Promising drug tested for rare liver disease in newborns
NCT ID NCT03471143
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This early-phase study tested a drug called adrabetadex (VTS-270) given through a vein to treat liver disease in infants with Niemann-Pick disease type C, a rare and fatal genetic disorder. Four babies up to 6 months old received multiple doses to see if the drug could lower abnormal bile acid levels in the blood, a sign of liver problems. The goal was to manage the liver disease, not cure the underlying condition.
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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.
Contacts and locations
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Locations
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St. Louis Children's Hospital
St Louis, Missouri, 63110, United States
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