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

Sponsor

Applied Therapeutics, Inc. (Industry)

Overall Status

Active, not recruiting

CT.gov ID

NCT05418829

Collaborator

(none)

Enrollment

Location

Arm

Anticipated Duration (Months)

0.5

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Classic Galactosemia	Drug: AT-007	Phase 3

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 Design

Study Type:

Interventional

Actual Enrollment :

7 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

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)

Actual Study Start Date :

Oct 1, 2021

Anticipated Primary Completion Date :

Nov 1, 2022

Anticipated Study Completion Date :

Dec 1, 2022

Arms and Interventions

Arm	Intervention/Treatment
Experimental: AT-007 Open-label AT-007 20 mg/kg once daily	Drug: AT-007 AT-007 20 mg/kg once daily

Arm

Intervention/Treatment

Experimental: AT-007

Open-label AT-007 20 mg/kg once daily

Drug: AT-007

AT-007 20 mg/kg once daily

Outcome Measures

Primary Outcome Measures

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 Outcome Measures

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 Outcome Measures

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 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]
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 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 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]

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years to 65 Years

Sexes Eligible for Study:

All

Accepts 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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Hassman Research Institute	Berlin	New Jersey	United States	08009

Sponsors and Collaborators

Applied Therapeutics, Inc.

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

Applied Therapeutics, Inc.

ClinicalTrials.gov Identifier:

NCT05418829

Other Study ID Numbers:

AT-007-1006

First Posted:

Jun 14, 2022

Last Update Posted:

Jun 14, 2022

Last Verified:

Jun 1, 2022

Studies a U.S. FDA-regulated Drug Product:

Yes

Studies a U.S. FDA-regulated Device Product:

Additional relevant MeSH terms:

Galactosemias

Study Results

No Results Posted as of Jun 14, 2022