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

NCT03717909 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 63

Last updated 2026-05-05

No results posted yet for this study

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.

Conditions

  • Wolfram Syndrome

Interventions

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)

Sponsors & Collaborators

  • University of Birmingham

    lead OTHER

Principal Investigators

  • Timothy Barrett, PhD, MB, BS · University of Birmingham

Study Design

Allocation
RANDOMIZED
Purpose
OTHER
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
6 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2018-12-28
Primary Completion
2024-11-15
Completion
2024-11-15

Countries

  • France
  • Poland
  • Spain
  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03717909 on ClinicalTrials.gov