Regulus

We are testing an investigational drug called RGLS8429. “Investigational” means that the study drug being tested is not approved by the United States Food and Drug Administration for use in humans. The main purpose of this study is to get more information about the safety, tolerability (whether side effects can be handled by a subject), pharmacodynamics (how it works on the kidneys), and pharmacokinetics (how the drug is processed in the body) of RGLS8429 in patients with ADPKD. This is a randomized, double blind study, meaning that subjects will be assigned either to the investigational drug or to a placebo. RGLS8429 will be administered by subcutaneous injection (a shot just under the skin) and is being tested in different dose levels. 

Inclusion Criteria

- Mayo Imaging Classification 1C, 1D, or 1E size kidneys (based on MRI) - eGFR between 30 to 90 mL/min/1.73 m2 - Body mass index (BMI) 18 to 35 kg/m2

Exclusion Criteria

- Administration of Tolvaptan in the 28 days before randomization - Only one kidney or kidney transplant - History of cancer (except skin cancer)

Study Requirements

You will be asked to visit the study clinic up to 3 times during the Screening Period and then at least 10 times during the actual study. The research study is in 4 parts: • Screening Period – up to 28 days • Study Treatment (Dosing) Period – approximately 85 days • Follow-up Period – approximately 14 days • End of Study Visit – approximately 14 days after the end of the Follow-up Period During study visits we will perform procedures such as asking questions about your health and medications, physical exams, vital signs, electrocardiograms (ECG), Magnetic Resonance Imaging (MRI) scans (2 total), blood and urine samples. RGLS8429 will be administered 7 times during the Study Treatment period and will be given as a subcutaneous injection (a shot just under the skin into the fatter layer of your abdomen).