Fabry Disease Clinical Trial
Official title:
A Phase 1 Study to Investigate the Absorption, Metabolism and Excretion of [14C] AT1001 (Migalastat Hydrochloride) Following a Single Oral Administration in Healthy Volunteers (AT1001-014)
This study is designed to describe the metabolism of AT1001 (migalastat HCl) and the
contribution of metabolism and urinary excretion to its overall elimination as part of the
continuing assessment of the safety and effectiveness of the drug.
This is a Phase 1, single-site, open-label, single dose study of the absorption, metabolism
and excretion of radiolabeled AT1001 in healthy male subjects between 30 and 55 years of
age, inclusive. Six subjects will be dosed, with the goal of having at least 4 subjects
complete the study through follow-up. All subjects will be screened within 28 days before
admission to the Clinical Unit. Subjects will be confined to the clinical unit for 10 days
after dosing and will return to the clinic for a follow-up visit 28 days after dosing.
Each subject will receive a single oral dose of AT1001 as an aqueous solution containing 150
mg [14C] AT1001 (1 μCi). Blood, duodenal bile, expired air, urine, and feces samples will be
collected at specified time points after dosing throughout the period of confinement at the
study site. Safety will be assessed throughout the study by monitoring clinical laboratory
tests, ECGs, physical examinations, vital signs, and adverse events. The total duration of
the study for each subject is approximately 8 weeks.
n/a
Endpoint Classification: Pharmacokinetics Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
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