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

• Status: Approved for marketing
• Trial end date: 1969-12-31
• Target enrollment: 0
• Participant gender: All

Summary

This program allows physicians to request permission from Amicus Therapeutics (Amicus) for treatment access to migalastat hydrochloride (HCl) for specific adult patients with Fabry disease. Treatment is open label for 6 months with renewal every 6 months.

• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Amicus Therapeutics

Criteria

Inclusion Criteria:

• Confirmed GLA mutation predicted to be responsive migalastat in the human embryonic kidney (HEK-293) cell-based assay

• 16-74 years of age

• Strong clinical indication for treatment of Fabry disease

• No other treatment option including either unsuitable for ERT or unable to access ERT

• Appropriate female and male contraception

• Willing to receive treatment with migalastat HCl via this program including having signed an authorization for sharing clinical data

Exclusion Criteria:

• Estimated glomerular filtration rate (eGFR) or GFR <30 mL/minute

• Scheduled for renal or other organ transplant or replacement therapy

• Receiving GLYSET® (miglitol), ZAVESCA® (miglustat) or enzyme replacement therapy FABRAZYME® (agalsidase beta) or REPLAGAL™ (agalsidase alpha)

• Contraindication to migalastat, i.e., sensitivity to other iminosugar such as miglustat, miglitol

• Treated with another investigational drug within 30 days of start of migalastat HCl treatment

• Unable to comply with study requirements or deemed otherwise unsuitable for study entry in the opinion of the investigator.