NCT05265078

Brief 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. Data from medical records of participants diagnosed with Von Willebrand Disease and treated with VEYVONDI will be evaluated during this study.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
102

participants targeted

Target at P50-P75 for all trials

Timeline
Completed

Started Jun 2022

Shorter than P25 for all trials

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

March 2, 2022

Completed
1 day until next milestone

First Posted

Study publicly available on registry

March 3, 2022

Completed
3 months until next milestone

Study Start

First participant enrolled

June 8, 2022

Completed
10 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

April 6, 2023

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

April 6, 2023

Completed
Last Updated

August 31, 2023

Status Verified

August 1, 2023

Enrollment Period

10 months

First QC Date

March 2, 2022

Last Update Submit

August 30, 2023

Conditions

Outcome Measures

Primary Outcomes (3)

  • 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).

    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.

    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.

    From 01 January 2019 up to one day before site activation at each site (up to approximately 4 years)

Study Arms (1)

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.

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

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

You may qualify if:

  • 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).

You may not qualify if:

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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

MVZ Gerinnungszentrum Hochtaunus

Bad Homburg, Hesse, 61352, Germany

Location

Related Links

MeSH Terms

Conditions

von Willebrand Diseases

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, InheritedBlood Coagulation DisordersHematologic DiseasesHemic and Lymphatic DiseasesCoagulation Protein DisordersBlood Platelet DisordersHemorrhagic DisordersGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

  • Study Director

    Takeda

    STUDY DIRECTOR

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
RETROSPECTIVE
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

March 2, 2022

First Posted

March 3, 2022

Study Start

June 8, 2022

Primary Completion

April 6, 2023

Study Completion

April 6, 2023

Last Updated

August 31, 2023

Record last verified: 2023-08

Data Sharing

IPD Sharing
Will share

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.

Shared Documents
STUDY PROTOCOL, SAP, ICF, CSR
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.
More information

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