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

Classic Galactosemia Clinical Trial

Official title:

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT05418829
• Other study ID #: AT-007-1006
• Status: Active, not recruiting
• Phase: Phase 3
• Start date: October 1, 2021
• Est. completion date: December 2022

Study information

• Verified date: June 2022
• Source: Applied Therapeutics, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 7
• Est. completion date: December 2022
• Est. primary completion date: November 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 65 Years

Eligibility

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

Related Conditions & MeSH terms

• Classic Galactosemia
• Galactosemias

Intervention

Drug:
• AT-007
AT-007 20 mg/kg once daily

Locations

Country	Name	City	State
United States	Hassman Research Institute	Berlin	New Jersey

Sponsors (1)

Lead Sponsor	Collaborator
Applied Therapeutics, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	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
Other	To evaluate the burden of illness (BOI) of adult subjects with CG- Medical History	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)	12 months
Other	To evaluate the burden of illness (BOI) of adult subjects with CG- NIH Motor Battery	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.	12 months
Other	To evaluate the burden of illness (BOI) of adult subjects with CG- Spiral Drawing Test	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.	12 months
Other	To evaluate quality of life (QOL) measures of adult subjects with CG- PROMIS Emotional Distress questionnaire	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]	12 months
Other	To evaluate quality of life (QOL) measures of adult subjects with CG- PROMIS Emotional Support questionnaire	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]	12 months
Other	To evaluate quality of life (QOL) measures of adult subjects with CG- PROMIS Companionship questionnaire	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]	12 months
Other	To evaluate quality of life (QOL) measures of adult subjects with CG- PROMIS Social Isolation questionnaire	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]	12 months
Other	To evaluate quality of life (QOL) measures of adult subjects with CG- PROMIS Ability to Participate in Social Roles and Activities questionnaire	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]	12 months
Primary	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
Primary	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
Primary	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	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
Secondary	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
Secondary	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