A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal Disease

Korábbi kezelés

• Subject has undergone kidney transplantation
• Subject is treated or has been treated with another investigational drug (except migalastat) within the 30 days
• Subject has undergone any gene therapy at any time prior to the study or anticipates undergoing gene therapy during the study.
• Subject requires concurrent treatment with Glyset® (miglitol), Replagal® (agalsidase alfa), or Fabrazyme® (agalsidase beta)
• Subject requires concurrent treatment with Zavesca® (miglustat) or has been treated with Zavesca

Más betegség

• Subject has had a documented transient ischemic attack, stroke, unstable angina, or myocardial infarction
• Subject has clinically significant unstable cardiac disease
• Subject has any intercurrent illness or condition that may preclude the subject from fulfilling the protocol requirements
• Subject has a history of allergy or sensitivity to migalastat (including excipients) or other iminosugars (eg, miglustat, miglitol)

Egyéb

• Subject is on peritoneal dialysis
• Female subject is pregnant or breast-feeding
• Subject is unable to comply with study requirements
• In France only, protected persons as defined by the Public Health Code

• Male or female subjects aged 18 years or older, diagnosed with Fabry disease.
• Subject (or legally authorized representative as applicable) is willing and able to provide written informed consent and authorization for use and disclosure of Personal Health Information
• Subject has a GLA variant that is amenable to migalastat recorded in their medical records
• Subject has at least 1 documented eGFR value of \< 30 mL/min/1.73 m2 within the last 3 months and has an eGFRMDRD value of \< 30 mL/min/1.73 m2 at Visit 1
• Subjects with ESRD have been on a stable 2- or 3-times a week HD (standard or HDF) regimen for at least 2 months prior to the screening visit
• Subjects with ESRD must commit to completing at least 4 standard HD or HDF sessions during each 2-week dosing interval.
• Subjects with ESRD must commit to completing the entire prescribed duration for each dialysis session.
• If of reproductive potential, both male and female patients agree to use a medically accepted method of contraception