AT-007 for Classic Galactosemia

Phase-Based Progress Estimates
2
Effectiveness
3
Safety
Hassman Research Institute, Berlin, NJ
Classic Galactosemia+1 More
AT-007 - Drug
Eligibility
18+
All Sexes
What conditions do you have?
Select

Study Summary

This study is a 12-month open-label extension (OLE) study of AT-007 in adult subjects with CG who previously participated in Study AT-007-1001 Part D and/or Part D Extension. The study is designed to assess the long-term safety of AT-007 in subjects with CG as well as the pharmacodynamics (PD) (inhibition of galactitol) and PK of AT-007. The effect of 12-month treatment with AT-007 on the levels of galactose and other galactose metabolites in subjects with CG will also be evaluated.

Eligible Conditions

  • Classic Galactosemia

Treatment Effectiveness

Effectiveness Progress

2 of 3
This is further along than 85% of similar trials

Other trials for Classic Galactosemia

Study Objectives

3 Primary · 3 Secondary · Reporting Duration: 12 months

12 months
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
To evaluate quality of life (QOL) measures of adult subjects with CG- PROMIS Emotional Support questionnaire
To evaluate quality of life (QOL) measures of adult subjects with CG- PROMIS Social Isolation questionnaire
To evaluate the burden of illness (BOI) of adult subjects with CG- Medical History
To evaluate the burden of illness (BOI) of adult subjects with CG- NIH Motor Battery
To evaluate the burden of illness (BOI) of adult subjects with CG- Spiral Drawing Test
To evaluate the effect of 12-month oral administration of AT-007 on the levels of galactose and its other metabolites in adult subjects with CG
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 long-term safety of 12-month oral administration of AT-007 in adult subjects with CG as assessed by adverse events.
To evaluate the long-term safety of 12-month oral administration of AT-007 in adult subjects with CG as assessed by clinical laboratory test results.
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.
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

Trial Safety

Safety Progress

3 of 3
This is further along than 85% of similar trials

Other trials for Classic Galactosemia

Trial Design

1 Treatment Group

AT-007
1 of 1
Experimental Treatment

7 Total Participants · 1 Treatment Group

Primary Treatment: AT-007 · No Placebo Group · Phase 3

AT-007
Drug
Experimental Group · 1 Intervention: AT-007 · Intervention Types: Drug
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
AT-007
2019
Completed Phase 2
~100

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: 12 months
Closest Location: Hassman Research Institute · Berlin, NJ
2017First Recorded Clinical Trial
1 TrialsResearching Classic Galactosemia
97 CompletedClinical Trials

Eligibility Criteria

Age 18+ · All Participants · 3 Total Inclusion Criteria

Mark “yes” if the following statements are true for you:
You have previously participated in the Study AT-007-1001 Part D and/or Part D Extension.

About The Reviewer

Michael Gill preview

Michael Gill - B. Sc.

First Published: October 9th, 2021

Last Reviewed: August 12th, 2022

Michael Gill holds a Bachelors of Science in Integrated Science and Mathematics from McMaster University. During his degree he devoted considerable time modeling the pharmacodynamics of promising drug candidates. Since then, he has leveraged this knowledge of the investigational new drug ecosystem to help his father navigate clinical trials for multiple myeloma, an experience which prompted him to co-found Power Life Sciences: a company that helps patients access randomized controlled trials.