Drug trial aims to slow vision loss in rare wolfram syndrome

NCT ID NCT03717909

First seen May 06, 2026 · Last updated May 06, 2026

Summary

This study tested the drug sodium valproate in 63 children and adults with Wolfram syndrome, a rare genetic disorder that causes vision loss, diabetes, and other problems. Participants were randomly assigned to receive either the drug or a placebo. The main goal was to see if the drug could slow the worsening of eyesight. The trial was stopped early in 2022, and results are being analyzed to determine if the treatment is safe and effective.

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Contacts and locations

Locations

  • Birmingham Women's and Children's Hospital

    Birmingham, B4 6NH, United Kingdom

  • CHU de Montpellier, Hopital Gui de Chauliac

    Montpellier, 34295, France

  • Hôpital Européen Georges-Pompidou

    Paris, 75015, France

  • Medical University of Lodz

    Lodz, 91-738, Poland

  • Unidad de Gestión Clínica Almería Periferia. Distrito Sanitario Almería

    Almería, 04120, Spain

  • University Hospitals Birmingham

    Birmingham, B15 2TH, United Kingdom

Conditions

Explore the condition pages connected to this study.