Clinical Trial

Testing Experimental Galactosemia Drug in Healthy Subjects

Study Description

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

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.

Location

Locations Selected Location

Methods

Pharmaceutical medication involved Pharmaceutical medication involved
Patients and healthy individuals accepted Patients and healthy individuals accepted

Drug - AT-007

AT-007 will be administered once daily before breakfast. Up to 4 different dose cohorts in part A; up to 4 dose cohorts in part B, up to 2 dose cohorts for part C, and up to 3 dose cohorts for part D will be enrolled in the study. The starting dose in Part A will be 0.5 mg/kg as a single dose. Subsequent doses in Part A and all doses in Parts B, C, and D will be based on the results of previous cohorts and/or previous parts of the study. Part B will start after all subjects in Part A have comple ...read more on ClinicalTrials.org

Drug - Placebo

Matching placebo capsules will be administered once in the morning before breakfast

Additional Information

Official Study Title

A Phase 1-2, Dose-Escalating, 4-Part Study to Evaluate the Safety and Pharmacokinetics of Single and Multiple Doses of AT-007 in Healthy Adult Subjects and Adult Subjects With Classic Galactosemia

Clinical Trial ID

NCT04117711

ParticipAid ID

dJ6o2d