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STAGED-PKD

Title 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)
Therapeutic Area Polycystic Kidney Disease, PKD
Principal Investigator Ronald D. Perrone
Min Age 18 Years
Max Age 50 Years
Gender All
Contact Phoenix Turoci / Nicole Merullo
617-636-7914 / 617-636-8117
pturoci@tuftsmedicalcenter.org / nmerullo@tuftsmedicalcenter.org
More Information https://clinicaltrials.gov/ct2/show/NCT03523728?term=NCT03523728

Overview

This is an international, multicenter, randomized, double-blind, placebo-controlled two stage study in adult patients at risk of rapidly progressive Autosomal Dominant Polycystic Kidney Disease (ADPKD) aged 18 to 50 years.

 

The study is divided into two stages. The main purpose of Stage 1 is to determine the effect of GZ/SAR402671 on the rate of total kidney volume (TKV) growth in patients with ADPKD and determine the dose that will be used in Stage 2. The main purpose of Stage 2 is to evaluate the impact of GZ/SAR402671 on the rate of renal function decline. Both Stage 1 and Stage 2 will also evaluate safety and tolerability of GZ/SAR402671 in patients with ADPKD.

Study Details

Inclusion Criteria

  • Diagnoses of ADPKD,
  • Mayo Imaging Class of ADPKD Class 1C, 1D, or 1E,
  • ed glomerular filtration rate between 45 to 90 mL/min/1.73 m2

Exclusion Criteria

  • Uncontrolled hypertension
  • Currently taking Tolvaptan
  • Patient is immunocompromised

Study Requirements

Length of participation is 26 months. Subjects will be seen every 3 months at Tufts Medical Center with MRIs every 9 months and eye exams taking place every 6 months. A blood draw and urine collection will be done at every visit to the study site. 24 hour urine collection will happen at home and be brought to the study site every 6 months.