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

Phase 1/2 Completed 114 enrolled Applied Therapeutics, Inc.

RandomizedParallel-groupTriple-blindTreatment

Summary

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.

Timeline

Start: 2019-06-21
Primary completion: 2021-12-14
Completion: 2021-12-14

Publications

Perfetti R, Bailey E, Wang S, Mills R, Mohanlal R, Shendelman S. Safety, Pharmacokinetics, and Pharmacodynamics of the New Aldose Reductase Inhibitor Govorestat (AT-007) After a Single and Multiple Doses in Participants in a Phase 1/2 Study. J Clin Pharmacol. 2024 Nov;64(11):1397-1406. doi: 10.1002/jcph.2495. Epub 2024 Jul 10.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Govorestat	Small molecule	0.5 mg/kg	Oral
Subject	Govorestat	Small molecule	5 mg/kg	Oral
Subject	Govorestat	Small molecule	20 mg/kg	Oral
Subject	Govorestat	Small molecule	40 mg/kg	Oral

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

Summary

Timeline

Publications

Drugs

Indications