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

Study Overview
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Brief Summary
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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
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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.

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

Conditions
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Fabry Disease

Intervention / Treatment
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* Drug: migalastat HCl


Participation Criteria
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Eligibility Criteria
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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.

Ages Eligible for Study
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Minimum Age: 16 Years
Maximum Age: 74 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Intervention/Treatment |
| --- |
|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|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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AT1001, Lysosomal storage disease, Lysosomal Storage Disorder, migalastat, Fabry disease, Fabry, Amicus