Trial Entry Criteria

Consult with a healthcare professional to see if you meet the entry criteria.
 

How do I qualify?

Inclusion Criteria:

  • Male or female between the ages of 16 and 74 diagnosed with Fabry disease
  • Confirmed GLA mutation that has shown to be responsive to migalastat HCl (AT1001) in vitro
  • Initiated treatment with agalsidase (ERT) at least 12 months before visit 2 (baseline)
  • Dose level and regiment of agalsidase (ERT) have been stable for the 3 months before visit 2 (baseline) and is at least 80% of the currently labeled dose and regimen for this time period.
  • GFR ≥ 30mL/min/1.73 m2
  • Participants taking angiotensin converting enzyme (ACE) inhibitors or angiotensin receptor blockers (ARBs) must be on a stable dose for at least 4 weeks before visit 1
  • Females who can become pregnant and all men must agree to be sexually abstinent or use medically accepted methods of birth control throughout the duration of the study and for up to 30 days after last dose of study medication
  • Participant is willing and able to provide written informed consent and assent if applicable
 

Exclusion Criteria:

  • Participant has undergone, or is scheduled to undergo, kidney transplantation or any other solid organ transplantation
  • Participant is on regular dialysis that is specifically for the treatment of chronic kidney disease
  • Participant has had a documented transient ischemic attack, stroke, unstable angina, or myocardial infarction within the 3 months before Visit 1
  • Participant has clinically significant unstable cardiac disease in the opinion of the investigator (e.g., cardiac disease requiring active management, such as symptomatic arrhythmia, unstable angina, or NYHA class III or IV congestive heart failure)
  • Pregnant or breast-feeding
  • History of allergy or sensitivity to study medication (including excipients) or other iminosugars (e.g., miglustat, miglitol)
  • Participant has absolute contraindication to iohexol and/or inability to undergo iohexol GFR testing
  • Participant requires treatment with Glyset* (miglitol), or Zavesca* (miglustat)
  • Participant received any investigational/experimental drug, biologic or device within 30 days of Visit 1
  • Any intercurrent illness or condition that may preclude the participant from fulfilling the protocol requirements or suggests to the investigator that the participant may have an unacceptable risk by participating in this study
  • Otherwise unsuitable for the study, in the opinion of the investigator
  * Glyset is a trademark of Bayer company. Zavesca is a trademark of Actelion Pharmaceuticals Ltd.