Venglustat (GZ402671): Pharmaceutical form: Tablet Route of administration: Oral
Placebo: Pharmaceutical form: Tablet Route of administration: Oral
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).
| Facility | City | Region | Status |
|---|---|---|---|
| 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 | Argentina | |
| Instituto de Investigaciones Clínicas Quilmes (IICQ) SRL_Investigational Site Number: 0320003 | Quilmes | Argentina | |
| Investigational Site Number : 0360001 | Parkville | Victoria | |
| Investigational Site Number: 0400001 | Vienna | Austria | |
| 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 | Brazil | |
| 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 | China | |
| No.197,2nd Ruijin road, Huangpu district_Site Number: 1560003 | Shanghai | China | |
| No.85 South Jiefang road, Yingze District_Site Number: 1560004 | Taiyuan | China | |
| Investigational Site Number : 1560006 | Zhengzhou | China | |
| Investigational Site Number: 2080001 | Copenhagen | Denmark | |
| Investigational Site Number: 2460001 | Turku | Finland | |
| Investigational Site Number : 2500001 | Garches | France | |
| ISphinCS GmbH_Investigational Site Number: 2760004 | Hochheim am Main | Germany | |
| Investigational Site Number : 2760005 | Mainz | Germany | |
| Investigational Site Number: 2760003 | München | Germany | |
| Investigational Site Number: 2760001 | Würzburg | Germany | |
| Investigational Site Number : 3000003 | Athens | Greece | |
| University Hospital of Heraklion_Investigational Site Number: 3000001 | Heraklion | Greece | |
| University Hospital of Ioannina_Investigational Site Number: 3000002 | Ioannina | Greece | |
| IRCCS Policlinico di Sant'Orsola_Investigational Site Number : 3800005 | Bologna | Italy | |
| Fondazione IRCCS San Gerardo dei Tintori, S.C. Nefrologia - Clinica Nefrologica_Investigational Site Number: 3800002 | Monza | Italy | |
| Azienda Ospedaliera Universitaria "Federico II", U.O. di Nefrologia- Diparimento di Sanità Pubblica_Investigational Site Number: 3800001 | Naples | Italy | |
| Azienda Ospedaliera Universitaria_Investigational Site Number: 3800003 | Palermo | Italy | |
| Fondazione Policlinico Universitario_Investigational Site Number: 3800004 | Roma | Italy |
+ 18 more sites — see the full list on the official registry below.
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT05206773 on ClinicalTrials.gov ↗ ← All trials in the UK