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

NCT05418829 | Unknown Phase 3 FDA Drug

• 1 other identifier: AT-007-1006
• Study Type: interventional
• Target: 7
• Locations: 1 country
• Sites: 1

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.

Conditions

Classic Galactosemia

Outcome Measures

Primary Outcomes (3)

• 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 Outcomes (3)

• 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

• 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 Outcomes (9)

• 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

• To evaluate the burden of illness (BOI) of adult subjects with CG- NIH Motor Battery

  12 months

Study Arms (1)

AT-007

EXPERIMENTAL

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

Drug: AT-007

Interventions

AT-007 DRUG

AT-007 20 mg/kg once daily

AT-007

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Applied Therapeutics, Inc.: lead

Study Sites (1)

Hassman Research Institute

Berlin, New Jersey, 08009, United States

Location

MeSH Terms

Conditions

Galactosemias

Condition Hierarchy (Ancestors)

Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Carbohydrate Metabolism, Inborn Errors; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 16, 2022
• First Posted: June 14, 2022
• Study Start: October 1, 2021
• Primary Completion: November 1, 2022
• Study Completion: December 1, 2022
• Last Updated: June 14, 2022

Record last verified: 2022-06

Locations

US (1)