Venglustat in Combination With Cerezyme in Adult and Pediatric Patients With Gaucher Disease Type 3

  • STATUS
    Recruiting
  • End date
    Jan 13, 2023
  • participants needed
    49
  • sponsor
    Genzyme, a Sanofi Company
Updated on 1 September 2021

Summary

Primary Objectives:

Part 1:

Cohorts 1, 2 and 3

  • Evaluate cerebrospinal fluid (CSF) biomarkers in adult Gaucher disease (GD) Type 3 patients that distinguish GD3 from adult Gaucher disease Type 1 (GD1) patients.
  • Screen adult and pediatric GD3 patients who qualify for treatment with venglustat in Parts 2 and 3.

Parts 2 and 3:

Cohort 2

  • Evaluate short-term (Part 2) and long-term (Part 3) safety and tolerability of venglustat in combination with Cerezyme in adult GD3 patients.
  • Evaluate the change in CSF central nervous system (CNS) biomarkers (glucosylceramide [GL-1] and lyso-glucosylceramide [lyso-GL1]) from adult GD3 patients receiving venglustat in combination with Cerezyme (Part 2 only).

Cohort 3

  • Evaluate the efficacy of venglustat in combination with Cerezyme in adult and pediatric GD3 patients by assessing:
  • Ataxia using the Scale for the Assessment and Rating of Ataxia (SARA)
  • Cognition using the Repeatable Battery for the Assessment of Neuropsychological Status (RBANS)

Secondary Objectives:

Parts 2 and 3:

Cohort 2

  • Evaluate the pharmacokinetics (PK) of venglustat in adult GD3 patients.

Cohort 3

  • Evaluate the efficacy of venglustat in combination with Cerezyme in adult and pediatric GD3 patients by assessing:
  • CSF Lyso-GL1 levels
  • Modified Friedreichs Ataxia Rating Scale - Activities of Daily Living (FARS-ADL)
  • Brain resting-state functional Magnetic Resonance Imaging (rs-fMRI) reflecting connectivity between parieto-occipital areas
  • Bone disease manifestations
  • Evaluate safety and tolerability of venglustat in combination with Cerezyme in adult and pediatric GD3 patients
  • Evaluate PK of venglustat in adult and pediatric GD3 patients.

Description

The total duration for GD1 participants is 45 days (Part 1), while for GD3 participants the total duration is up to 4.2 years for Cohort 2 (up to 5.2 years for US participants) and up to 3.7 years for Cohort 3.

Details
Condition Gaucher Disease Type 1-Gaucher Disease Type 3, Gaucher Disease Type 1-Gaucher Disease Type 3, Gaucher Disease Type 1-Gaucher Disease Type 3, Gaucher Disease Type 1-Gaucher Disease Type 3, Gaucher Disease Type 1-Gaucher Disease Type 3, Gaucher Disease Type 1-Gaucher Disease Type 3, Gaucher Disease Type 1-Gaucher Disease Type 3, Gaucher Disease Type 1-Gaucher Disease Type 3, Gaucher Disease Type 1-Gaucher Disease Type 3, Gaucher Disease Type 1-Gaucher Disease Type 3, Gaucher Disease Type 1-Gaucher Disease Type 3, Gaucher Disease Type 1-Gaucher Disease Type 3, Gaucher Disease Type 1-Gaucher Disease Type 3, Gaucher Disease Type 1-Gaucher Disease Type 3, Gaucher Disease Type 1-Gaucher Disease Type 3, Gaucher Disease Type 1-Gaucher Disease Type 3, Gaucher Disease Type 1-Gaucher Disease Type 3, Gaucher Disease Type 1-Gaucher Disease Type 3, Gaucher Disease Type 1-Gaucher Disease Type 3, Gaucher Disease Type 1-Gaucher Disease Type 3, Gaucher Disease Type 1-Gaucher Disease Type 3, Gaucher Disease Type 1-Gaucher Disease Type 3, Gaucher Disease Type 1-Gaucher Disease Type 3, Gaucher Disease Type 1-Gaucher Disease Type 3, Gaucher Disease Type 1-Gaucher Disease Type 3, Gaucher Disease Type 1-Gaucher Disease Type 3, Gaucher Disease Type 1-Gaucher Disease Type 3, Gaucher Disease Type 1-Gaucher Disease Type 3, Gaucher Disease Type 1-Gaucher Disease Type 3, Gaucher Disease Type 1-Gaucher Disease Type 3, Gaucher Disease Type 1-Gaucher Disease Type 3, Gaucher Disease Type 1-Gaucher Disease Type 3, Gaucher Disease Type 1-Gaucher Disease Type 3
Treatment Placebo, GZ402671, venglustat (GZ/SAR402671), imiglucerase, venglustat (GZ402671)
Clinical Study IdentifierNCT02843035
SponsorGenzyme, a Sanofi Company
Last Modified on1 September 2021

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