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

Launched by AMICUS THERAPEUTICS · Jul 12, 2019

Keywords

ClinConnect Summary

This clinical trial is studying a medication called Migalastat, which is being tested for its safety and effectiveness in people with Fabry disease who have specific genetic variants and kidney problems. Fabry disease is a genetic disorder that can affect various organs, including the kidneys. The trial aims to see how well Migalastat works in individuals aged 18 and older who have severe kidney impairment or are on dialysis. Participants need to have a certain type of genetic variant that allows them to take Migalastat and must agree to certain medical monitoring during the study.

To be eligible for this trial, participants must have a documented history of Fabry disease and have significant kidney issues, specifically an eGFR (a measure of kidney function) of less than 30. They should also be able to provide consent for their participation. If someone joins the trial, they can expect to receive Migalastat and will undergo regular check-ups to monitor their health and how their body is responding to the treatment. It's important for potential participants to note that certain conditions, like having had a kidney transplant or being on peritoneal dialysis, would exclude them from the study.

Gender

ALL

Eligibility criteria

• Inclusion 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
• Exclusion Criteria:
• • 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