New hope for kids with rare sugar disorder: drug shows promise in trial
NCT ID NCT04902781
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tested an experimental drug called AT-007 in 47 children aged 2 to 18 with Classic Galactosemia, a rare genetic disorder that makes it hard to break down a sugar called galactose. The goal was to see if the drug could improve behavior, daily living skills, and language, while also reducing harmful galactitol levels in the blood. The trial is now complete, and results will help determine if AT-007 can help manage this 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
Locations
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Children's Hospital Colorado
Aurora, Colorado, 80045, United States
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Rare Disease Research Center
Atlanta, Georgia, 30318, United States
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University of Michigan
Ann Arbor, Michigan, 48109, United States
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