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

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).