A Medical Research Study Designed to Determine if Venglustat Can be a Future Treatment for ADPKD Patients (STAGED-PKD)

Conditions

Diseases of the Kidney & Urinary Tract

Phase III

What is the purpose of this trial?

Brief Summary:

Primary Objective:

To determine the effect of venglustat on the rate of total kidney volume (TKV) growth and estimated glomerular filtration rate (eGFR) decline in patients at risk of rapidly progressive Autosomal Dominant Polycystic Kidney Disease (ADPKD).

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).
  • 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).
  • To evaluate change in nocturia based on patient reported diary (Stages 1 and 2).
  • To evaluate the effect of venglustat on kidney concentrating ability by assessing urine osmolality (in patients not on diuretic) (Stage 2 only).
  • Trial with
    Genzyme, A Sanofi Company
  • Start Date
    04/17/2019
  • End Date
    11/06/2022
Trial Image

For more information about this study, contact:

Lauren Liberti

I'm interested in volunteering

If you would prefer to contact a member of the Help us Discover team about this trial and other similar trials, please email helpusdiscover@yale.edu or call 877.978.8348

  • Last Updated
    09/05/2019
  • Study HIC
    #2000023931