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

Classic Galactosemia Clinical Trial

Official title: A Sequential, Two-Part Study to Evaluate the Clinical Benefit, Safety, Pharmacokinetics, and Pharmacodynamic of AT-007 in Pediatric Subjects With Classic Galactosemia (CG)

Status Active, not recruiting

Clinical Trial 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).

Clinical Trial Description

This is a randomized, double-blind, placebo-controlled study in pediatric subjects with CG. Three age groups (≥2 to ≤6-year olds, ≥7 to ≤12-year olds, and ≥13 to <18- year olds) will be enrolled. The study is designed to assess the clinical benefit (changes in how pediatric patients with CG feels and function, changes in ocular disorders, and changes in sexual maturation, growth and development) as well as the safety, PK, and PD (reduction of galactitol levels) of AT-007 in pediatric subjects with CG. The study consists of 2 main parts, Part A and Part B, and an open-label extension (OLE). Part A is an intra-patient dose escalation evaluating multiple ascending doses (MAD) of AT-007. Part B is designed to assess the clinical benefits of long-term administration of AT-007 (at the optimum dose identified in Part A) on how patients with CG feel and function. Open-label extension (OLE) is an active treatment extension for patients who received placebo treatment in Part B. ;

Related Conditions & MeSH terms

• Classic Galactosemia
• Galactosemias

• NCT number: NCT04902781
• Study type: Interventional
• Source: Applied Therapeutics, Inc.
• Status: Active, not recruiting
• Phase: Phase 2/Phase 3
• Start date: March 20, 2021
• Completion date: September 2023