Open-Label Phase 3 Long-Term Safety Study of Migalastat (MGM116041)

• Incidence of adverse events (AEs) [ Time Frame: Up to 5 years ] [ Designated as safety issue: No ]
  non-serious AEs, serious AEs, and possible suicidality related AEs
  
  
• Withdrawal from treatment due to AEs [ Time Frame: Up to 5 years ] [ Designated as safety issue: No ]
  including non-serious and serious AEs
  
  
• Change from baseline in vital signs [ Time Frame: Up to 5 years ] [ Designated as safety issue: No ]
  blood pressure, heart rate
  
  
• Change from baseline in laboratory parameters [ Time Frame: Up to 5 years ] [ Designated as safety issue: No ]
  blood chemistry, hematology, urinalysis
  
  
• Change from baseline in echocardiogram (ECG) [ Time Frame: Up to 5 years ] [ Designated as safety issue: No ]
  12-lead ECG
  
  
• Change from baseline in electrocardiography (ECHO) [ Time Frame: Up to 5 years ] [ Designated as safety issue: No ]
  ECHO ultrasound
  
  

• Estimated glomerular filtration rate [ Time Frame: Up to 5 years ] [ Designated as safety issue: No ]
  based on the Modification of Diet in Renal Disease equation
  
  
• Measurement of 24-hour urine [ Time Frame: Up to 5 years ] [ Designated as safety issue: No ]
  protein, albumin, creatinine and globotriaosylceramide (GL-3)
  
  
• Evaluation of left ventricular mass index and ejection fraction [ Time Frame: Up to 5 years ] [ Designated as safety issue: No ]
  as measured by echocardiography
  
  
• Evaluation of leukocyte alpha-galactosidase A (a-Gal A) activity [ Time Frame: Up to 5 years ] [ Designated as safety issue: No ]
  enzyme responsible for breaking down GL-3
  
  
• Evaluation of patient reported assessment of pain [ Time Frame: Up to 5 years ] [ Designated as safety issue: No ]
  as assessed by the Brief Pain Inventory short form
  
  
• Evaluation of patient reported Quality of Life [ Time Frame: Up to 5 years ] [ Designated as safety issue: No ]
  as assessed by the Short Form-36 survey
  
  

MGM116041 is an open-label, non-comparative, multicenter, long term extension study for subjects with Fabry disease who have completed treatment in a previous trial of migalastat HCl (also known as AT1001) given as monotherapy. This is an extension study designed to evaluate the long-term safety and efficacy of migalastat HCl for the treatment of Fabry disease. Study visits will occur every 6 months to evaulate safety and assess efficacy. Vist evaluations will include physical examination, clinical laboratory parameters, adverse events and patient reported outcomes.