New drug trial aims to slow rare wolfram syndrome

NCT ID NCT05676034

First seen Apr 22, 2026 · Last updated May 14, 2026 · Updated 4 times

Summary

This study tests an experimental drug called AMX0035 in 12 adults with Wolfram syndrome, a rare genetic disorder that causes diabetes and vision loss. The goal is to see if the drug can protect insulin-producing cells and is safe over up to 4 years. Participants take the drug by mouth and wear a continuous glucose monitor.

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Contacts and locations

Locations

  • Washington University

    St Louis, Missouri, 63110, United States

Conditions

Explore the condition pages connected to this study.