Introduction

The Wilson's Disease Support Group – UK is grateful to all parties interested in conducting clinical trials or doing research into Wilson's disease.  The following is a list of organisations/pharma companies who are currently conducting studies/trials.  Should you wish to put yourself forward for a particular Study/trial, then if you have any concerns please consult your treating physician.  Meanwhile, if you are a Wilson's disease patient living in the UK and would, in general, like to take part in research or being interviewed by a Medical Research company, please consider completing and returning the WD Patient Register - UK form (see link opposite / below), so that we can contact you directly when a suitable study comes to our attention.  Thank you.

Vivet Therapeutics – September 2023
Gene Therapy for Wilson Disease clinical trial – This study has been suspended

Vivet Therapeutics is a French-based company seeking to improve the treatment of Wilson's disease through the potential of gene therapy to target the cause of disease. 

This investigational gene therapy aims to be a one-time treatment that delivers a working ATP7B gene into cells to produce working transporter proteins - ATP7B is the gene that doesn't work in people with this disease. The goal is to restore proper liver function and remove excess copper. By clearing copper from the bloodstream the toxic effect on other organs, like the brain, should be reduced.

Alexion Pharmaceuticals, Inc - April 2021
The Wilson Disease Copper Balance Study

This Study has now been terminated.

The aim of this clinical study in people with Wilson's disease was to find out whether Alexion's investigational product is able to help restore copper balance by promoting the removal of more copper from the body than is taken in through food and drink. 

UCL Queen Square Institute of Neurology, London - The CROWD Study  (Cohort Research On Wilson's Disease) – A UK wide research Study on Wilson's disease

This Study has now been completed.

This Study was launched in 2019 and its aims were to see why some Wilson's disease patients develop liver problems and others develop neurological or psychiatric problems. 

University of Sheffield, Dept of Neuroscience
 
This is an ongoing Study in which a team at the University of Sheffield is investigating mitochondrial biomarkers in Parkinson's disease and other neurodegenerative diseases, including Wilson's disease. If abnormalities are found in mitochondria, there is the possibility of using so-called "mitochondrial rescue" drugs as a treatment. For further information please contact WDSG-UK.

Public Health England (PHE) – The National Congenital Anomaly and Rare Disease Registration – Wilson's Disease Pilot Study

PHE is working on a project to identify the national prevalence rate for Wilson's disease in England. We are very fortunate that Wilson's disease was chosen over all other rare diseases to pilot this scheme. PHE's aim is to help commissioners make the right healthcare decisions for WD patients and to improve their care.

iGEM – A Synthetic Biology Project

In 2016, a group of Oxford University students investigating biological techniques in treating rare diseases looked into the possibility of using novel probiotic therapeutics to treat Wilson's disease.  They presented their findings at an iGem jamboree in Boston, USA, and received a gold medal for their innovative work.