International Wilson’s Disease Patient Registry (iWilson Registry)
Research type
Research Study
Full title
International Wilson’s Disease Patient Registry (iWilson Registry)
IRAS ID
309318
Contact name
Joanna Moore
Contact email
Sponsor organisation
Orphalan
Duration of Study in the UK
5 years, 0 months, 0 days
Research summary
Longitudinal, observational, non-interventional, standard of care Registry. This registry is being funded and carried out by the Sponsor, Orphalan – the pharmaceutical organization that has already produced a drug for the treatment of Wilson’s Disease. Registry duration approx 5 years. After evaluation an extension to this registry could be possible.
This Registry will include 500 patients with potentially 45 patients in United Kingdom.
The multicenter study is planned to be conducted in at least 20 sites and 10 countries worldwide.
The launch will focus on European nations including Belgium, Germany, Poland, Spain, UK commencing Q1 2022 and continuing with other EU nations into 2023. It is anticipated, once the registry is established in Europe, the sponsor will extend recruitment to additional countries such as Australia, Brazil and US.
There is currently no global registry for WD. In conjunction with leading WD clinicians, among others the main objectives of the International Registry are to describe the natural history of WD, to explore the clinical phenotype, geographic, ethnic and gender influences on the course of the condition and to facilitate the development of best practice and management guidelines and recommendations to optimize care in order to improve quality of life, health outcomes and standards of care.
Data will be collected from the routinely scheduled Wilson’s Disease (WD) clinic visits at approximately 6-12 month intervals. At enrolment, in addition to data from the clinic visit, retrospective data will be collected from the diagnostic evaluation and any relevant past medical history and a summary of WD medication history.
There are no expected medical benefits to patients from participation in this Registry. The data will be used to describe changes in Wilson’s Disease over time, differences in patient experience and physician’s practice and to plan future studies.REC name
London - Brighton & Sussex Research Ethics Committee
REC reference
22/LO/0085
Date of REC Opinion
23 May 2022
REC opinion
Further Information Favourable Opinion