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

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 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: 16-74 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. Requirements for sufficient kidney function. If permission is granted, initial approval is for a 6 month supply of migalastat HCl with renewal every 6 months available upon meeting continued eligibility.

Approved for marketing
Fabry Disease

Drug: migalastat HCl
150 mg capsule taken every other day by mouth. An inactive reminder capsule may be provided to take on the days in between migalastat HCl
Other Name: AT1001, migalastat

Eligibility 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

Eligibility Gender
Eligibility Age
Minimum: 16 Years~Maximum: 74 Years
United States

CHOC Children's Hospital Division of Metabolic Disease
Orange, California, 92868

University of Maryland Medical Center
Baltimore, Maryland, 21201

Kidney Care and Transplant Services of New England
Springfield, Massachusetts, 01104

Icahn School of Medicine at Mount Sinai
New York, New York, 10029

Medical Monitor, Clinical Research
Study Director
Amicus Therapeutics

Amicus Therapeutics
NCT Number
Lysosomal storage disease
Lysosomal Storage Disorder
Fabry disease
MeSH Terms
Fabry Disease