A Study to Evaluate the Effect of Venglustat Tablets on Left Ventricular Mass Index in Male and Female Adult Participants With Fabry Disease (NCT05280548)
A Randomized, Open-label, Parallel-group, 18-month Phase 3 Study to Evaluate the Effect of Venglustat Compared With Usual Standard of Care on Left Ventricular Mass Index in Participants With Fabry Disease and Left Ventricular Hypertrophy
🩺 Plain-English Summary (8th-Grade Level)
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.
Who can join: Review the eligibility criteria below.
Location: University of Alabama -The Kirklin Clinic- Site Number : 8400010 — Birmingham, Alabama
Age range: 18 Years to 65 Years
🧪 Interventions in This Study
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (54 Sites)
Showing the first 25 of 54 sites.
- University of Alabama -The Kirklin Clinic- Site Number : 8400010 — Birmingham, Alabama
- University of California Los Angeles Medical Center- Site Number : 8400008 — Los Angeles, California
- Emory University School of Medicine - Atlanta- Site Number : 8400009 — Atlanta, Georgia
- Ann & Robert H. Lurie Children's Hospital of Chicago- Site Number : 8400005 — Chicago, Illinois
- Maryam Banikazemi, MD- Site Number : 8400001 — Hawthorne, New York
- Renal Disease Research Institute- Site Number : 8400012 — Dallas, Texas
- University of Utah Health Hospital- Site Number : 8400006 — Salt Lake City, Utah
- Lysosomal and Rare Disorders Research and Treatment Center (LDRTC)- Site Number : 8400004 — Fairfax, Virginia
- Investigational Site Number : 0400001 — Graz,
- Investigational Site Number : 1240003 — Calgary, Alberta
- Investigational Site Number : 1240006 — Edmonton, Alberta
- Investigational Site Number : 1240002 — Vancouver, British Columbia
- Investigational Site Number : 1240005 — Toronto, Ontario
- Investigational Site Number : 1560002 — Beijing,
- Investigational Site Number : 1560005 — Beijing,
- Investigational Site Number : 1560001 — Chengdu,
- Investigational Site Number : 1560007 — Guangzhou,
- Investigational Site Number : 1560003 — Shanghai,
- Investigational Site Number : 2030001 — Prague,
- Investigational Site Number : 2080001 — Copenhagen,
- Investigational Site Number : 2500001 — Garches,
- Investigational Site Number : 2760003 — Berlin,
- Investigational Site Number : 2760004 — Hochheim am Main,
- Investigational Site Number : 2760005 — Mainz,
- Investigational Site Number : 2760001 — Würzburg,
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
More studies in Fabry Disease
- 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 — Birmingham, Alabama
- A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fabry Disease — Birmingham, Alabama
- Long-Term Follow-up of Subjects Who Were Treated With ST-920 — Irvine, California
- An Open-label, Phase 1/2 Trial of Gene Therapy 4D-310 in Adults With Fabry Disease — La Jolla, California
- A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal Disease — Atlanta, Georgia
Explore by condition: All Fabry Disease clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Review the official NIH record below to find the coordinating site.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.