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Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gaucher Disease Type 3

Active, not recruiting · Phase 3 · NCT05222906

Conditions: Gaucher's Disease Type III

At a glance

Research payments reported, 2025
$595,612
U.S. investigators named
4
Sites paid
4
Planned or actual enrollment
43

About the trial

Sponsor
Sanofi
Tested
Venglustat (drug), imiglucerase (drug)
Dates
Started 2022-04-18; completion 2026-10-30
Results posted
Not yet

This is a parallel arm, Phase 3, double-blind, double-dummy, active-comparator, 2 arm study to evaluate the efficacy and safety of daily oral venglustat versus intravenous Cerezyme infusions every two weeks for improvement or stabilization of the neurological manifestations and maintenance of systemic disease stability in participants aged ≥12 and \<18 years and adult patients with Gaucher disease Type 3 (GD3) who have been treated with Enzyme Replacement Therapy (ERT) for at least 3 years.

From ClinicalTrials.gov, last updated 2026-07-13. Full record, eligibility and contacts (opens in a new tab)

Industry funding

Sanofi US Services Inc. reported $595,612 in research payments for this study in 2025 (35 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

Sources: CMS Open Payments research payments (program year 2025) and ClinicalTrials.gov, U.S. National Library of Medicine.