Physician Initiated Expanded Access Request for Treatment Use of Migalastat Hydrochloride (AT1001), an Investigational Treatment for Individual Patients With Fabry Disease (AT1001-188)

To request permission from Amicus Therapeutics (Amicus) for treatment access to migalastat hydrochloride (HCl) for specific adult patients with Fabry disease

CT.gov Identifier
NCT01476163
EudraCT Identifier
N/A
CTIS:
N/A
Sponsor
Amicus Therapeutics
Collaborator
N/A
Study Contact Information
+1 609 662 2000
Recruiting
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Study Details

Diseases
Fabry Disease
Study Drug
Migalastat hydrochloride (capsule)
Genes
GLA
Study Dates
Nov 2011 - Oct 2016
Sex
Female & Male
Age
2+ years
Inclusion and Exclusion Criteria
Inclusion Criteria:
  • Confirmed GLA mutation predicted to be responsive migalastat in the human embryonic kidney (HEK-293) cell-based assay
  • At least 2 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:
  • Scheduled for renal or other organ transplant or replacement therapy
  • Receiving GLYSET® (miglitol), ZAVESCA® (miglustat) or enzyme replacement therapy FABRAZYME® (agalsidase beta), REPLAGAL™ (agalsidase alpha), or Elfabrio® (pegunigalsidase alfa)
  • 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.

Protocol Summary

To request permission from Amicus Therapeutics (Amicus) for treatment access to migalastat hydrochloride (HCl) for specific adult patients with Fabry disease

Study Locations

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