NCT01218659

Study to Compare the Efficacy and Safety of Oral AT1001 and Enzyme Replacement Therapy in Patients With Fabry Disease

A Randomized, Open-Label Study to Compare the Efficacy and Safety of AT1001 and Enzyme Replacement Therapy (ERT) in Patients With Fabry Disease and AT1001-Responsive GLA Mutations, Who Were Previously Treated With ERT

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 68 (actual)
• Sponsor: Amicus Therapeutics · Industry
• Sex: All
• Age: 16 Years – 74 Years
• Healthy volunteers: Not accepted

Summary

Study to compare the efficacy and safety of migalastat and enzyme replacement therapy (ERT) in male and female participants with Fabry disease who are currently receiving ERT and who have an alpha galactosidase-A (α Gal-A) mutation that is amenable to migalastat, based on the clinical trial human embryonic kidney cell (HEK) assay.

Detailed description

This was a Phase 3, randomized, open-label, active-controlled study to evaluate the efficacy and safety of 150 milligrams (mg) of migalastat hydrochloride (migalastat) (equivalent to 123 mg of migalastat) once every other day (QOD) and ERT in male and female participants with Fabry disease who were receiving ERT and who have an α Gal-A mutation that is amenable to migalastat, based on the clinical trial HEK assay. This was a 2-part study. Part 1, the 18-month randomized phase, evaluated participants who received either migalastat 150 mg QOD or ERT per prescribing physicians' instructions for efficacy and safety. Part 2, the optional 12-month open-label extension (OLE) phase in which all participants received migalastat, also explored efficacy and safety. For Part 2, all participants who received ERT in Part 1 were given migalastat. Data presented in this posting include efficacy data from the 18-month randomized period and safety data from the entire study (18-month randomized period and 12-month optional OLE \[total of 30 months\]).

Conditions

• Fabry Disease

Interventions

Type	Name	Description
DRUG	migalastat hydrochloride	150-mg capsule administered orally QOD
BIOLOGICAL	agalsidase	Agalsidase via intravenous infusion as prescribed by the participant's treating physician and in accordance with the approved prescribing information

Timeline

• Start date: 2011-09-08
• Primary completion: 2014-05-27
• Completion: 2015-05-28
• First posted: 2010-10-11
• Last updated: 2018-11-01
• Results posted: 2018-11-01

Locations

25 sites across 10 countries: United States, Australia, Austria, Belgium, Brazil, Denmark, France, Italy, Japan, United Kingdom