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

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.

Timeline

Start

2018-12-28

Primary completion

2024-11-15

Completion

2024-11-15

Publications

• Dias RP, Brock K, Hu K, Gupta R, Martin U, Peet A, Wilson M, Yu-Wai-Man P, Wright B, Mollan S, Kulkarni A, Meunier I, Billingham L, Nagy Z, Rose H, Koks S, Zatyka M, Astuti D, Lynch T, Morrison KE, Barton D, Cronier S, Malpass R, Evans R, Malhi A, Takhar P, Lamb A, Esteban-Bueno G, Mlynarski W, Orssaud C, Roubertie A, Homer V, Barrett T. Sodium valproate, a potential repurposed treatment for the neurodegeneration in Wolfram syndrome (TREATWOLFRAM): trial protocol for a pivotal multicentre, randomised double-blind controlled trial. BMJ Open. 2025 Feb 26;15(2):e091495. doi: 10.1136/bmjopen-2024-091495.

Drugs

Indications