of this study is to estimate the risks of certain adverse events in adults with Von Willebrand Disease treated with VEYVONDI. No study medicines will be provided to participants in this study.
Data from medical records of participants diagnosed with Von Willebrand Disease and treated with VEYVONDI will be evaluated during this study.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Participants Diagnosed With VWD | – | –Participants who have been diagnosed with VWD and prescribed VEYVONDI for the approved indications for the index infusion (first eligible VEYVONDI infusion) will be assessed using secondary data obtained from medical records to evaluate the safety of VEYVONDI in real-world clinical practice. All study data will be retrospectively abstracted from medical records by dedicated clinical research staff in partnership with the treating physician. The data window for this study will begin on 01 January 2019 and end one day before site activation at each site. |
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 VWD and were prescribed VEYVONDI in Europe.
Key inclusion criteria (5 listed)
- Participants with 18 years of age or older
- Participants with diagnosis of congenital VWD.
- Participants received VEYVONDI with or without a FVIII concentrate.
- Participants must have at least 7 days of observation time after the index dose.
- Provides informed consent, if required, in accordance with local ethical and institutional requirements. All medical record data will be abstracted retrospectively; therefore, an exemption or waiver of informed consent will be sought from all associated independent ethics committees (IECs) and institutional review boards (IRBs).
Key exclusion criteria (2 listed)
- History of any other coagulation or platelet disorder (including acquired VWD).
- History of neutralizing antibodies / inhibitors to VWF or FVIII.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Percentage of Participants With Hypersensitivity Reactions
Time frame: From 01 January 2019 up to one day before site activation at each site (up to approximately 4 years)
Hypersensitivity reactions are acute events and are considered an adverse events of special interest (AESI) in this study if they occur within 7 days of the infusion (including the day of the initial VEYVONDI infusion).
Percentage of Participants With Thromboembolic Events
Time frame: From 01 January 2019 up to one day before site activation at each site (up to approximately 4 years)
Thrombotic events considered as an AESIs if diagnosed during the 30 days after a VEYVONDI infusion. Thromboembolic events include venous thrombosis, arterial thrombosis, pulmonary embolism, and cerebral artery thrombosis.
Percentage of Participants With VWF or Factor VIII (FVIII) Inhibitor Formation
Time frame: From 01 January 2019 up to one day before site activation at each site (up to approximately 4 years)
Percentage of participants with VWF or FVIII inhibitor formation will be reported.
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 31 Aug 2023.
- To obtain more information on the study, click here/on this linkLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
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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
1 site 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 1 site in Germany.
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 2 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 31 Aug 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Germany. 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?
Percentage of Participants With Hypersensitivity Reactions (time frame: From 01 January 2019 up to one day before site activation at each site (up to approximately 4 years)). 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 31 Aug 2023.