Hope for rare wolfram syndrome: drug aims to slow diabetes progression
NCT ID NCT05676034
First seen Jun 27, 2026 · Last updated Jun 27, 2026
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 help preserve the pancreas's ability to make insulin and to check its safety over up to 4 years. Participants take the drug by mouth and wear a continuous glucose monitor throughout the study.
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
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Locations
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Washington University
St Louis, Missouri, 63110, United States