NCT03717909

Efficacy and Safety Trial of Sodium Valproate, in Paediatric and Adult Patients With Wolfram Syndrome

A Pivotal, International, Randomised, Double-blind, Efficacy and Safety Trial of Sodium Valproate, in Paediatric and Adult Patients With Wolfram Syndrome

• Status: Active Not Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 63 (actual)
• Sponsor: University of Birmingham · Academic / Other
• Sex: All
• Age: 6 Years
• Healthy volunteers: Not accepted

Summary

This trial aims to investigate the efficacy, safety and tolerability of sodium valproate in the treatment of patients with Wolfram syndrome. 70 paediatric and adult patients were initially planned to be randomised 2:1 to receive either sodium valproate or placebo at 6 international centres. 63 patients were recruited when a decision was made to stop the study recruitment in November 2022.

Detailed description

This phase II clinical trial is planned as a randomised, double-blind, placebo-controlled 3 year intervention Trial in 70 patients with Classical Wolfram Syndrome aged 6 years and over. The primary outcome of the Trial is considered to be clinically relevant and of sufficient magnitude to be beneficial, as a successful Trial outcome will mean that patients will retain a clinically useful degree of visual acuity and it will decline at a slower rate than in the untreated patients. The MRI Pons Volume change has been shown to correlate with changes in the Wolfram Unified Rating Scale. Patients will be randomised to balance the individual differences across the treatment and placebo groups, therefore reducing the potential for extraneous bias. This will ensure that the treatment effect can be established without the need to account for confounding factors. The value of a placebo arm adds robustness to the Trial by removing the potential for bias from both the investigator and patient perspectives. Investigators will be blinded to the results of the assessments. Certain assessments will be performed by subspecialists (such as ophthalmologists and neurologists), with the Principal Investigator prevented from having access to the results. This subspecialist-led treatment is in line with the current multi-disciplinary management of these patients and will not result in patients being denied access to effective treatment. Patients and investigators will be blinded to treatment. The Trial treatment will be a tablet formulation. This Trial involves 11 clinic visits and 7 follow up telephone calls to reduce the burden of additional travel to the patients.

Conditions

• Wolfram Syndrome

Interventions

Type	Name	Description
DRUG	Sodium Valproate 200Mg E/C Tablet	Treatment with twice-daily oral tablet(s)
DRUG	Sodium Valproate matched placebo	Treatment with twice-daily oral 200mg tablet(s)

Timeline

• Start date: 2018-12-28
• Primary completion: 2024-11-15
• Completion: 2024-11-15
• First posted: 2018-10-24
• Last updated: 2024-07-31

Locations

6 sites across 4 countries: France, Poland, Spain, United Kingdom