Pinnacle Kidney Disease Study

About the Condition

Autosomal Dominant Polycystic Kidney Disease (ADPKD) is an inherited condition that causes fluid-filled cysts to grow in the kidneys over time. Most people do not develop symptoms until they are 30 to 40 years old. These cysts gradually enlarge the kidneys and can make them work less effectively. Over many years, this can lead to high blood pressure, pain or discomfort in the abdomen or back, and eventually reduced kidney function or kidney failure in some people.

ADPKD is one of the most common inherited kidney diseases, affecting about 1 in every 1,000 people worldwide. It’s most often caused by a change (mutation) in one of two genes: PKD1 or PKD2. Although symptoms usually start in adulthood, often between the ages of 30 and 40, cysts can start forming much earlier. Hypertension affects 70% to 80% of patients with ADPKD, and approximately 9% to 14% develop intracranial aneurysms, which have a rupture rate of 0.57 per 1000 patient-years. Approximately 50% of individuals with ADPKD require kidney replacement therapy by 62 years of age.

While there’s no cure yet, research studies like this one are exploring new treatments that could slow the disease’s progression and help protect kidney function.

Study Aim

Other procedures may include:

• Vital signs, blood draws and urine tests
• Genetic testing
• Height & weight measurements
• ECG’s
• MRI or CT scans
• Safety labs
• Questionnaires  
  
  

The study medication is administered as an intravenous (IV) infusion, meaning it is slowly delivered into a vein via an IV line. All infusions are performed in the clinical research unit (CRU) under direct medical supervision. Participants are usually observed for several hours (or overnight) after dosing for safety monitoring. Depending on which cohort you are a part of, you will be dosed once or up to three times throughout the study.