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

NCT ID: NCT05418829

Last Updated: 2022-06-14

Basic Information

• Recruitment Status: UNKNOWN
• Clinical Phase: PHASE3
• Total Enrollment: 7 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2021-10-01
• Study Completion Date: 2022-12-31

Brief 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

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

AT-007

Open-label AT-007 20 mg/kg once daily

Group Type: EXPERIMENTAL

AT-007

Intervention Type: DRUG

AT-007 20 mg/kg once daily

Interventions

AT-007

AT-007 20 mg/kg once daily

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

• Male or non-pregnant, non-lactating female subject with a CG diagnosis
• Previous participation in Study AT-007-1001 Part D and/or Part D Extension
• No other significant health problems which preclude participation

Exclusion Criteria

• Concomitant use of certain medications or over-the-counter therapies
• Discontinuation from Study AT-007-1001 Part D and/or Part D Extension due an adverse event

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 65 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Applied Therapeutics, Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Hassman Research Institute

Berlin, New Jersey, United States

Countries

United States

Other Identifiers

AT-007-1006

Identifier Type: -

Identifier Source: org_study_id