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

Phase 3

• Conditions: Classic Galactosemia
• Interventions: Drug: AT-007

• Registration Number: NCT05418829
• Lead Sponsor: Applied Therapeutics, Inc.

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

Study Timeline

• Study Start: 2021-10-01
• Study First Submitted: 2022-02-16
• Status Verified: 2022-06
• Study First Submitted QC: 2022-06-09
• Last Update Submitted: 2022-06-09
• Study First Posted: 2022-06-14
• Last Update Posted: 2022-06-14
• Primary Completion: 2022-11
• Study Completion: 2022-12

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 7

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
AT-007	AT-007	Open-label AT-007 20 mg/kg once daily

Primary Outcome Measures

Name	Time	Method
To evaluate the long-term safety of 12-month oral administration of AT-007 in adult subjects with CG as assessed by physical exam parameters.	12 months	Number of participants with clinically-significant changes in physical examination findings, vital sign abnormalities, and electrocardiogram findings.
To evaluate the long-term safety of 12-month oral administration of AT-007 in adult subjects with CG as assessed by adverse events.	12 months	Number of participants with treatment-emergent adverse events and broken down by organ class and relatedness.
To evaluate the long-term safety of 12-month oral administration of AT-007 in adult subjects with CG as assessed by clinical laboratory test results.	12 months	Number of participants with clinically-significant changes in clinical laboratory test results.

Secondary Outcome Measures

Name	Time	Method
To evaluate the pharmacokinetic (PK) parameter maximum concentration (Cmax) of 12-month oral administration of AT-007 in adult subjects with CG	12 months	Maximum concentration will be determined by individual concentration time-data (mg/L)
To evaluate the pharmacokinetic (PK) parameter Area-Under-the-Curve (AUC) of 12-month oral administration of AT-007 in adult subjects with CG (mg*h/L)	12 months
To evaluate the long-term change of galactitol, a biomarker of AR activity, induced by 12-month oral administration of AT-007 to adult subjects with CG	12 months

Trial Locations

Locations (1)

Hassman Research Institute; 🇺🇸 Berlin, New Jersey, United States