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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. ;


Study Design


Related Conditions & MeSH terms


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

See also
  Status Clinical Trial Phase
Active, not recruiting NCT05418829 - AT-007 in Adult Subjects With Classic Galactosemia (CG) Phase 3
Enrolling by invitation NCT03838016 - Preventing Speech and Language Disorders in Children With Classic Galactosemia N/A
Completed NCT04117711 - Safety and Pharmacokinetics of AT-007 in Healthy Subjects and in Adult Subjects With Classic Galactosemia Phase 1/Phase 2
Completed NCT03580122 - The Effect of Arginine on Classic Galactosemia Phase 2