Status:
AVAILABLE
Physician Initiated Expanded Access Request for Migalastat in Individual Patients With Fabry Disease
Lead Sponsor:
Amicus Therapeutics
Conditions:
Fabry Disease
Eligibility:
All Genders
2+ years
Brief Summary
This program allows physicians to request permission from Amicus Therapeutics (Amicus) for treatment access to migalastat hydrochloride (HCl) for specific patients with Fabry disease. Treatment is ope...
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 tre...
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
Trial Type :
EXPANDED_ACCESS
End Date :
Estimated Enrollment :
Patients enrolled
Trial Details
Trial ID
NCT01476163
Last Update
July 3 2025
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