Recruitment has started for children, young people and adults with Wolfram Syndrome to the first ever clinical trial into a medicine which has shown promise in laboratory tests to slow down the progression of this rare disease.
The team running the international TREATWOLFRAM trial plans to recruit 70 patients from four countries and will report their findings in 2023 following three years of monitoring to test the real-life effects on patients.
This exciting research will test the medicine, sodium valproate, which is already widely available and commonly prescribed for epilepsy and migraine, to investigate its ability to slow down the loss of sight – a common symptom in Wolfram Syndrome.
There is currently no cure or treatment for this rare, life-limiting genetic condition where symptoms such as diabetes, renal and neurological complications develop in early childhood often leading to blindness and other problems.
Professor Timothy Barrett, Honorary Consultant in Paediatric Endocrinology and Diabetes and Professor of Paediatrics at University of Birmingham, is heading up the trial, which is funded by the Medical Research Council and sponsored by the University of Birmingham.
Local investigators Dr Renuka Dias and Dr Benjamin Wright are working closely with national children’s charity, WellChild, and Wolfram Syndrome UK to incorporate the needs and perspective of families affected by the condition into the trial design and to facilitate effective communication, along with the necessary support for people taking part.
Professor Barrett said:
“The children’s charity WellChild has supported research into this condition for more than 20 years. Their investment, and the support of Wolfram Syndrome UK, has been crucial to the development of the TREATWOLFRAM trial. Thanks to WellChild and Wolfram Syndrome UK, and a fantastic team of healthcare and research professionals, the first children were recruited in January 2019.”
Commenting on the impact on families and the hope that the clinical trial will provide evidence that sodium valproate slows down progression of Wolfram Syndrome, Professor Barrett added:
“This could give affected people more years of useful vision, and buy time while we await gene or other cell therapy treatments.”
Through a nationwide network of children’s nurses, home and garden transformation projects and family support services, WellChild exists to give the growing population of children and young people with serious illness the best possible chance to thrive at home with their families.
Building on a history of investment in medical research into rare diseases, including Wolfram Syndrome, WellChild currently funds a Wolfram Syndrome Family Coordinator, in partnership with Birmingham Children’s Hospital, part of Birmingham Women’s and Children’s NHS Foundation Trust.
WellChild’s Wolfram Syndrome Family Coordinator, Jody Blake, works with families affected by the condition to provide information, support and advice and will play a vital role in supporting families taking part in the trial.