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
Condition(s) studied
Investigational drug(s) / intervention(s)
Venglustat (GZ402671): Pharmaceutical form: Tablet Route of administration: Oral
Placebo: Pharmaceutical form: Tablet Route of administration: Oral
Study summary
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).
Eligibility
Primary outcome measure(s)
- Percent change from baseline at 6 months in the most bothersome symptom of 3 Fabry Disease Patient-Reported Outcome (FD-PRO) items (neuropathic pain in upper extremities, neuropathic pain in lower extremities, or abdominal pain) — From baseline to 6 months
- Percent change from baseline at 12 months in the most bothersome symptom of 3 Fabry Disease Patient-Reported Outcome (FD-PRO) items (neuropathic pain in upper extremities, neuropathic pain in lower extremities, or abdominal pain) — From baseline to 12 months
Trial sites (50)
| Facility | City | Region | Status |
|---|---|---|---|
| University of Alabama -The Kirklin Clinic- Site Number : 8400011 | Birmingham | Alabama | |
| David Geffen School of Medicine at UCLA- Site Number : 8400006 | Los Angeles | California | |
| University of California Irvine Medical Center- Site Number : 8400019 | Orange | California | |
| AdventHealth Orlando- Site Number : 8400008 | Orlando | Florida | |
| Emory University School of Medicine - Atlanta- Site Number : 8400010 | Atlanta | Georgia | |
| WMCHealth Advanced Physician Services- Site Number : 8400001 | Hawthorne | New York | |
| Cincinnati Children's Hospital Medical Center- Site Number : 8400013 | Cincinnati | Ohio | |
| UPMC Montefiore- Site Number : 8400009 | Pittsburgh | Pennsylvania | |
| Renal Disease Research Institute - Dallas - Live Oak Street- Site Number : 8400012 | Dallas | Texas | |
| University of Utah Health Hospital- Site Number : 8400005 | Salt Lake City | Utah | |
| Lysosomal and Rare Disorders Research and Treatment Center (LDRTC)- Site Number : 8400004 | Fairfax | Virginia | |
| Investigational Site Number : 0320001 | Pergamino | Buenos Aires | |
| Investigational Site Number : 0320003 | Quilmes | Buenos Aires | |
| Investigational Site Number : 0320002 | La Rioja | Argentina | |
| Investigational Site Number : 0360001 | Parkville | Victoria | |
| Investigational Site Number : 0400001 | Vienna | Austria | |
| Hospital de Clinicas de Porto Alegre- Site Number : 0760001 | Porto Alegre | Rio Grande do Sul | |
| Instituto de Genética e Erros Inatos do Metabolismo- Site Number : 0760002 | São Paulo | Brazil | |
| Investigational Site Number : 1240003 | Calgary | Alberta | |
| Investigational Site Number : 1240001 | Halifax | Nova Scotia | |
| Investigational Site Number : 1240005 | Toronto | Ontario | |
| Investigational Site Number : 1560001 | Beijing | China | |
| Investigational Site Number : 1560003 | Shanghai | China | |
| Investigational 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 | |
| Investigational Site Number : 2760003 | Munich | Germany | |
| Investigational Site Number : 2760001 | Würzburg | Germany | |
| Investigational Site Number : 3000003 | Athens | Greece | |
| Investigational Site Number : 3000002 | Ioannina | Greece | |
| Investigational Site Number : 3800001 | Naples | Napoli | |
| Investigational Site Number : 3800004 | Rome | Roma | |
| Investigational Site Number : 3800005 | Bologna | Italy | |
| Investigational Site Number : 3920004 | Fukuoka | Japan | |
| Investigational Site Number : 3920003 | Tokyo | Japan | |
| Investigational Site Number : 4840001 | Monterrey | Nuevo León | |
| Investigational Site Number : 4840002 | Mexico City | Mexico | |
| Investigational Site Number : 5780001 | Bergen | Norway |
+ 10 more sites — see the full list on the official registry below.
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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 USA