NCT04902781

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

A Sequential, Two-Part Study to Evaluate the Clinical Benefit, Safety, Pharmacokinetics, and Pharmacodynamics of AT-007 in Pediatric Patients With Classic Galactosemia (CG)

• Status: Completed
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 47 (actual)
• Sponsor: Applied Therapeutics, Inc. · Industry
• Sex: All
• Age: 2 Years – 17 Years
• Healthy volunteers: Not accepted

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).

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 benefit using validated, standardized Clinical Outcome Assessments (COAs) 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 validated, standardized COAs. Open-label extension (OLE) is an active treatment extension for patients who received placebo treatment in Part B.

Conditions

• Classic Galactosemia

Interventions

Type	Name	Description
DRUG	AT-007	Treatment with AT-007 given orally
OTHER	Placebo	Placebo given orally

Timeline

• Start date: 2021-03-20
• Primary completion: 2023-03-03
• Completion: 2025-05-21
• First posted: 2021-05-26
• Last updated: 2025-10-29

Locations

3 sites across 1 country: United States

Regulatory

• FDA-regulated drug study