Research Project

Project Title:	A Medical Research Study Designed to Determine if Venglustat Can be a Future Treatment for ADPKD Patients
Project Type:	Clinical Trial, Enrolment ongoing
Disease group(s):	AD structural kidney disorders
Project 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).
Lead principal investigator(s):	Bertrand Knebelmann, Paris
Co-investigator(s):	Myriam Dao , Paris Aurelie Hummel, Paris
Project Period:	02/2019 - 10/2023
Sponsors:	Industry
ClinicalTrials.gov:	NCT03523728