STAGED-PKD is a clinical study evaluating the effectiveness and safety of GZ/SAR402671 (or Venglustat – an oral investigational drug) in patients with Autosomal Dominant PKD. The study will be will be run in 2 parts and aims to enroll 640 people across approximately 80 international sites located in the Americas, Europe and Asia-Pacific. The company expects to complete the trial by 2023.

The study is divided into 2 separate stages. Each stage will consist of a 30-day screening period, a 2-week period where placebo is taken, a 24-month study treatment period, and a 4-week post-study treatment follow up period.

The total length of participation and treatment in the study will be the same in Stage 1 and Stage 2 (up to 26 months participation for each stage). Stage 1 of the study enrollment is currently ongoing. Participants are randomly assigned to 1 of 3 study groups and will take the oral study medication Venglustat (8mg or 15mg), or matching placebo, once daily over the duration of approximately 24 months.

After Stage 1 of the study finishes, Stage 2 will start. Patients in Stage 2 will receive either placebo or Venglustat at the highest dose determined in Part 1 to be safe and effective.

The investigational drug, study-related procedures and doctor visits will be provided at no cost. If you travel to the site for your study visits, travel expenses will be reimbursed and compensation for study-related time may be compensated.

 

You may be eligible for this study if you:

  • Are between 18 to 50 years of age
  • Have been diagnosed with ADPKD
  • Have a lab value eGFR between 45 to 90 mL/min/1.73 m2

 

Exclusion criteria:

  • Uncontrolled diabetes;
  • Kidney or any other solid organ transplant recipient or a planned transplant during the study;
  • Women who are pregnant or breastfeeding;
  • Currently taking tolvaptan

 

Find out if you might be eligible

 

Locations:

UNITED STATES —

Alabama
University of Alabama at Birmingham
Birmingham, Alabama

California
University of California Los Angeles
Los Angeles, CA

University of California San Francisco
San Francisco, CA

Colorado
University of Colorado
Aurora, CO

Connecticut
Yale University
New Haven, CT

Georgia
Emory University
Atlanta, GA

Illinois
University of Chicago
Chicago, IL

Iowa
University of Iowa
Iowa City, IA

Kansas
University of Kansas Medical Center
Kansas City, KS

Maryland
University of Maryland
Baltimore, MD

Massachusetts
Tufts Medical Center
Boston, MA

Minnesota
Mayo Clinic
Rochester, MN

Pennsylvania
University of Pennsylvania
Philadelphia, PA

Texas
Clinical Advancement Center
San Antonio, TX

West Virginia
West Virginia University
Morgantown, WV

Wisconsin
University of Wisconsin
Madison, WI

Medical College of Wisconsin
Milwaukee, WI

AUSTRALIA —

Westmead Hospital
Sydney

Royal Brisbane and Women’s Hospital
Herston

CANADA —

Ontario
Toronto General Hospital
Toronto, Ontario

Quebec
Montreal Clinical Research Institute
Montreal, Quebec

Alberta
University of Alberta
Edmonton, Alberta

UNITED KINGDOM —

University of Sheffield
Sheffield, UK