Ignite Creation Date:
2025-12-24 @ 11:45 PM
Ignite Modification Date:
2026-02-23 @ 1:28 AM
Study NCT ID:
NCT04705051
Status:
TERMINATED
Last Update Posted:
2025-09-17
First Post:
2021-01-08
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Long-term Treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD) With Venglustat
Sponsor:
Sanofi
Organization:
Study Overview
Official Title:
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
Status Verified Date:
2025-09
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
LTS15823 (long-term extension of the EFC15392 study) was stopped after protocol specified interim analysis for futility of the parent EFC15392 study met the prespecified stopping rule based on the primary outcome measure.
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
STAGED-PKD-EXT
Brief 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).
-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).
Study Oversight
Has Oversight DMC:
True
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?: