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

• ClinicalTrials.gov Identifier: NCT05418829
• Recruitment Status: Active, not recruiting
• First Posted: June 14, 2022
• Last Update Posted: June 14, 2022
• Sponsor: Applied Therapeutics, Inc.
• Information provided by (Responsible Party): Applied Therapeutics, Inc.

Tracking Information

• First Submitted Date: February 16, 2022
• First Posted Date: June 14, 2022
• Last Update Posted Date: June 14, 2022
• Actual Study Start Date: October 1, 2021
• Estimated Primary Completion Date: November 2022 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: June 9, 2022)

• To evaluate the long-term safety of 12-month oral administration of AT-007 in adult subjects with CG as assessed by adverse events. [ Time Frame: 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. [ Time Frame: 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. [ Time Frame: 12 months ]
  Number of participants with clinically-significant changes in physical examination findings, vital sign abnormalities, and electrocardiogram findings.

• Original Primary Outcome Measures: Same as current
• Change History: No Changes Posted

Current Secondary Outcome Measures (submitted: June 9, 2022)

• 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 [ Time Frame: 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) [ Time Frame: 12 months ]

• Original Secondary Outcome Measures: Same as current

Current Other Pre-specified Outcome Measures (submitted: June 9, 2022)

• 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 [ Time Frame: 12 months ]
• To evaluate the burden of illness (BOI) of adult subjects with CG- Medical History [ Time Frame: 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 [ Time Frame: 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 [ Time Frame: 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 [ Time Frame: 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 [ Time Frame: 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 [ Time Frame: 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 [ Time Frame: 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 [ Time Frame: 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]

• Original Other Pre-specified Outcome Measures: Same as current

Descriptive Information

• Brief Title: AT-007 in Adult Subjects With Classic Galactosemia (CG)
• 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)

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

• Study Type: Interventional
• Study Phase: Phase 3
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: Classic Galactosemia

Intervention

Drug: AT-007

AT-007 20 mg/kg once daily

Study Arms

Experimental: AT-007

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

Intervention: Drug: AT-007

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Active, not recruiting
• Actual Enrollment (submitted: June 9, 2022): 7
• Original Actual Enrollment: Same as current
• Estimated Study Completion Date: December 2022
• Estimated Primary Completion Date: November 2022 (Final data collection date for primary outcome measure)

Eligibility Criteria

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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years to 65 Years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT05418829
• Other Study ID Numbers: AT-007-1006
• Has Data Monitoring Committee: No

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

• IPD Sharing Statement: Not Provided
• Current Responsible Party: Applied Therapeutics, Inc.
• Original Responsible Party: Same as current
• Current Study Sponsor: Applied Therapeutics, Inc.
• Original Study Sponsor: Same as current
• Collaborators: Not Provided
• Investigators: Not Provided
• PRS Account: Applied Therapeutics, Inc.
• Verification Date: June 2022