New hope for kids with rare sugar disorder: drug shows promise in trial

NCT ID NCT04902781

Completed Disease control Sponsor: Applied Therapeutics, Inc. Source: ClinicalTrials.gov ↗

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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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.

classic galactosemia galactosemia

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

CLASSIC GALACTOSEMIA

Contacts and locations

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Locations

Children's Hospital Colorado

Aurora, Colorado, 80045, United States
Rare Disease Research Center

Atlanta, Georgia, 30318, United States
University of Michigan

Ann Arbor, Michigan, 48109, United States