Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
For general information about clinical research, read Learn About Studies.
Eligibility Criteria
- 1. Male or female subjects aged 18 years or older, diagnosed with Fabry disease.
- 2. 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
- 3. Subject has a GLA variant that is amenable to migalastat recorded in their medical records
- 4. 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
- 5. 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
- 6. Subjects with ESRD must commit to completing at least 4 standard HD or HDF sessions during each 2-week dosing interval.
- 7. Subjects with ESRD must commit to completing the entire prescribed duration for each dialysis session.
- 8. If of reproductive potential, both male and female patients agree to use a medically accepted method of contraception
- 1. Subject has undergone kidney transplantation
- 2. Subject is on peritoneal dialysis
- 3. Subject is treated or has been treated with another investigational drug (except migalastat) within the 30 days
- 4. Subject has undergone any gene therapy at any time prior to the study or anticipates undergoing gene therapy during the study.
- 5. Subject has had a documented transient ischemic attack, stroke, unstable angina, or myocardial infarction
- 6. Subject has clinically significant unstable cardiac disease
- 7. Subject has any intercurrent illness or condition that may preclude the subject from fulfilling the protocol requirements
- 8. Subject has a history of allergy or sensitivity to migalastat (including excipients) or other iminosugars (eg, miglustat, miglitol)
- 9. Subject requires concurrent treatment with Glyset® (miglitol), Replagal® (agalsidase alfa), or Fabrazyme® (agalsidase beta)
- 10. Subject requires concurrent treatment with Zavesca® (miglustat) or has been treated with Zavesca
- 11. Female subject is pregnant or breast-feeding
- 12. Subject is unable to comply with study requirements
- 13. In France only, protected persons as defined by the Public Health Code
Ages Eligible for Study
18 Years to
Sexes Eligible for Study
ALL
Accepts Healthy Volunteers
No