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Could a common drug slow vision loss in rare wolfram syndrome?

NCT ID NCT03717909

First seen May 06, 2026 · Last updated May 22, 2026 · Updated 2 times

Summary

This study tested whether sodium valproate, a drug already used for other conditions, can help people with Wolfram syndrome—a rare genetic disorder that causes diabetes, vision loss, and other problems. The trial included 63 children and adults who 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 over time.

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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.