A Study to Evaluate the Effect of Venglustat Tablets on Left Ventricular Mass Index in Male and Female Adult Participants With Fabry Disease
Active, not recruiting · Phase 3 · NCT05280548
Conditions: Fabry Disease
At a glance
- Research payments reported, 2025
- $616,033
- U.S. investigators named
- 7
- Sites paid
- 8
- Planned or actual enrollment
- 104
About the trial
- Sponsor
- Sanofi
- Tested
- Venglustat (GZ402671) (drug), Agalsidase alfa (drug), Agalsidase beta (GZ419828) (drug), Migalastat (drug)
- Dates
- Started 2022-05-03; completion 2027-12-06
- Results posted
- Not yet
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.
From ClinicalTrials.gov, last updated 2026-06-24. Full record, eligibility and contacts (opens in a new tab)
Industry funding
Genzyme Corporation reported $616,033 in research payments for this study in 2025 (64 payments).
Research payments usually go to hospitals, universities and research sites to run a study, not to the investigators personally. A payment is not evidence of wrongdoing.
U.S. investigators named on this study
- Eric Wallace Nephrology (Kidney) · Birmingham, AL
- Nicola Longo, MD Medical Genetics · Los Angeles, CA
- Anjay Rastogi, MD Nephrology (Kidney) · Los Angeles, CA
- William Wilcox Medical Genetics · Decatur, GA
- Carlos E. Prada, M.D. Medical Genetics · Chicago, IL
- Ankit N. Mehta, M.D. Nephrology (Kidney) · Dallas, TX
- Ozlem Goker-alpan, M.D. Pediatrics · Fairfax, VA
Clinicians the company named as principal investigators in its Open Payments reports.
Sites paid to run it
- Lysosomal and Rare Disorders Research and Treatment Center, Inc $284,529
- University of Alabama Hospital, AL $141,683
- Ann and Robert H Lurie Childrens Hospital of Chicago $55,483
- New York Medical College Office of Research Administration Skyline Rm J23 $39,191
- Ronald Reagan UCLA Medical Center, CA $36,006
- University of Utah $30,313
- Renal Disease Reasearch Institute L $24,508
- Emory University Hospital, GA $4,320
Sources: CMS Open Payments research payments (program year 2025) and ClinicalTrials.gov, U.S. National Library of Medicine.