NCT01196871

Drug-Drug Interaction Study Between AT1001 (Migalastat Hydrochloride) and Agalsidase in Participants With Fabry Disease

An Open-label Phase 2A Study to Investigate Drug-Drug Interactions Between AT1001 (Migalastat Hydrochloride) and Agalsidase in Subjects With Fabry Disease

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 20 (actual)
• Sponsor: Amicus Therapeutics · Industry
• Sex: Male
• Age: 18 Years – 65 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

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.

Conditions

• Fabry Disease

Interventions

Type	Name	Description
DRUG	Migalastat HCl	Oral capsules, single dose
BIOLOGICAL	Agalsidase Beta	IV infusion, single dose
BIOLOGICAL	Agalsidase Alfa	IV infusion, single dose

Timeline

• Start date: 2011-02-02
• Primary completion: 2012-10-09
• Completion: 2012-10-09
• First posted: 2010-09-09
• Last updated: 2018-12-19
• Results posted: 2018-11-01

Locations

10 sites across 5 countries: United States, Australia, Belgium, Canada, Netherlands