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

Study Overview
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Brief Summary
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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
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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

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

Conditions
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Classic Galactosemia

Intervention / Treatment
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* Drug: AT-007


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 65 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: AT-007<br>Open-label AT-007 20 mg/kg once daily | Drug: AT-007<br>* AT-007 20 mg/kg once daily<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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. | 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. | 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. | 12 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 | 
| 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) | 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) |  | 12 months |