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)
A Randomized, Double-blind, Placebo-controlled, 12-month Phase 3 Study to Evaluate the Effect of Venglustat on Neuropathic and Abdominal Pain in Male and Female Participants ≥16 Years of Age With Fabry Disease Who Are Treatment-naïve or Untreated for at Least 6 Months
🩺 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: Review the eligibility criteria below.
Location: Nephrology Clinic at Kirklin Clinic of UAB Hospital_Investigational Site Number: 8400011 — Birmingham, Alabama
Age range: 16 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 (58 Sites)
Showing the first 25 of 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.
More studies in Fabry Disease
- 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 the Effect of Venglustat Tablets on Left Ventricular Mass Index in Male and Female Adult Participants With Fabry Disease — Birmingham, Alabama
- 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.