Venglustat in Combination With Cerezyme in Adult Patients With Gaucher Disease Type 3 With Venglustat Monotherapy Extension
Active, not recruiting · Phase 2 · NCT02843035
Conditions: Gaucher Disease Type 1, Gaucher Disease Type 3
At a glance
- Research payments reported, 2025
- $98,258
- U.S. investigators named
- 1
- Sites paid
- 1
- Planned or actual enrollment
- 12
About the trial
- Sponsor
- Genzyme, a Sanofi Company
- Tested
- venglustat (GZ402671) (drug), imiglucerase (drug)
- Dates
- Started 2017-01-04; completion 2026-10-30
- Results posted
- Not yet
Part 1: Biomarker evaluation/screening phase Primary Objectives: * Evaluate cerebrospinal fluid (CSF) biomarkers in adult Gaucher disease Type 3 (GD3) participants that distinguish GD3 from adult Gaucher disease Type 1 (GD1) participants * Screen adult GD3 participants who qualify for treatment with venglustat in Parts 2, Part 3, and Part 4 Parts 2 and 3: Combination treatment phases Primary objectives: * Evaluate short-term (Part 2) and long-term (Part 3) safety and tolerability of venglustat in combination with Cerezyme in adult GD3 participants * Evaluate the change in CSF central nervous system (CNS) biomarkers (glucosylceramide \[GL-1\] and lyso-glucosylceramide \[lyso-GL-1\]) from a…
From ClinicalTrials.gov, last updated 2026-07-10. Full record, eligibility and contacts (opens in a new tab)
Industry funding
Genzyme Corporation reported $98,258 in research payments for this study in 2025 (5 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
Clinicians the company named as principal investigators in its Open Payments reports.
Sites paid to run it
- Lysosomal and Rare Disorders Research and Treatment Center, Inc $98,258
Sources: CMS Open Payments research payments (program year 2025) and ClinicalTrials.gov, U.S. National Library of Medicine.