A Medical Research Study Designed to Determine if Venglustat Can be a Future Treatment for ADPKD Patients
Status:
Terminated
Trial end date:
2021-08-03
Target enrollment:
Participant gender:
Summary
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).