Experimental pill targets toxic fat buildup in rare genetic disease
NCT ID NCT05443685
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tested an oral drug called ADX-629 in 8 people with Sjögren-Larsson syndrome, a rare inherited disorder that causes harmful fatty substances to build up in the skin, brain, and eyes. The main goals were to see if the drug is safe and can reduce these fatty aldehydes. Participants took the drug daily for 12 weeks and were monitored for side effects and changes in disease markers.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
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Locations
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University of Nebraska Medical Center
Omaha, Nebraska, 68198, United States
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