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

Polycystic Kidney, Autosomal Dominant Clinical Trial

Official title: Multicenter, Randomized, Double-blind, Placebo-controlled Two Stage Study to Characterize the Efficacy, Safety, Tolerability and Pharmacokinetics of GZ/SAR402671 in Patients at Risk of Rapidly Progressive Autosomal Dominant Polycystic Kidney Disease (ADPKD)

Status Terminated

Clinical Trial 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 participants 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 ADPKD participants (Stages 1 and 2).

• To determine the effect of venglustat on pain and fatigue, based on participant reported diary (Stages 1 and 2).

• Safety/tolerability objectives:

• 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).

Clinical Trial Description

Study duration per participant was 26 months (maximal) that included 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). ;

Related Conditions & MeSH terms

• Polycystic Kidney Diseases
• Polycystic Kidney, Autosomal Dominant

• NCT number: NCT03523728
• Study type: Interventional
• Source: Sanofi
• Status: Terminated
• Phase: Phase 2/Phase 3
• Start date: October 4, 2018
• Completion date: August 3, 2021