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

Applied Therapeutics, Inc.7 enrolled

Overview

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.

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 Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Classic Galactosemia

Interventions

AT-007DRUG

AT-007 20 mg/kg once daily

Eligibility

Sex: ALLMin age: 18 YearsMax age: 65 Years

Medical Language ↔ Plain English

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

Locations (1)

Hassman Research Institute

Berlin, New Jersey, United States

Outcomes

Primary Outcomes

To evaluate the long-term safety of 12-month oral administration of AT-007 in adult subjects with CG as assessed by adverse events.

Number of participants with treatment-emergent adverse events and broken down by organ class and relatedness.

Time frame: 12 months

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.

Number of participants with clinically-significant changes in clinical laboratory test results.

Time frame: 12 months

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.

Number of participants with clinically-significant changes in physical examination findings, vital sign abnormalities, and electrocardiogram findings.

Time frame: 12 months

Secondary Outcomes

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

Time frame: 12 months

To evaluate the pharmacokinetic (PK) parameter maximum concentration (Cmax) of 12-month oral administration of AT-007 in adult subjects with CG

Maximum concentration will be determined by individual concentration time-data (mg/L)

Time frame: 12 months

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)

Time frame: 12 months

Data from ClinicalTrials.gov

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