Efficacy Study of Daily Administration of VPA in Patients Affected by Wolfram Syndrome
Status:
Not yet recruiting
Trial end date:
2025-12-01
Target enrollment:
Participant gender:
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).