A Medical Research Study Designed to Determine if Venglustat Can be a Future Treatment for ADPKD Patients

  • End date
    Nov 12, 2023
  • participants needed
  • sponsor
    Genzyme, a Sanofi Company
Updated on 22 August 2021
renal function
treatment regimen
glomerular filtration rate
chronic kidney disease
human chorionic gonadotropin
renal disease
ophthalmological examination
urine osmolality


Primary Objective:

To determine the effect of venglustat on the rate of total kidney volume (TKV) growth (Stage

  1. and estimated glomerular filtration rate (eGFR) decline in patients at risk of rapidly progressive Autosomal Dominant Polycystic Kidney Disease (ADPKD) (Stage 2).

Secondary Objectives:

  • To determine the effect of venglustat on the rate of renal function decline (Stage 1) and on the rate of TKV growth (Stage 2).
  • To evaluate the pharmacokinetics (PK) of venglustat in Autosomal Dominant Polycystic Kidney Disease patients (Stages 1 and 2).
  • To determine the effect of venglustat on pain and fatigue, based on patient reported diary (Stages 1 and 2).
  • Safety/tolerability objective:
  • To characterize the safety profile of venglustat (Stages 1 and 2).
  • To evaluate the effect of venglustat on mood using Beck Depression Inventory II (BDI-II) (Stages 1 and 2).
  • To evaluate the effect of venglustat on the lens by ophthalmological examination (Stages 1 and 2).


Study duration per participant is 26 months (maximal) per stage, including a screening period of 15 days, run-in period of 2 weeks, a 24-month treatment period, and a follow-up 30 days after final dose of investigational medicinal product (IMP).

Condition Polycystic Kidney, Autosomal Dominant
Treatment Placebo, Venglustat GZ/SAR402671, Venglustat GZ402671
Clinical Study IdentifierNCT03523728
SponsorGenzyme, a Sanofi Company
Last Modified on22 August 2021

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