A pivotal, international, randomised, double-blind, efficacy and safety trial of sodium valproate in paediatric and adult patients with Wolfram Syndrome
Wolfram syndrome is a rare inherited condition that usually begins in childhood. It often starts with diabetes and gradual eyesight loss, and can later cause hearing problems, balance issues, and bladder difficulties. Currently, there is no treatment available to prevent or slow down these symptoms. This clinical trial is testing a medicine called sodium valproate to see if it can slow down or halt the damage caused by Wolfram syndrome. Sodium valproate is already used safely to treat other conditions, such as epilepsy. Researchers now want to find out if it can protect nerve cells in people with Wolfram syndrome. The study includes children and adults aged 6 and older across the UK and Europe. Participants take either sodium valproate or a dummy pill (placebo) every day. Doctors will closely check their vision, brain scans, and overall health throughout the study.
At a glance
What is this study about?
Wolfram syndrome is a rare genetic condition that is passed down through families. It usually shows up in childhood, often starting with diabetes and a gradual loss of vision caused by damage to the optic nerve. Over time, people with Wolfram syndrome can also develop hearing loss, bladder problems, and trouble with their balance. Because the condition slowly damages parts of the brain and nervous system, it can significantly affect daily life.
At the moment, there is no cure for Wolfram syndrome, and there are no medicines that can stop it from getting worse. Doctors can only treat the individual symptoms as they appear. However, laboratory experiments have shown that a medicine called sodium valproate might protect cells from the stress and damage caused by this condition. Because sodium valproate is already well known and used for other illnesses like epilepsy, researchers want to see if it can help people with Wolfram syndrome too.
This trial is taking place at specialist NHS hospital trusts in the UK and partner hospitals in France, Poland, and Spain. Half of the participants will receive sodium valproate tablets, while the other half will receive dummy tablets, known as a placebo. Comparing the two groups allows the medical team to see clearly whether sodium valproate really helps protect eyesight and brain health, and whether it causes any unwanted side effects.
If the study is successful, it could provide doctors with the very first proven treatment to slow down or delay the progression of Wolfram syndrome. This could make a meaningful difference to the long-term health, vision, and independence of children and adults living with this rare condition.
Key takeaways
- This trial is testing whether sodium valproate can slow down or stop the progression of Wolfram syndrome.
- It is open to children and adults aged 6 and older with a confirmed diagnosis.
- Participants take either sodium valproate or a dummy tablet (placebo) every day.
- Because sodium valproate harms unborn babies, strict pregnancy prevention rules are required.
- You can withdraw from the study at any time without giving a reason and without affecting your normal NHS care.
Who may be eligible?
This trial is open to adults and children aged 6 years and older who weigh at least 20 kg (about 3 stone 2 lbs). To take part, you must have a confirmed medical diagnosis of Wolfram syndrome. This usually means you were diagnosed with diabetes or optic nerve changes before the age of 16, and genetic testing has shown changes in the WFS1 gene.
Participants also need to have enough remaining eyesight in at least one eye so that doctors can measure whether the medicine is protecting their vision. You must be able and willing to attend regular hospital check-ups. If you have braces, a cochlear implant, or cannot have an MRI brain scan for any reason, you can still take part in the rest of the study.
Because sodium valproate can cause severe harm to an unborn baby, there are strict safety rules. Females who could become pregnant must have regular negative pregnancy tests and use highly effective contraception. Sexually active males must also use condoms and ensure their partner uses reliable contraception.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- You are aged 6 years or older and weigh at least 20 kg.
- You have a confirmed diagnosis of Wolfram syndrome (including diabetes or vision changes diagnosed before age 16).
- You have a confirmed WFS1 gene mutation on a genetic test.
- You have enough eyesight in at least one eye to complete standard vision tests.
- You are willing to use reliable contraception and take regular pregnancy tests if there is any chance of pregnancy.
What does participation involve?
Taking part in this study involves taking tablets by mouth every day. You will be randomly chosen to receive either sodium valproate or a matching dummy tablet (placebo). Neither you nor your doctor can choose which one you receive. You will need to attend regular clinic appointments at a specialist hospital, such as Birmingham Children's Hospital or University Hospitals Birmingham. During these visits, doctors will test your vision, take blood samples, check your general health, and carry out brain MRI scans if you are able to have them. Females of childbearing age will also take regular pregnancy tests. The study monitors your health over several years to see how well the medicine works and to keep you safe.
Potential risks and benefits
Locations (6)
- Birmingham Children’s Hospital NHS Foundation TrustCity onlyBirmingham, England
- University Hospitals Birmingham NHS Foundation TrustCity onlyBirmingham, England
- CHU de Montpellier - Hôpital Gui de ChauliacCity onlyMontpellier, France
- Hôpital Européen Georges PompidouApproximateParis, France
- Medical University of LodzCity onlyLodz, Poland
- Unidad de Gestión Clínica Almería PeriferiaUnverifiedAlmeria, Spain
Common questions
What is a placebo and why is it used?
A placebo is a dummy tablet that looks and tastes identical to the real medicine but has no active drug inside. It allows researchers to compare results fairly and see if the real medicine makes a genuine difference.
Why is sodium valproate being tested for Wolfram syndrome?
Sodium valproate is already used to treat epilepsy. Laboratory tests on cells showed that it might protect nerve cells from the specific damage caused by Wolfram syndrome.
Can I take part if I have a cochlear implant?
Yes. A cochlear implant means you cannot safely have an MRI scan, but the study team can still include you and carry out all other vision and health checks.
What happens if I experience unpleasant side effects?
The trial doctors will check you regularly. If you develop side effects, your doctor can lower your dose or stop the medicine completely to keep you safe and comfortable.
Do I have to pay to join this trial?
No. Taking part in NHS clinical trials is free, and the study medication, health checks, and tests are provided at no cost to you.
How to find out more
Darren Barton
Always speak to your GP or specialist before deciding to take part in a study.
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