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

Estimating Risk of Selected Adverse Events in Patients With Von Willebrand Disease Treated With VEYVONDI® (Vonicog Alfa; Recombinant Von Willebrand Factor)

Status

Completed

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT05265078

Enrollment

102

Registered

2022-03-03

Start date

2022-06-08

Completion date

2023-04-06

Last updated

2023-08-31

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Von Willebrand Disease (VWD)

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.

Interventions

None listed

Study design

Observational model

COHORT

Time perspective

RETROSPECTIVE

Eligibility

Sex/Gender

ALL

Age

18 Years to No maximum

Healthy volunteers

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.

Design outcomes

Primary

Measure	Time frame	Description
Percentage of Participants With Hypersensitivity Reactions	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	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	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.

Countries

Germany

Outcome results