NCT03523728

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

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
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 478 (actual)
• Sponsor: Genzyme, a Sanofi Company · Industry
• Sex: All
• Age: 18 Years – 55 Years
• Healthy volunteers: Not accepted

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).

Detailed 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).

Conditions

• Polycystic Kidney, Autosomal Dominant

Interventions

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

Timeline

• Start date: 2018-10-04
• Primary completion: 2021-08-03
• Completion: 2021-08-03
• First posted: 2018-05-14
• Last updated: 2023-02-03
• Results posted: 2022-11-09

Locations

95 sites across 23 countries: United States, Argentina, Australia, Austria, Belgium, Canada, China, Czechia, Denmark, France, Germany, Israel, Italy, Japan, Netherlands, Poland, Portugal, Romania, South Korea, Spain, Taiwan, Turkey (Türkiye), United Kingdom

Regulatory

• FDA-regulated drug study