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ACTION


Title A Phase 3 Study of the Efficacy and Safety of Lixivaptan in Participants with Autosomal Dominant Polycystic Kidney Disease Consisting of a 1-year Double-blind, Placebo-controlled, Randomized Phase and a 1-year Open-Label Phase: The ACTION Study
Therapeutic Area Polycystic Kidney Disease
Principal Investigator Ronald Perrone, MD
Min Age 18 Years
Max Age 60 Years
Gender Any
Contact Phoenix Turoci and Nicole Merullo
617-636-7914 or 617-636-8117
pturoci@tuftsmedicalcenter.org or nmerullo@tuftsmedicalcenter.org
More Information https://clinicaltrials.gov/ct2/show/NCT04064346

Overview

This is a Phase 3 trial consisting of a 2-arm, double-blind, placebo-controlled, randomized phase (Part 1) followed by a single-arm open-label phase (Part 2) to demonstrate the efficacy and safety of lixivaptan in participants with ADPKD. Part 1 of the trial is designed to demonstrate the efficacy of lixivaptan in slowing the decline in kidney function as measured by the difference in change from baseline of eGFR between the lixivaptan-treated and placebo-treated participants. Part 2 of the study is designed to provide confirmation of the durability of this effect. 

Study Details

Inclusion Criteria

  • Diagnoses of ADPKD
  • Mayo Imaging Class of ADPKD Class 1C, 1D, or 1E
  • Estimated glomerular filtration rate between 25 to 90 mL/min/1.73 m2

Exclusion Criteria

  • Uncontrolled diabetes
  • Prior use of Tolvaptan 2 months prior to screening visit
  • Requires ongoing diuretic use

Study Requirements

Length of participation is 2.5 years and up to 45 study visits (not all participants will need all visits). Blood and urine samples will be collected, in addition to vitals, an ECG, and an MRI scan at 3 time-points during the study.