Autosomal dominant polycystic kidney disease

This study tests an experimental drug called VX-407 in 24 adults with autosomal dominant polycystic kidney disease (ADPKD) who have specific changes in the PKD1 gene. The goal is to see if the drug can slow kidney growth and is safe. Participants take VX-407 tablets by mouth and …

This study tests an experimental drug called GSK4771261 for safety and how the body processes it. Part A involves healthy volunteers, and Part B includes people with autosomal dominant polycystic kidney disease (ADPKD), a genetic condition that causes kidney cysts and can lead to…

This study tests a drug called CAM2029 (octreotide) given as a weekly or every-other-week shot to people with symptomatic polycystic liver disease. The goal is to see if it can shrink the liver and reduce symptoms like belly pain, bloating, and nausea. About 71 adults with an enl…

This study tests a device that uses ultrasound to burn nerves around the kidney arteries, aiming to lower blood pressure in people with autosomal dominant polycystic kidney disease (ADPKD) whose blood pressure remains high despite medication. 44 participants will be randomly assi…

This study tests whether empagliflozin, a drug used for diabetes, is safe and tolerable for people with autosomal dominant polycystic kidney disease (ADPKD). About 50 adults with moderate kidney function will receive either the drug or a placebo for 12 months. Researchers will mo…

This phase 4 trial tests whether empagliflozin, a diabetes drug, can safely slow kidney growth and preserve kidney function in 44 adults with rapidly progressing autosomal dominant polycystic kidney disease (ADPKD). Participants take either empagliflozin or a placebo for 18 month…

This study tests a new DNA-reading technology called ultra-long read sequencing to find hidden genetic changes that standard tests miss. Researchers will analyze blood samples from 15 patients with known or suspected genetic variants in difficult-to-read DNA regions. The goal is …

This study looks at 401 people with autosomal dominant polycystic kidney disease (ADPKD) to see how different versions of the PKD1 gene relate to patient traits like age at diagnosis and body size. Researchers are not testing any treatment—they are simply gathering information to…

This study looks at whether losing weight over two years can improve how the kidneys use oxygen and how the body responds to insulin in people with autosomal dominant polycystic kidney disease (ADPKD) who are overweight. Researchers will use PET scans and insulin tests to measure…

This study looks at how certain blood chemicals and blood vessel function relate to kidney disease severity in people with early-stage Autosomal Dominant Polycystic Kidney Disease (ADPKD). Researchers will measure homocysteine levels and blood vessel health in 80 participants age…

This study collects kidney tissue from 100 adults with autosomal dominant polycystic kidney disease (ADPKD) who are already scheduled for kidney removal. Researchers will analyze the tissue for additional genetic mutations that may drive cyst formation. The goal is to better unde…

This study looks at whether using a robot to remove one kidney in people with autosomal dominant polycystic kidney disease (ADPKD) is safe and effective compared to traditional open surgery. Researchers will track 57 adult patients to see if the robotic approach leads to fewer co…

This study is testing a new type of ultrasound called super-resolution ultrasound (SRU) to get a detailed look at the tiny blood vessels in the kidneys. Researchers will compare images from 34 people with ADPKD and healthy volunteers to see if SRU can detect differences in blood …

This study from Mayo Clinic is looking at 100 people with autosomal dominant polycystic kidney disease (ADPKD) to see if certain markers in the blood and urine can predict how fast their kidney disease gets worse. Researchers will measure kidney size and function over time and co…

This observational study reviews medical records from 70 adults with advanced autosomal dominant polycystic kidney disease (ADPKD) who are already taking the drug octreotide LAR. Researchers want to see if baseline kidney volume and other factors can predict how quickly kidney fu…

This study looks at whether artificial intelligence can create realistic, privacy-safe copies of health records from people with a rare kidney disease called ADPKD. Researchers will use these simulated datasets to run virtual clinical trials and see if they produce similar result…