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

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ClinicalTrials.gov Identifier: NCT01476163
Recruitment Status : Available
First Posted : November 22, 2011
Last Update Posted : August 25, 2017
Sponsor:
Information provided by (Responsible Party):

November 17, 2011
November 22, 2011
August 25, 2017
 
Physician Initiated Expanded Access Request for Migalastat in Individual Patients With Fabry Disease
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.
Expanded Access
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
Not Provided
 
Available
Contact: Amicus Therapeutics Patient Advocacy clinicaltrials@amicusrx.com
Australia,   United States
 
 
NCT01476163
Amicus Therapeutics
Amicus Therapeutics
Not Provided
Study Director: Medical Monitor, Clinical Research Amicus Therapeutics
Amicus Therapeutics
August 2017