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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

Study Summary

This trial is an extension of a previous study on the effects of AT-007 in subjects with CG. The purpose of the extension is to assess the long-term safety of AT-007 and its effects on galactose metabolism in subjects with CG.

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

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
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 outcome measures
To evaluate the long-term change of galactitol, a biomarker of AR activity, induced by 12-month oral administration of AT-007 to adult subjects with CG
To evaluate the pharmacokinetic (PK) parameter Area-Under-the-Curve (AUC) of 12-month oral administration of AT-007 in adult subjects with CG (mg*h/L)
To evaluate the pharmacokinetic (PK) parameter maximum concentration (Cmax) of 12-month oral administration of AT-007 in adult subjects with CG
Other outcome measures
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
+6 more

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
878 Total Patients Enrolled

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Is the age limit for this clinical trial 75 years or older?

"The age requirements for this study state that all participants must be at least 18 years old, and cannot be older than 65."

Answered by AI
Recent research and studies
clinicaltrials.govStudy
AT-007 in Adult Subjects With Classic Galactosemia (CG)
2021. "AT-007 in Adult Subjects With Classic Galactosemia (CG)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05418829.
~2 spots leftby Apr 2025
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