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A Medical Research Study Desi...

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

Last Updated: 2023-02-03

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE2/PHASE3

Total Enrollment

478 participants

Study Classification

INTERVENTIONAL

Study Start Date

2018-10-04

Study Completion Date

2021-08-03

Brief Summary

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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).

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Detailed Description

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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

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Polycystic Kidney, Autosomal Dominant

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Investigators Outcome Assessors

Study Groups

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Stage 1- Placebo

Participants from Stage 1 were randomized to receive 2 capsules of placebo matched to venglustat once daily for treatment period of 24 months.

Group Type PLACEBO_COMPARATOR