A study to determine the long-term safety and tolerability of oral lucerastat in adult subjects with Fabry disease. This study includes a sub-study evaluating kidney Gb3 inclusions (and other histologic lesions) in male participants with classic Fabry disease who have been treated for at least 2 years with lucerastat monotherapy in study ID-069A302.
Study ID-069A302 will continue at each site until lucerastat is commercially available in the respective country, or until all subjects have (prematurely) discontinued the trial, or until the sponsor terminates the study, whichever is earliest. Note that, in Europe (local protocol amendment), the maximum individual study participation is up to Month 96.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
107
Administered in hard gelatin capsules containing 250 mg of lucerastat.
Treatment-emergent adverse events (AEs)
Time frame: From enrollment to Follow-up 1 (FU1) visit; duration: for up to 10 years including 1 month Follow-up
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
University of Alabama at Birmingham - Nephrology Research Clinic
Birmingham, Alabama, United States
University of California Irvine
Irvine, California, United States
UCSF Benioff Children's Hospital Oakland
Oakland, California, United States
University of Florida Clinical and Translational Science Institute, UF Clinical Research Center
Gainesville, Florida, United States
Rush University Medical Center - Dept of Pediatrics
Chicago, Illinois, United States
University of Iowa Stead Family Children's Hospital - Division of Medical Genetics
Iowa City, Iowa, United States
Massachusetts General Hospital
Boston, Massachusetts, United States
Infusion Associates
Grand Rapids, Michigan, United States
University of Pennsylvania - Dept of Medicine
Philadelphia, Pennsylvania, United States
Greenwood Genetics Center
Greenville, South Carolina, United States
...and 32 more locations