NCT01476163

Physician Initiated Expanded Access Request for Migalastat in Individual Patients With Fabry Disease

Physician Initiated Expanded Access Request for Treatment Use of Migalastat Hydrochloride (AT1001), an Investigational Treatment for Individual Patients With Fabry Disease (AT1001-188)

• Status: Available
• Phase: —
• Study type: Expanded Access
• Enrollment: —
• Sponsor: Amicus Therapeutics · Industry
• Sex: All
• Age: 2 Years
• Healthy volunteers: Not accepted

Summary

This program allows physicians to request permission from Amicus Therapeutics (Amicus) for treatment access to migalastat hydrochloride (HCl) for specific patients with Fabry disease. Treatment is open label.

Detailed description

This Physician Initiated Request program allows physicians to request permission from Amicus to receive migalastat HCl for specific patients with Fabry disease who have a mutation amenable to this treatment, who do not have access to commercial Galafold or available treatment alternatives, or do not meet requirements for participation in an existing migalastat clinical study. Up to 20 patients worldwide may be treated. Patients must meet specific criteria to receive Amicus permission for participation. Key criteria for participation include: At least 2 years old; Confirmed GLA gene mutation shown to be responsive to migalastat; Have no treatment option because either unsuitable for enzyme replacement therapy (ERT) or unable to access ERT.

Conditions

• Fabry Disease

Interventions

Type	Name	Description
DRUG	migalastat HCl 150 mg	150 mg capsule
DRUG	migalastat HCl 20 mg	20 mg dispersible tablets; dosing will be based on body weight

Timeline

• First posted: 2011-11-22
• Last updated: 2025-07-03