Clinical trial NCT05418829: 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)
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE3
Conditions: Classic Galactosemia
Lead sponsor: Applied Therapeutics, Inc.
Sponsor class: INDUSTRY
First posted: 2022-06-14
Enrollment: 7
Sex: ALL
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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

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

Primary outcomes:
measure: To evaluate the long-term safety of 12-month oral administration of AT-007 in adult subjects with CG as assessed by adverse events.
timeFrame: 12 months
measure: 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.
timeFrame: 12 months
measure: 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.
timeFrame: 12 months
Secondary outcomes:
measure: 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
timeFrame: 12 months
measure: To evaluate the pharmacokinetic (PK) parameter maximum concentration (Cmax) of 12-month oral administration of AT-007 in adult subjects with CG
timeFrame: 12 months
measure: 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)
timeFrame: 12 months
Other outcomes:
measure: 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
timeFrame: 12 months
measure: To evaluate the burden of illness (BOI) of adult subjects with CG- Medical History
timeFrame: 12 months
measure: To evaluate the burden of illness (BOI) of adult subjects with CG- NIH Motor Battery
timeFrame: 12 months
measure: To evaluate the burden of illness (BOI) of adult subjects with CG- Spiral Drawing Test
timeFrame: 12 months
measure: To evaluate quality of life (QOL) measures of adult subjects with CG- PROMIS Emotional Distress questionnaire
timeFrame: 12 months
measure: To evaluate quality of life (QOL) measures of adult subjects with CG- PROMIS Emotional Support questionnaire
timeFrame: 12 months
measure: To evaluate quality of life (QOL) measures of adult subjects with CG- PROMIS Companionship questionnaire
timeFrame: 12 months
measure: To evaluate quality of life (QOL) measures of adult subjects with CG- PROMIS Social Isolation questionnaire
timeFrame: 12 months
measure: To evaluate quality of life (QOL) measures of adult subjects with CG- PROMIS Ability to Participate in Social Roles and Activities questionnaire
timeFrame: 12 months
Locations:
city: Berlin
country: United States
facility: Hassman Research Institute
state: New Jersey
Study officials:
References: