Status:
COMPLETED
Clinical Benefit, Safety, PK and PD Study of AT-007 in Pediatric Subjects With Classic Galactosemia
Lead Sponsor:
Applied Therapeutics, Inc.
Conditions:
Classic Galactosemia
Eligibility:
All Genders
2-17 years
Phase:
PHASE2
PHASE3
Brief Summary
This study is designed to assess the clinical benefit as well as the safety, pharmacokinetics (PK), and pharmacodynamics (PD) (reduction of galactitol levels) of AT-007 in pediatric subjects with Clas...
Detailed Description
This is a randomized, double-blind, placebo-controlled study in pediatric subjects with CG. Children with CG ages 2 through 17 years will be enrolled. The study is designed to assess the clinical bene...
Eligibility Criteria
Inclusion
- 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.
Exclusion
Key Trial Info
Start Date :
March 20 2021
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
May 21 2025
Estimated Enrollment :
47 Patients enrolled
Trial Details
Trial ID
NCT04902781
Start Date
March 20 2021
End Date
May 21 2025
Last Update
October 29 2025
Active Locations (3)
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1
Children's Hospital Colorado
Aurora, Colorado, United States, 80045
2
Rare Disease Research Center
Atlanta, Georgia, United States, 30318
3
University of Michigan
Ann Arbor, Michigan, United States, 48109