NCT00283933

A 24-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease

A Phase 2, Open-Label, Single Dose Level, 24-Week Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of AT1001 in Patients With Fabry Disease

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 5 (actual)
• Sponsor: Amicus Therapeutics · Industry
• Sex: Male
• Age: 18 Years – 65 Years
• Healthy volunteers: Not accepted

Summary

Study to evaluate the safety, tolerability, and pharmacodynamics of migalastat hydrochloride (HCl) (migalastat) in participants with Fabry disease.

Detailed description

This was a Phase 2, open-label study in male participants with Fabry disease. The study consisted of a 4-week screening period, during which participants' galactosidase (GLA) genotype was assessed for α-galactosidase A (α-Gal A) activity in response to migalastat. Participants were required to have α-Gal A activity responsive to migalastat. The study consisted of a 24-week treatment period, followed by an optional 24-week extension period. Participants received migalastat once every other day (QOD) for 24 weeks during the treatment period and the optional 24-week extension for a total treatment duration of up to 48 weeks.

Conditions

• Fabry Disease

Interventions

Type	Name	Description
DRUG	migalastat HCl

Timeline

• Start date: 2006-05-09
• Primary completion: 2008-03-12
• Completion: 2008-03-12
• First posted: 2006-01-31
• Last updated: 2018-09-07
• Results posted: 2018-09-07

Locations

2 sites across 2 countries: France, United Kingdom