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

NCT ID NCT03717909

Completed Disease control Sponsor: University of Birmingham Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tested a drug called sodium valproate in 63 children and adults with Wolfram syndrome, a rare genetic disorder that causes vision loss and other health problems. Participants were randomly assigned to receive either the drug or a placebo to see if it could slow the worsening of eyesight. The trial was stopped early in 2022, and the results will help determine if this treatment is safe and effective for managing the disease.

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Conditions

The condition(s) this trial relates to.

Wolfram syndrome

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

WOLFRAM SYNDROME

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

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