Treat Wolfram
Research type
Research Study
Full title
A pivotal, international, randomised, double-blind, efficacy and safety trial of sodium valporate in paediatric and adult patients with Wolfram Syndrome
IRAS ID
222620
Contact name
Timothy Barrett
Contact email
Sponsor organisation
University of Birmingham
Eudract number
2017-001215-37
ISRCTN Number
10176118
Clinicaltrials.gov Identifier
Duration of Study in the UK
5 years, 2 months, 0 days
Research summary
Wolfram syndrome is a rare, (1:700,000) genetic life limiting disease. It causes diabetes and blindness in children, then deafness, loss of bladder control, loss of balance, sleep disorders, and sometimes depression. Death is in mid-life from damage to brain cells and brain shrinkage. There is no cure, and nothing to prevent or slow down the disease. The goal of our research is to develop a treatment that will prevent or delay the disease progressing.
We have found a medicine, Sodium Valproate, that treats the disease in our cell models of Wolfram. Sodium Valproate is already used to treat epilepsy in children. However, it should not be used in people with liver disorders, or in pregnancy, and it can cause side effects including nausea, anaemia, tremor, and mood disturbances. Therefore, we need to test its safety and effectiveness for Wolfram patients, in a clinical trial.
We have been awarded a grant from the UK Medical Research Council to lead the first clinical trial of a treatment for Wolfram syndrome. The grant was awarded after rigorous review from many scientists and doctors. The aim of the trial is to show that Sodium Valproate is safe, and effective, in slowing the disease process. We will invite 70 people to take part, from the UK, Spain, France, and Poland. For every 1 person who receives placebo (dummy medicine), 2 will receive the active treatment.
The trial period is 36 months. Participants will be closely monitored for any side effects; for changes in vision; and for changes in brain scans. At the end of the trial, we will know if Sodium Valproate is safe, and slows the progress of the disease. We hope this will allow us to prescribe Sodium Valproate as the first treatment for Wolfram.
REC name
West of Scotland REC 1
REC reference
18/WS/0020
Date of REC Opinion
19 Mar 2018
REC opinion
Further Information Favourable Opinion