A Clinical Trial of Dantrolene Sodium in Pediatric and Adult Patients With Wolfram Syndrome
The purpose of this study is to assess the safety and tolerability of dantrolene sodium in patients with Wolfram syndrome.
A Clinical Trial of Sodium Valproate in the UK and Europe
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.
A Phase II Study of Safety and Efficacy of AMX0035 in Adult Patients with Wolfram Syndrome
The purpose of this research study is to find out how safe and tolerable the investigational drug AMX0035 is in participants with Wolfram syndrome. It will also see if AMX0035 may slow the worsening of clinical signs and symptoms of Wolfram syndrome.
Clinical Trials in US, UK and Europe
Recruitment started in the UK in Autumn 2017; and in Spain, France and Poland, in Spring 2018.