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

Phases

Phase 3

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT05418829

Enrollment

Registered

2022-06-14

Start date

2021-10-01

Completion date

2022-12-31

Last updated

2022-06-14

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Classic Galactosemia

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

Interventions

DRUGAT-007

AT-007 20 mg/kg once daily

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

18 Years to 65 Years

Healthy volunteers

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

Design outcomes

Primary

Measure	Time frame	Description
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.
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.

Secondary

Measure	Time frame	Description
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	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	—

Other

Measure	Time frame	Description
To evaluate quality of life (QOL) measures of adult subjects with CG- PROMIS Companionship questionnaire	12 months	The questionnaire assesses this function with higher scores relating to better function and may be used to compare over time for change from baseline to month 12. \[PROMIS: Patient Reported Outcome Measure Information System\]
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	12 months	—
To evaluate quality of life (QOL) measures of adult subjects with CG- PROMIS Ability to Participate in Social Roles and Activities questionnaire	12 months	The questionnaire assesses this function with higher scores relating to better function and may be used to compare over time for change from baseline to month 12. \[PROMIS: Patient Reported Outcome Measure Information System\]
To evaluate quality of life (QOL) measures of adult subjects with CG- PROMIS Social Isolation questionnaire	12 months	The questionnaire assesses this function with higher scores relating to better function and may be used to compare over time for change from baseline to month 12. \[PROMIS: Patient Reported Outcome Measure Information System\]
To evaluate the burden of illness (BOI) of adult subjects with CG- Medical History	12 months	Narrative description of natural course of CG as extracted by chart review and physical exam findings noted in chart review including the baseline neurologic exam upon entering the clinical trial. (There are no units for a descriptive narrative of medical history)
To evaluate the burden of illness (BOI) of adult subjects with CG- NIH Motor Battery	12 months	NIH Motor Battery produces normalized percentile scores with higher scores being more normal. As an exploratory endpoint, changes in the score from baseline to month 12 will assessed.
To evaluate the burden of illness (BOI) of adult subjects with CG- Spiral Drawing Test	12 months	Spiral Drawing Test produces normalized percentile scores with higher scores being more normal. As an exploratory endpoint, changes in the score from baseline to month 12 will assessed.
To evaluate quality of life (QOL) measures of adult subjects with CG- PROMIS Emotional Distress questionnaire	12 months	The questionnaire assesses this function with higher scores relating to better function and may be used to compare over time for change from baseline to month 12. \[PROMIS: Patient Reported Outcome Measure Information System\]
To evaluate quality of life (QOL) measures of adult subjects with CG- PROMIS Emotional Support questionnaire	12 months	The questionnaire assesses this function with higher scores relating to better function and may be used to compare over time for change from baseline to month 12. \[PROMIS: Patient Reported Outcome Measure Information System\]

Countries

United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026