MedTrace Logo

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

NCT04902781 · Status: COMPLETED · Phase: PHASE2/PHASE3 · Type: INTERVENTIONAL · Enrollment: 47

Last updated 2025-10-29

No results posted yet for this study

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).

Conditions

  • Classic Galactosemia

Interventions

DRUG

AT-007

Treatment with AT-007 given orally

OTHER

Placebo

Placebo given orally

Sponsors & Collaborators

  • Applied Therapeutics, Inc.

    lead INDUSTRY

Principal Investigators

  • Susan E Waisbren, PhD · Harvard University

  • Jonathan W Mink, MD · University of Rochester

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
2 Years
Max Age
17 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-03-20
Primary Completion
2023-03-03
Completion
2025-05-21
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04902781 on ClinicalTrials.gov