Status:
RECRUITING
A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal Disease
Lead Sponsor:
Amicus Therapeutics
Conditions:
Fabry Disease
Eligibility:
All Genders
18+ years
Phase:
PHASE3
Brief Summary
An Open-label Study to Evaluate the Safety and Pharmacokinetics of Migalastat HCl in Subjects with Fabry Disease and Amenable GLA Variants and Severe Renal Impairment (SRI) or End Stage Renal Disease ...
Detailed Description
This is an open-label, non-comparative study for subjects with Fabry disease who have an estimated glomerular filtration rate (eGFR) based on the Modification of Diet in Renal Disease equation (eGFRMD...
Eligibility Criteria
Inclusion
- 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
Exclusion
- Subject has undergone kidney transplantation
- Subject is on peritoneal dialysis
- 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 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)
- 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
- 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
Key Trial Info
Start Date :
October 31 2022
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
December 31 2026
Estimated Enrollment :
14 Patients enrolled
Trial Details
Trial ID
NCT04020055
Start Date
October 31 2022
End Date
December 31 2026
Last Update
October 22 2025
Active Locations (15)
Enter a location and click search to find clinical trials sorted by distance.
1
Emory University
Atlanta, Georgia, United States, 30322
2
The Cleveland Clinic
Cleveland, Ohio, United States, 44195
3
Renal Disease Research Institute
Dallas, Texas, United States, 75235
4
Lysosomal and Rare Disorders Research and Treatment Center, Inc
Fairfax, Virginia, United States, 22030