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.
- 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
- Uncontrolled hypertension
- Currently taking Tolvaptan
- Patient is immunocompromised
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.