Fabry Disease Clinical Trial
Official title:
An Open-label Phase 2A Study to Investigate Drug-Drug Interactions Between AT1001 (Migalastat Hydrochloride) and Agalsidase in Subjects With Fabry Disease
The objective was to determine the effects of a single dose of migalastat hydrochloride (HCl) (migalastat) 150 and 450 milligrams (mg) on the safety and plasma pharmacokinetics (PK) of agalsidase and the effects of agalsidase on the safety and PK of migalastat 150 mg.
This open-label study was conducted in 2 stages (Stage 1, Stage 2). Stage 1 included
migalastat 150 mg; Stage 2 included migalastat 450 mg. Each dose of migalastat was selected
to evaluate interaction with each of 3 doses of recombinant agalsidase: 0.5 mg/kilogram (kg)
agalsidase beta; 1.0 mg/kg agalsidase beta; 0.2 mg/kg agalsidase alfa.
Migalastat was administered orally. Agalsidase alfa was administered as a 40-minute
intravenous (IV) infusion and agalsidase beta was administered as a 2-hour (hr) IV infusion.
Stage 1 consisted of 3 treatment periods with 14 days intervening between each period.
Period 1, Day 1: agalsidase was administered alone.
Period 2, Day 1: migalastat was administered, followed 2 hrs later by agalsidase.
Period 3, Day 7: migalastat was administered alone.
Stage 2 consisted of two 14-day treatment periods in which the plasma exposure of migalastat
was characterized when migalastat was administered with agalsidase solely to confirm the
attainment of adequate migalastat plasma concentrations.
Period 1, Day 1: agalsidase was administered as an IV infusion using a calibrated infusion
pump.
Period 2, Day 1: migalastat was administered, followed 2 hrs later by agalsidase.
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