A Study to Evaluate the Effect of Venglustat Tablets on Neuropathic and Abdominal Pain in Male and Female Participants ≥16 Years of Age With Fabry Disease
Active, not recruiting · Phase 3 · NCT05206773
Conditions: Fabry Disease
At a glance
- Research payments reported, 2025
- $460,956
- U.S. investigators named
- 15
- Sites paid
- 15
- Planned or actual enrollment
- 122
About the trial
- Sponsor
- Sanofi
- Tested
- Venglustat (GZ402671) (drug), Placebo (drug)
- Dates
- Started 2022-03-11; completion 2027-01-04
- Results posted
- Not yet
This is a 12-month, parallel treatment, Phase 3, double-blind, randomized, placebo-controlled study to evaluate the effect of venglustat on neuropathic and abdominal pain symptoms of Fabry disease in participants ≥16 years of age with Fabry disease who are treatment-naïve or untreated for at least 6 months. * Study visits will take place approximately every 3 months. * The double-blind period will be followed by an open-label extension (OLE) during which participants who have completed the double-blind period will be treated with venglustat for an additional 12 months or until the Common Study End of Treatment Day (CSEOTD).
From ClinicalTrials.gov, last updated 2026-10-13. Full record, eligibility and contacts (opens in a new tab)
Industry funding
Genzyme Corporation reported $460,956 in research payments for this study in 2025 (71 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
- Tooraj J. Raoof, MD Internal Medicine · Encino, CA
- Changrui Xiao, M.D. Medical Genetics · Orange, CA
- Kimberly M. Neyman, M.D. Dermatology · Wheat Ridge, CO
- William Wilcox Medical Genetics · Decatur, GA
- Majed J. Dasouki, MD Medical Genetics · Kansas City, MO
- Maryam Banikazemi, MD Ph.D. Medical Genetics (non-physician) · Valhalla, NY
- Robert J. Hopkin, M.D. Medical Genetics · Cincinnati, OH
- Xiangling Wang, MD, PHD Medical Genetics · Cleveland, OH
- Gerard Vockley, MD, PHD Specialist · Pittsburgh, PA
- Ankit N. Mehta, M.D. Nephrology (Kidney) · Dallas, TX
- Ozlem Goker-alpan, M.D. Pediatrics · Fairfax, VA
- Brian L. Johnson, MD Mohs Surgery · Norfolk, VA
- Krystal J. Massey, M.D. Dermatology · Cheyenne, WY
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 $99,216
- Renal Disease Reasearch Institute L $67,338
- University of Pittsburgh $53,419
- New York Medical College Office of Research Administration Skyline Rm J23 $42,660
- University of Alabama Hospital, AL $37,267
- Emory University Hospital, GA $31,989
- The Clinical Trials Network Llc $20,439
- Cincinnati Children's Hospital Medical Center, OH $19,701
- Cleveland Clinic, OH $18,280
- Uci Health-Orange, CA $16,687
- T Joseph Raoof MD Inc $14,443
- University of Utah Hospital and Clinics, UT $14,229
- Adventhealth Orlando, FL $10,300
- Vaishali PAtel Summit Clinical Research Llc $9,423
- Paradigm Clinical Research Centers, Inc $5,566
Sources: CMS Open Payments research payments (program year 2025) and ClinicalTrials.gov, U.S. National Library of Medicine.