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 (NCT05206773)
🩺 Plain-English Summary (8th-Grade Level)
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).
Who can join: To join this study, participants generally need to meet the listed inclusion criteria (6 items) and avoid the listed exclusions (18 items).
Location: Nephrology Clinic at Kirklin Clinic of UAB Hospital_Investigational Site Number: 8400011 — 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:
- Any manifestations of Fabry disease that preclude placebo administration.
- History of transient ischemic attack, stroke, myocardial infarction, heart failure, evidence of left ventricular hypertrophy and/or cardiac fibrosis, major cardiovascular surgery, or kidney transplantation.
- 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.
- Patients with hepatitis C, HIV, or hepatitis B infection.
- Neuropathic pain in upper or lower extremities, or abdominal pain not related to Fabry disease.
- History of seizures currently requiring treatment.
- Uncontrolled hypertension over the past 12 months prior to screening, or systolic BP \>=150 or diastolic BP \>=100 at screening.
- Estimated glomerular filtration rate \<60 mL/min/1.73m².
- Urine protein to creatinine ratio \>= 1 g/g at screening.
- 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.
- Positive SARS-CoV-2 virus test within 2 weeks of enrollment, or COVID 19 requiring hospitalization within 6 months of enrollment.
🔍 Extracted verbatim from the official NIH eligibility criteria. Always confirm with the study coordinator.
📍 All Participating Trial Locations (58 Sites)
- Nephrology Clinic at Kirklin Clinic of UAB Hospital_Investigational Site Number: 8400011 — Birmingham, Alabama
- UCLA Medical Center_Investigational Site Number: 8400006 — Los Angeles, California
- University of California Irvine Medical Center- Site Number : 8400019 — Orange, California
- Advent Health Orlando_Investigational Site Number: 8400008 — Orlando, Florida
- Emory Genetics- Site Number : 8400010 — Atlanta, Georgia
- Westchester Medical Center Healthcare Corporation- Site Number : 8400001 — Hawthorne, New York
- Cincinnati Children's Hospital Medical Center - PIN- Site Number : 8400013 — Cincinnati, Ohio
- Cleveland Clinic Site Number : 8400016 — Cleveland, Ohio
- Children's Hospital Of Pittsburgh- Site Number : 8400009 — Pittsburgh, Pennsylvania
- Renal Disease Research Institute, An affiliate of: Dallas Nephrology Associates_Investigational Site Number: 8400012 — Dallas, Texas
- University Of Utah Health Sciences Center- Site Number : 8400005 — Salt Lake City, Utah
- Lysosomal and Rare Disorders Research and Treatment Center_Investigational Site Number: 8400004 — Fairfax, Virginia
- Fundacion Cori para la Investigación y Prevención del Cancer_Investigational Site Number: 0320002 — La Rioja,
- Instituto de Investigaciones Clínicas Quilmes (IICQ) SRL_Investigational Site Number: 0320003 — Quilmes,
- Investigational Site Number : 0360001 — Parkville, Victoria
- Investigational Site Number: 0400001 — Vienna,
- Hospital de Clínicas de Porto Alegre_Investigational Site Number: 0760001 — Porto Alegre, Rio Grande do Sul
- Instituto de Genética e Erros Inatos do Metabolismo - IGEIM- Site Number : 0760002 — São Paulo,
- M.A.G.I.C Calgary LTD_Investigational Site Number: 1240003 — Calgary, Alberta
- Medicine Dalhousie University_Investigational Site Number : 1240001 — Halifax, Nova Scotia
- University Health Network_Investigational Site Number : 1240005 — Toronto, Ontario
- No.8, Xishiku Street, Xicheng District_Site Number: 1560001 — Beijing,
- No.197,2nd Ruijin road, Huangpu district_Site Number: 1560003 — Shanghai,
- No.85 South Jiefang road, Yingze District_Site Number: 1560004 — Taiyuan,
- Investigational Site Number : 1560006 — Zhengzhou,
💰 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.