Clinical Benefit, Safety, PK and PD Study of AT-007 in Pediatric Subjects With Classic Galactosemia

• Status: Recruiting
• Trial end date: 2023-06-01
• Target enrollment: 0
• Participant gender: All

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 Classic Galactosemia (CG).

• Phase: Phase 2/Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Applied Therapeutics, Inc.

Criteria

Inclusion:

• Male

• Female non-pregnant

• Female non-lactating subjects aged ≥2 to <18 years.

• Diagnosis of Classic Galactosemia, confirmed by decreased GALT (galactose-1-phosphate uridyltransferase gut-associated lymphoid tissue )activity in erythrocytes, or a historical record of diagnosis of GALT (galactose-1-phosphate uridyltransferase gut-associated lymphoid tissue ) deficiency.

Exclusion:

• Male/Female with no significant health problems (other than classic Galactosemia)

• No other disease that would preclude participation in the study.