Safety and Pharmacokinetics of AT-007 in Healthy Subjects and in Adult Subjects With Classic Galactosemia

Applied Therapeutics

Status and phase: Completed; Phase 2; Phase 1

Conditions: Classic Galactosemia

Treatments: Drug: Placebo; Drug: AT-007

Study type: Interventional

Funder types: Industry

Identifiers

NCT04117711

AT-007-1001

Details and patient eligibility

About

This study is a first-in-human, randomized, placebo-controlled, 4-Part, single ascending dose (SAD) and multiple ascending dose (MAD) study in healthy adult subjects and adult subjects with Classic Galactosemia.

Full description

The study is designed to assess the safety and PK of AT-007 in healthy subjects and subjects with Classic Galactosemia as well as the effect of AT-007 on biomarkers of galactose metabolism (galactose, galactitol, and other galactose metabolites) in subjects with Classic Galactosemia.

This study consists of 4 parts:

• Part A (SAD) in 32 healthy subjects. Once daily oral escalating dose (6 active, 2 placebo).
• Part B and C (MAD for 7 days) in 36 healthy subjects. Once daily multiple daily dosing (8 active, 2 placebo per each dose cohort).
• Part D (SAD followed by MAD for 27 days) in 18 subjects with Classic Galactosemia. Once daily followed by multiple daily oral dosing (6 active, 2 placebo for each dose cohort).

Enrollment: 114 patients

Sex: All

Ages: 18 to 55 years old

Volunteers: Accepts Healthy Volunteers

Inclusion criteria

• Diagnosis of Classic Galactosemia confirmed by evidence of absent or significantly decreased (<1%) GALT activity in red blood cells and by GALT gene analysis
• Urine galactitol >100 mmol/mol creatinine
• Galactose-restricted diet

Exclusion criteria

• Complications of CG resulting in disability that, in the opinion of the Investigator, may prevent the subject from completing all study requirements (e.g., severe neurological deficits, severe cognitive impairment, or severe language difficulty).
• Renal disease (eGFR < 90 mL/min/1.73 m2 or albuminuria).

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Triple Blind

---

114 participants in 2 patient groups, including a placebo group

AT-007

Experimental group

Description:

AT-007 is a CNS and retina penetrant aldose reductase inhibitor.

Treatment:

Drug: AT-007

Placebo Comparator

Placebo Comparator group

Description:

Placebo is used as a comparator to the experimental arm.

Treatment:

Drug: Placebo