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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
Expanded Access Status : Approved for marketing
First Posted : November 22, 2011
Last Update Posted : January 23, 2019
Sponsor:
Information provided by (Responsible Party):
Amicus Therapeutics

Tracking Information
First Submitted Date November 17, 2011
First Posted Date November 22, 2011
Last Update Posted Date January 23, 2019
 
Descriptive Information
Brief Title Physician Initiated Expanded Access Request for Migalastat in Individual Patients With Fabry Disease
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.
Study Type Expanded Access
Intervention 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
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Expanded Access Status Approved for marketing
Contacts Contact information is only displayed when the study is recruiting subjects
Listed Location Countries Australia,   United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT01476163
Responsible Party Amicus Therapeutics
Study Sponsor Amicus Therapeutics
Collaborators Not Provided
Investigators
Study Director: Medical Monitor, Clinical Research Amicus Therapeutics
PRS Account Amicus Therapeutics
Verification Date January 2019