Efficacy Study of Daily Administration of VPA in Patients Affected by Wolfram Syndrome

Summary

Open label, phase II study non randomized single group assignment of 20 evaluable patients 13 years and older, over 37,5 kg body-weight, with sensorineural hearing loss of at least 20 dB at 8 kHz in high frequency average (HFA), and with documented genetic mutations in the WFS1 gene and with at least one other major documented clinical symptom pertaining to Wolfram syndrome (i.e. diabetes mellitus, diabetes insipidus, optic atrophy). Every patients will receive over three years a treatment by VPA (Depakine chrono).

Timeline

Start

2021-11-26

Primary completion

2025-11-01

Completion

2025-12-01

Drugs

Indications