A Study to Evaluate the Effect of Venglustat Tablets on Left Ventricular Mass Index in Male and Female Adult Participants With Fabry Disease (NCT05280548)
🩺 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: To join this study, participants generally need to meet the listed inclusion criteria (5 items) and avoid the listed exclusions (17 items).
Location: University of Alabama -The Kirklin Clinic- Site Number : 8400010 — Birmingham, Alabama
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistCheck every box below. If you can check all of them, you may meet the study's basic screening requirements (final eligibility is confirmed by the site team).
Usually NOT eligible if any of the following apply:
- Exclusion Criteria:
- History of transient ischemic attack, stroke, myocardial infarction, heart failure, major cardiovascular surgery or kidney transplantation.
- History of seizures currently requiring treatment.
- Underlying medical condition that may cause or contribute to left ventricular hypertrophy.
- Asymmetric hypertrophy by cardiac MRI at screening if considered by central reader to be not related to Fabry disease.
- Advanced cardiac fibrosis, defined as significant late gadolinium enhancement affecting 3 or more segments involving \>50% of myocardial thickness on screening cardiac MRI.
- History of clinically significant cardiac arrhythmia. Atrial fibrillation that is well controlled on a stable medical regimen for at least 12 months is not an exclusion if the CHA2DS2-VASc score is 0 for males or 1 for females.
- Estimated glomerular filtration rate \<45 mL/min/1.73m2.
- Presence of severe depression as measured by Beck's Depression Inventory (BDI)-II \>28 and/or a history of an untreated, unstable major affective disorder within 1 year of the screening visit.
- Patients with hepatitis C, HIV, or hepatitis B infection.
- Positive SARS-CoV-2 virus test within 2 weeks of enrollment, or COVID-19 requiring hospitalization within 6 months of enrollment.
- History of drug and/or alcohol abuse.
🔍 Extracted verbatim from the official NIH eligibility criteria. Always confirm with the study coordinator.
📍 All Participating Trial Locations (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.