NCT05418829

AT-007 in Adult Subjects With Classic Galactosemia (CG)

An Open-Label Study to Evaluate the Long-Term Safety and Pharmacodynamic Efficacy of AT-007 in Adult Subjects With Classic Galactosemia (CG)

• Status: Unknown
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 7 (actual)
• Sponsor: Applied Therapeutics, Inc. · Industry
• Sex: All
• Age: 18 Years – 65 Years
• Healthy volunteers: Not accepted

Summary

This study is a 12-month open-label extension (OLE) study of AT-007 in adult subjects with CG who previously participated in Study AT-007-1001 Part D and/or Part D Extension. The study is designed to assess the long-term safety of AT-007 in subjects with CG as well as the pharmacodynamics (PD) (inhibition of galactitol) and PK of AT-007. The effect of 12-month treatment with AT-007 on the levels of galactose and other galactose metabolites in subjects with CG will also be evaluated.

Detailed description

Primary: \- To evaluate the long-term safety of 12-month oral administration of AT-007 in adult subjects with Classic Galactosemia (CG) Secondary: * To evaluate the long-term inhibition of galactitol, a biomarker of aldose reductase (AR) activity, induced by 12-month oral administration of AT-007 to adult subjects with CG * To evaluate the pharmacokinetic (PK) parameters of 12-month oral administration of AT-007 in adult subjects with CG Exploratory: * To evaluate the effect of 12-month oral administration of AT-007 on the levels of galactose and its other metabolites in adult subjects with CG * To evaluate the burden of illness (BOI) of adult subjects with CG * To evaluate quality of life (QOL) measures of adult subjects with CG * To evaluate the effect of 12-month oral administration of AT-007 on the BOI and QOL of adult subjects with CG

Conditions

• Classic Galactosemia

Interventions

Type	Name	Description
DRUG	AT-007	AT-007 20 mg/kg once daily

Timeline

• Start date: 2021-10-01
• Primary completion: 2022-11-01
• Completion: 2022-12-01
• First posted: 2022-06-14
• Last updated: 2022-06-14

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study