Den här översättningen är inte klar ännu. Den här sidan är just nu på engelska.

Gå till den engelska sidan

Cure My Disease

Don't miss your cure!

 Log in / Sign up

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

NCT ID NCT04902781

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

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2/3

Runs two stages together: whether the treatment works, then large-scale confirmation.

Participants

47 people

The number who actually took part.

Started

Mar 2021

Finished

May 2025

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

2 to 17 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion: * Male * Female non-pregnant * Female non-lactating subjects aged ≥2 to \<18 years. * Diagnosis of Classic Galactosemia, confirmed by \<1% GALT (galactose-1-phosphate uridyltransferase) enzyme activity in erythrocytes, or a historical record of diagnosis of \<1% GALT enzyme activity. Exclusion: * Male/Female with no significant health problems (other than classic Galactosemia) * No other disease that would preclude participation in the study.

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for Classic galactosemia are added.

Vår säkerhetsrekommendation!

Genom att skicka in godkänner du våra Användarvillkor

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

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

More trials for these conditions

Other studies related to the condition(s) this trial covers.