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

NCT05222906 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 43

Last updated 2025-10-17

No results posted yet for this study

Summary

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.

Conditions

  • Gaucher's Disease Type III

Interventions

DRUG

Venglustat

tablet; oral

DRUG

imiglucerase

sterile lyophilized product; intravenous

Sponsors & Collaborators

Principal Investigators

  • Clinical Sciences & Operations · Sanofi

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
12 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-04-18
Primary Completion
2025-10-02
Completion
2026-10-30
FDA Drug
Yes

Countries

  • United States
  • Argentina
  • Canada
  • China
  • France
  • Germany
  • Hungary
  • Italy
  • Japan
  • Turkey (Türkiye)
  • United Kingdom

Study Locations

More Related Trials

Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05222906 on ClinicalTrials.gov