Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) With Fabry Disease

Exclusion Criteria

• •Subject's last available estimated glomerular filtration rate (eGFR) in the previous study was \< 60 mL/min/1.73 m2
• •Subject had advanced kidney disease requiring dialysis or kidney transplantation
• •Subject received any investigational/experimental drug, biologic, or device within 30 days before baseline, with the exception of migalastat
• •Subject anticipated starting gene therapy during the study period
• •Subject had any intercurrent illness or condition at Visit 1 that may have precluded the subject from fulfilling the protocol requirements or suggested to the investigator that the potential subject may have an unacceptable risk by participating in this study
• •Subject had a history of allergy or sensitivity to migalastat (including excipients) or other iminosugars (eg, miglustat, miglitol)
• •Subject required treatment with Replagal® (agalsidase alfa) or Fabrazyme® (agalsidase beta)
• •Subject required treatment with Glyset® (miglitol) or Zavesca® (miglustat)
• •Female subject was pregnant or breast-feeding, or was planning to become pregnant during the study period
• •In the opinion of the investigator, the subject and/or parent or legally-authorized representative was unlikely or unable to comply with the study requirements