NCT04705051

Long-term Treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD) With Venglustat

Multicenter, Open-label, Extension Study to Characterize the Long-term Efficacy and Safety of Early Versus Delayed Treatment With Venglustat (GZ/SAR402671) in Patients at Risk of Rapidly Progressive Autosomal Dominant Polycystic Kidney Disease (ADPKD)

• Status: Terminated
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 24 (actual)
• Sponsor: Sanofi · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

Primary Objective: -To determine the effect of early versus delayed treatment with venglustat on the total kidney volume (TKV) in participants at risk of rapidly progressive autosomal dominant polycystic kidney disease (ADPKD). Secondary Objective: * To determine the effect of early versus delayed treatment with venglustat on the renal function (estimated glomerular filtration rate \[eGFR\] \[Chronic Kidney Disease Epidemiology Collaboration {CKD-EPI} equation\]). * To characterize the safety profile of venglustat. * To evaluate the effect of venglustat on the lens by ophthalmological examination. * To evaluate the effect of venglustat on mood using Beck Depression Inventory-II (BDI-II).

Detailed description

The planned study duration per participant was 25.5 months (maximal). Screening period (when applicable): up to 2 weeks. Core treatment period: 24 months. Follow-up: 30 days after final dose of the investigational medicinal product (IMP) (venglustat).

Conditions

• Congenital Cystic Kidney Disease

Interventions

Type	Name	Description
DRUG	Venglustat GZ402671	Pharmaceutical form: capsule Route of administration: oral

Timeline

• Start date: 2021-02-09
• Primary completion: 2021-07-13
• Completion: 2021-07-13
• First posted: 2021-01-12
• Last updated: 2025-09-17
• Results posted: 2022-10-06

Locations

11 sites across 8 countries: United States, Australia, Belgium, Germany, Japan, Netherlands, South Korea, Spain

Regulatory

• FDA-regulated drug study