The two possible treatments are
A Sodium Valproate treatment
This treatment is for 3 years, until final assessments to see if it works. These are tablets.
B Placebo treatment
This is also for 3 years. The dummy tablets are identical to the real medicine. The patient and doctor will not know which treatment is which.
This study is in Birmingham, UK; Spain; France; and Poland. 70 people will take part. 2 people will have the medicine to every 1 the placebo, decided by chance.
First visit, up to 28 days before starting
• Ask consent to take part
• Baseline tests and brain scan.
• Check person is eligible to take part
Second visit, randomise to valproate or placebo
• Start treatment
Follow-up visit at 6 weeks
• Visit for safety checks
Visits at 6, 12, 18, 24, 36 months.
• Safety checks, blood tests, eye tests
• MRI brain scans at 12, 24, 36 months
Final visit after treatment finished
• Final safety checks
This trial will tell us if Sodium Valproate is SAFE and EFFECTIVE in Wolfram. If these are shown, then doctors may prescribe this medicine for almost everyone with Wolfram in any country.