A Study on Estimated Risks of Certain Adverse Events in Adults With Von Willebrand Disease (VWD) Treated With Veyvondi

August 30, 2023 updated by: Takeda

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.

Study Overview

Status

Completed

Conditions

Study Type

Observational

Enrollment (Actual)

102

Contacts and Locations

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

Study Locations

  • Germany
    • Hessen
      • Bad Homburg vor der Höhe, Hessen, Germany, 61352
        • MVZ Gerinnungszentrum Hochtaunus

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

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

Participants who have been diagnosed with VWD and were prescribed VEYVONDI in Europe.

Description

Inclusion criteria:

  1. Participants with 18 years of age or older
  2. Participants with diagnosis of congenital VWD.
  3. Participants received VEYVONDI with or without a FVIII concentrate.
  4. Participants must have at least 7 days of observation time after the index dose.
  5. 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).

Exclusion criteria:

  1. History of any other coagulation or platelet disorder (including acquired VWD).
  2. History of neutralizing antibodies / inhibitors to VWF or FVIII.

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

  • Observational Models: Cohort
  • Time Perspectives: Retrospective

Cohorts and Interventions

Group / Cohort
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 the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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).
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
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.
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
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.
From 01 January 2019 up to one day before site activation at each site (up to approximately 4 years)

Collaborators and Investigators

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

Sponsor

Takeda

Investigators

  • Study Director: Study Director, Takeda

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)

June 8, 2022

Primary Completion (Actual)

April 6, 2023

Study Completion (Actual)

April 6, 2023

Study Registration Dates

First Submitted

March 2, 2022

First Submitted That Met QC Criteria

March 2, 2022

First Posted (Actual)

March 3, 2022

Study Record Updates

Last Update Posted (Actual)

August 31, 2023

Last Update Submitted That Met QC Criteria

August 30, 2023

Last Verified

August 1, 2023

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • TAK-577-4005
  • EUPAS45617 (Registry Identifier: EUPAS Register)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

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.

IPD Sharing Access Criteria

IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/ For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.

IPD Sharing Supporting Information Type

  • STUDY_PROTOCOL
  • SAP
  • ICF
  • CSR

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

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.

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