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



Status:Approved for marketing
Conditions:Hematology, Metabolic
Therapuetic Areas:Hematology, Pharmacology / Toxicology
Healthy:No
Age Range:16 - 74
Updated:1/25/2019

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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)

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.

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.

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.
We found this trial at
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Parkville, Victoria
Principal Investigator: Kathleen Nicholls, MD
Phone: +61 03 934 24219
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22 S Greene St
Baltimore, Maryland 21201
(410) 328-8667
Phone: 410-706-6236
University of Maryland Medical Center Founded in 1823 as the Baltimore Infirmary, the University of...
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1428 Madison Ave
New York, New York 10029
(212) 241-6500
Phone: 212-241-0915
Icahn School of Medicine at Mount Sinai Icahn School of Medicine at Mount Sinai is...
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Orange, California 92868
Principal Investigator: Raymond Y Wang, MD
Phone: 714-509-3008
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Orange, CA
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100 Wason Avenue
Springfield, Massachusetts 01104
Phone: 413-733-0010
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Springfield, MA
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