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

Applied Therapeutics

Status and phase: Active, not recruiting; Phase 3

Conditions: Classic Galactosemia

Treatments: Drug: AT-007

Study type: Interventional

Funder types: Industry

Identifiers

NCT05418829

AT-007-1006

Details and patient eligibility

About

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.

Full 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

Enrollment: 7 patients

Sex: All

Ages: 18 to 65 years old

Volunteers: No 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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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7 participants in 1 patient group

AT-007

Experimental group

Description:

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

Treatment:

Drug: AT-007