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Galactose Metabolism Disorder Treatment

AT-007 for Galactosemia

Phase 3
Waitlist Available
Research Sponsored by Applied Therapeutics, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be older than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 12 months
Awards & highlights
No Placebo-Only Group
Pivotal Trial

Summary

This trial tests the long-term safety and effectiveness of AT-007, a medication taken by mouth, in adults with Classic Galactosemia. The study aims to see if AT-007 can safely reduce harmful substances that build up due to the condition. Researchers will also look at how the medication affects patients' quality of life over several months.

Eligible Conditions
  • Galactosemia

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~12 months
This trial's timeline: 3 weeks for screening, Varies for treatment, and 12 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
To evaluate the long-term safety of 12-month oral administration of AT-007 in adult subjects with CG as assessed by adverse events.
Lymphocyte immune globulin, anti-thymocyte globulin
To evaluate the long-term safety of 12-month oral administration of AT-007 in adult subjects with CG as assessed by physical exam parameters.
Secondary study objectives
To evaluate the pharmacokinetic (PK) parameter maximum concentration (Cmax) of 12-month oral administration of AT-007 in adult subjects with CG
Other study objectives
To evaluate quality of life (QOL) measures of adult subjects with CG- PROMIS Ability to Participate in Social Roles and Activities questionnaire
To evaluate quality of life (QOL) measures of adult subjects with CG- PROMIS Companionship questionnaire
To evaluate quality of life (QOL) measures of adult subjects with CG- PROMIS Emotional Distress questionnaire
+5 more

Side effects data

From 2021 Phase 1 & 2 trial • 114 Patients • NCT04117711
17%
Infections/Infestations
100%
80%
60%
40%
20%
0%
Study treatment Arm
Part A Cohort 1: AT-007 0.5mg/kg
Part B & C Cohort 2: AT-007 10mg/kg
Part D Cohort 1: AT-007 5mg/kg
Part A Cohort 2: AT-007 5mg/kg
Part A Cohort 4: AT-007 20mg/kg
Part A Cohort 3: AT-007 10mg/kg
Part A Cohort 5: 40mg/kg
Part B & C Cohort 3: AT-007 20mg/kg
Part A Placebo Comparator
Part D Placebo Comparator
Part B & C Cohort 1: AT-007 5mg/kg
Part B & C Cohort 4: AT-007 40mg/kg
Part B & C Placebo Comparator
Part D Cohort 2: AT-007 20mg/kg
Part D Extension Placebo Comparator
Part D Cohort 3: AT-007 40mg/kg
Part D Extension Cohort 1: 20mg/kg
Part D Extension Cohort 2: 40mg/kg

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.
Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

1Treatment groups
Experimental Treatment
Group I: AT-007Experimental Treatment1 Intervention
Open-label AT-007 20 mg/kg once daily
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Gavorestat
Not yet FDA approved

Find a Location

Who is running the clinical trial?

Applied Therapeutics, Inc.Lead Sponsor
4 Previous Clinical Trials
892 Total Patients Enrolled
~2 spots leftby Nov 2025
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