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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT01476163
Recruitment Status : Available
First Posted : November 22, 2011
Last Update Posted : August 25, 2017
Information provided by (Responsible Party):

Study Description
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.

Condition or disease Intervention/treatment
Fabry Disease Drug: migalastat HCl

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 Design

Study Type : Expanded Access
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)

Resource links provided by the National Library of Medicine

U.S. FDA Resources


Intervention Details:
    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

Information from the National Library of Medicine

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Ages Eligible for Study:   16 Years to 74 Years   (Child, Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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.
Contacts and Locations

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01476163

Contact: Amicus Therapeutics Patient Advocacy clinicaltrials@amicusrx.com

United States, California
CHOC Children's Hospital Division of Metabolic Disease
Orange, California, United States, 92868
Contact: Nina Movsesyan, PhD    714-509-3008    nmovsesyan@CHOC.org   
Principal Investigator: Raymond Y Wang, MD         
United States, Maryland
University of Maryland Medical Center
Baltimore, Maryland, United States, 21201
Contact: Elizabeth A Streeten, MD    410-706-6236    estreete@som.umaryland.edu   
United States, Massachusetts
Kidney Care and Transplant Services of New England
Springfield, Massachusetts, United States, 01104
Contact: Gregory L Braden, MD    413-733-0010    Gregory.Braden@baystatehealth.org   
Contact: Ann Vasseur    (413) 733-0010 ext 143    ann@kidneycare-ne.com   
United States, New York
Icahn School of Medicine at Mount Sinai
New York, New York, United States, 10029
Contact: Amy C Yang, MD    212-241-6770    amy.yang@mssm.edu   
Contact: Chanan Stauffer, MS, RN    703-597-6777    Chanan78@gmail.com   
Australia, Victoria
Royal Melbourne Hospital
Parkville, Victoria, Australia, 3050
Contact: Donna North    +61 03 934 24219    donna.north@mh.org.au   
Principal Investigator: Kathleen Nicholls, MD         
Sponsors and Collaborators
Amicus Therapeutics
Study Director: Medical Monitor, Clinical Research Amicus Therapeutics
More Information

Responsible Party: Amicus Therapeutics
ClinicalTrials.gov Identifier: NCT01476163     History of Changes
Other Study ID Numbers: AT1001-188
First Posted: November 22, 2011    Key Record Dates
Last Update Posted: August 25, 2017
Last Verified: August 2017

Keywords provided by Amicus Therapeutics:
Lysosomal storage disease
Lysosomal Storage Disorder
Fabry disease

Additional relevant MeSH terms:
Fabry Disease
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Cerebral Small Vessel Diseases
Cerebrovascular Disorders
Vascular Diseases
Cardiovascular Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Metabolism, Inborn Errors
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders