Study to Compare the Efficacy and Safety of Oral AT1001 and Enzyme Replacement Therapy in Patients With Fabry Disease
Condition: Fabry Disease
MeSH terms: Fabry Disease
Intervention: migalastat hydrochloride; agalsidase
Intervention description: 150-mg capsule administered orally QOD; Agalsidase via intravenous infusion as prescribed by the participant's treating physician and in accordance with the approved prescribing information
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Enrollment: 68.0
Sex: All
Minimum age: 16 Years
Maximum age: 74 Years
Healthy volunteers: No
Keywords: Amicus Therapeutics; Galafold; AT1001; Migalastat

Summary:

      Study to compare the efficacy and safety of migalastat and enzyme replacement therapy (ERT)
      in male and female participants with Fabry disease who are currently receiving ERT and who
      have an alpha galactosidase-A (α Gal-A) mutation that is amenable to migalastat, based on the
      clinical trial human embryonic kidney cell (HEK) assay.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Male or female between the ages of 16 and 74 diagnosed with Fabry disease

          -  Confirmed α Gal-A mutation that is amenable to migalastat, based on the clinical trial
             HEK assay

          -  Participant has been on ERT for at least 12 months before screening/baseline

          -  Dose level and regimen of ERT have been stable for 3 months before screening/baseline
             and is at least 80% of the currently labeled dose and regimen for this time period

          -  Glomerular filtration rate (GFR) ≥ 30 milliliter (mL)/minute (min) /1.73 m^2

          -  Participants taking angiotensin converting enzyme inhibitors or angiotensin receptor
             blockers must be on a stable dose for at least 4 weeks before screening/baseline

          -  Women who can become pregnant and all men 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 screening/baseline

          -  Participant has clinically significant unstable cardiac disease in the opinion of the
             investigator (for example, cardiac disease requiring active management, such as
             symptomatic arrhythmia, unstable angina, or New York Heart Association (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 (for example, 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 screening/baseline

          -  Any intercurrent illness or condition that may preclude the participant from
             fulfilling the study requirements or suggests to the investigator that the participant
             may have an unacceptable risk by participating in this study