Estimating Risk of Selected AEs in Pts with VWD Treated with VEYVONDI®
Research type
Research Study
Full title
Estimating Risk of Selected Adverse Events in Patients with Von Willebrand Disease Treated with VEYVONDI® (vonicogalfa; recombinant Von Willebrand Factor)
IRAS ID
312058
Contact name
Martin Scott
Contact email
Sponsor organisation
Takeda Development Center Americas, Inc.
Duration of Study in the UK
0 years, 7 months, 29 days
Research summary
The proposed study is a retrospective chart review, Estimating Risk of Selected Adverse Events in Patients with Von Willebrand Disease Treated With VEYVONDI® (vonicog alfa; recombinant Von Willebrand Factor). The study is sponsored by Takeda Development Center Americas, Inc.
Von Willebrand disease (VWD) is the most common inherited bleeding condition caused by a low level of von Willebrand factor (VWF) in the blood. VWF helps blood cells stick together (clot) when bleeding. If there's not enough VWF of it or it does not work properly, it takes longer for bleeding to stop.
VEYVONDI is a recombinant VWF (rVWF) that was granted marketing authorisation by the European Medicines Agency (EMA) in September of 2018. VEYVONDI became commercially available in France, Germany, Denmark, Norway, and Sweden in 2018; and in 2019, in Ireland, Austria, and the Netherlands. In 2020, VEYVONDI became available in the UK and Switzerland.
VEYVONDI is indicated in adults with VWD, when desmopressin (DDAVP) treatment alone is ineffective or not indicated for the treatment of haemorrhage and surgical bleeding and prevention of surgical bleeding.
The purpose of the proposed study is to provide further information about the safety of VEYVONDI used in real-world clinical practice. This protocol focuses on the risk of selected AEs in adult patients with VWD treated with VEYVONDI, primarily hypersensitivity reactions, thromboembolic events, and inhibitor formation.
All study data will be retrospectively abstracted from medical records from 80-100 participants from France, Germany, the Netherlands and the UK.
REC name
West Midlands - South Birmingham Research Ethics Committee
REC reference
22/WM/0076
Date of REC Opinion
11 Apr 2022
REC opinion
Further Information Favourable Opinion