Trial record 1 of 1 for:    NCT01476163
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Physician Initiated Request for Migalastat in Individual Patients With Fabry Disease

This study is currently recruiting participants. (see Contacts and Locations)
Verified October 2014 by Amicus Therapeutics
Information provided by (Responsible Party):
Amicus Therapeutics Identifier:
First received: November 17, 2011
Last updated: October 20, 2014
Last verified: October 2014

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 Intervention Phase
Fabry Disease
Drug: migalastat HCl
Phase 3

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Physician Initiated Request for Treatment Use of Migalastat Hydrochloride (AT1001), an Investigational Treatment for Individual Patients With Fabry Disease (AT1001-188)

Resource links provided by NLM:

Further study details as provided by Amicus Therapeutics:

Primary Outcome Measures:
  • Incidence of serious adverse events (SAEs), pregnancy [ Time Frame: 12/31/2015 ] [ Designated as safety issue: No ]
    Serious adverse events and reports of pregnancy

Secondary Outcome Measures:
  • Withdrawal from treatment due to adverse events (AEs) [ Time Frame: 12/31/2015 ] [ Designated as safety issue: No ]
    including non-serious and serious AEs

Estimated Enrollment: 20
Study Start Date: December 2011
Estimated Study Completion Date: December 2015
Estimated Primary Completion Date: December 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
migalastat HCI
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
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

Detailed Description:

This Physician Initiated Request program allows physicians to request Amicus permission to receive migalastat HCL for specific patients with Fabry disease who do not meet requirements for participation in an existing migalastat clinical study. Requesting physicians must have served as an investigator in a migalastat HCl 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: Adults; 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.


Ages Eligible for Study:   18 Years to 74 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Confirmed GLA mutation shown to be responsive migalastat in the human embryonic kidney (HEK-293) cell-based assay
  • 18-74 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 with GSK

Exclusion Criteria:

  • Estimated glomerular filtration rate (eGFR) or GFR <30 mL/minute
  • On dialysis
  • Has undergone or is scheduled to undergo kidney or other organ transplant
  • 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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT01476163

Contact: Amicus Therapeutics Patient Advocacy

Australia, Victoria
Royal Melbourne Hospital Recruiting
Parkville, Victoria, Australia, 3050
Contact: Donna North    +61 03 934 24219   
Principal Investigator: Kathleen Nicholls, MD         
Sponsors and Collaborators
Amicus Therapeutics
Study Director: Medical Monitor, Clinical Research Amicus Therapeutics
  More Information

No publications provided

Responsible Party: Amicus Therapeutics Identifier: NCT01476163     History of Changes
Other Study ID Numbers: 116188
Study First Received: November 17, 2011
Last Updated: October 20, 2014
Health Authority: Australia: Department of Health and Ageing Therapeutic Goods Administration

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

Additional relevant MeSH terms:
Fabry Disease
Brain Diseases
Brain Diseases, Metabolic
Brain Diseases, Metabolic, Inborn
Cardiovascular Diseases
Central Nervous System Diseases
Cerebral Small Vessel Diseases
Cerebrovascular Disorders
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Lipid Metabolism Disorders
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Lysosomal Storage Diseases, Nervous System
Metabolic Diseases
Metabolism, Inborn Errors
Nervous System Diseases
Vascular Diseases processed this record on March 26, 2015