Physician Initiated Expanded Access Request for Migalastat in Individual Patients With Fabry Disease
ConditionFabry Disease
Trial statusAvailable
Trial phaseNot listed
Trial typeExpanded Access
Biological sexAll
Age2+
SponsorAmicus Therapeutics
About this trial
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 open label.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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
Disqualifiers
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
Trial design
Treatments tested in this trial
migalastat HCl 150 mg
Drug150 mg capsule
migalastat HCl 20 mg
Drug20 mg dispersible tablets; dosing will be based on body weight
Treatment groups
No treatment groups listed
Trial outcomes
This trial has no outcomes listed.Locations
Sponsors and contacts
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Source ClinicalTrials.gov