This is an 18-month, multicenter, randomized, active-control, parallel-group Phase 3 study, in which participants will be randomized to venglustat versus standard of care therapy (agalsidase alfa, agalsidase beta, or migalastat) to evaluate the effect of venglustat on left ventricular mass index (LVMI) in adult participants with Fabry disease and left ventricular hypertrophy. * Study visits will take place approximately every 3 to 6 months * Participants who complete the randomized period may continue to the long-term extension (LTE) to receive venglustat for up to additional 45 months with the total study duration up to 5.3 years maximum.
Randomized period: the total duration will be up to approximately of 20 months (1 month screening 18 months of treatment and a possible follow-up period of 1 month if no participation in the long-term extension period) Long-term extension period: the total duration will be from minimum 19 months (18 months of treatment and 1 month of follow-up period) to maximum 46 months (45 months of treatment and 1 month of follow-up period). The maximum total study duration is approximately 5.3 years
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
104
Tablet; Oral
Concentrate for solution for infusion; IV infusion
Powder for concentrate for solution for infusion; IV infusion
Hard capsules; Oral
University of Alabama -The Kirklin Clinic- Site Number : 8400010
Birmingham, Alabama, United States
University of California Los Angeles Medical Center- Site Number : 8400008
Los Angeles, California, United States
Emory University School of Medicine - Atlanta- Site Number : 8400009
Atlanta, Georgia, United States
Ann & Robert H. Lurie Children's Hospital of Chicago- Site Number : 8400005
Chicago, Illinois, United States
Maryam Banikazemi, MD- Site Number : 8400001
Hawthorne, New York, United States
Slope of left ventricular mass index as measured by cardiac magnetic resonance imaging (MRI) (central reading)
Time frame: from baseline to 18 months
Slope of estimated glomerular filtration rate (eGFR) as assessed by the chronic kidney disease epidemiology collaboration (CKD-EPI) creatinine equation
Time frame: from baseline to 18 months
Change in T1 relaxation time, measured by cardiac MRI (central reading)
Time frame: from baseline to 18 months
Change in global longitudinal strain, measured by echocardiography (central reading)
Time frame: from baseline to 18 months
Percent Change in tiredness component of FD-PRO
Time frame: from baseline to 18 months
Percent Change in swelling in lower extremities component of FD-PRO
Time frame: from baseline to 18 months
Number of participants with adverse event (AE) and serious adverse event (SAE)
Time frame: from baseline to 18 months
Change in Beck Depression Inventory-II (BDI-II) score
Time frame: from baseline to 18 months
Change in the lens clarity by ophthalmological examination
Time frame: from baseline to 18 months
Plasma venglustat concentrations at prespecified visits over the study duration
Time frame: from baseline to 18 months
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Renal Disease Research Institute- Site Number : 8400012
Dallas, Texas, United States
University of Utah Health Hospital- Site Number : 8400006
Salt Lake City, Utah, United States
Lysosomal and Rare Disorders Research and Treatment Center (LDRTC)- Site Number : 8400004
Fairfax, Virginia, United States
Investigational Site Number : 0400001
Graz, Austria
Investigational Site Number : 1240003
Calgary, Alberta, Canada
...and 44 more locations