A Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, and Safety of Venglustat in Late-onset GM2
Terminated · Phase 3 · NCT04221451
Conditions: Tay-Sachs Disease, Sandhoff Disease
At a glance
- Research payments reported, 2025
- $106,627
- U.S. investigators named
- 4
- Sites paid
- 4
- Planned or actual enrollment
- 75
About the trial
- Sponsor
- Genzyme, a Sanofi Company
- Tested
- venglustat GZ402671 (drug), placebo (drug)
- Dates
- Started 2020-06-29; completion 2024-12-26
- Results posted
- Yes
Primary Objectives: Primary population (adult participants with late-onset GM2 gangliosidosis): To assess the efficacy and pharmacodynamics (PD) of daily oral dosing of venglustat when administered over a 104-week period Secondary population (participants with juvenile/adolescent late-onset GM2 gangliosidosis, GM1 gangliosidosis, saposin C deficiency, sialidosis type 1 or juvenile/adult galactosialidosis): To assess PD response (plasma and CSF GL-1 biomarker and disease specific biomarkers) of venglustat when administered once daily over a 104-week period Secondary Objectives: Primary population: * To assess the PD of daily oral dosing of venglustat and the effect of venglustat on selecte…
From ClinicalTrials.gov, last updated 2026-01-28. Full record, eligibility and contacts (opens in a new tab)
Industry funding
Sanofi US Services Inc. reported $106,627 in research payments for this study in 2025 (9 payments).
Research payments usually go to hospitals, universities and research sites to run a study, not to the investigators personally. A payment is not evidence of wrongdoing.
U.S. investigators named on this study
- Susan L. Perlman, MD Neurology · Los Angeles, CA
- William Wilcox Medical Genetics · Decatur, GA
- Florian S. Eichler, MD Neurology · Boston, MA
- Heather A. Lau, M.D. Child Neurology · New York, NY
Clinicians the company named as principal investigators in its Open Payments reports.
Sites paid to run it
- Ronald Reagan UCLA Medical Center, CA $77,912
- Emory University Hospital, GA $17,690
- Massachusetts General Hospital, MA $11,000
- New York University NYU Grossman School of Medicine An Administrative Unit of New York University $25
Sources: CMS Open Payments research payments (program year 2025) and ClinicalTrials.gov, U.S. National Library of Medicine.