Familial cystic renal disease

This early-stage study tests the safety of a new drug called AZD1613 in 40 adults with autosomal dominant polycystic kidney disease (ADPKD), a condition that causes kidney cysts. Participants receive either the drug or a placebo to check for side effects and how the drug moves th…

This early-stage study is testing a new drug called M0324, a bispecific antibody designed to target cancer cells. The trial will look at M0324 alone, with the immunotherapy pembrolizumab, and with chemotherapy in about 77 people with advanced solid tumors that have not responded …

This study tests an investigational drug called ABBV-CLS-628 in adults with autosomal dominant polycystic kidney disease (ADPKD), a genetic condition that causes fluid-filled cysts to grow in the kidneys. About 240 participants worldwide will receive either the drug or a placebo …

This study tests whether adding a common water pill (hydrochlorothiazide) to the standard drug tolvaptan can slow kidney function decline and reduce side effects like frequent urination in people with autosomal dominant polycystic kidney disease (ADPKD). About 300 adults on a sta…

This trial tests the safety of tolvaptan, a drug that helps the body remove extra water, in 20 children aged 28 days to 18 years with autosomal recessive polycystic kidney disease (ARPKD). The goal is to see if the drug can slow kidney function decline and reduce the need for dia…

This phase 3 trial tests whether the drug dapagliflozin can slow the loss of kidney function in adults with autosomal dominant polycystic kidney disease (ADPKD), a common genetic cause of kidney failure. The study will enroll 420 people aged 18 to 60 with rapid disease progressio…

This study is the first time a new drug called PYC-003 is being tested in humans. It is for people with a genetic kidney disease that causes cysts to grow and damage the kidneys. The trial will first test the drug in healthy volunteers to check safety, then in patients with the d…

This study tests whether adding dapagliflozin (a diabetes drug) to standard care reduces protein leakage in the urine of children with hereditary kidney diseases. About 44 children will receive either dapagliflozin plus standard care or standard care alone for 12 weeks, then swit…

This study tests whether a rotigotine skin patch, a drug used for Parkinson's disease, is safe for people with autosomal dominant polycystic kidney disease (ADPKD). ADPKD causes fluid-filled cysts in the kidneys and can lead to kidney failure. The trial will give 120 adults eithe…

This study looks at whether a well-formulated ketogenic diet can delay the progression of polycystic kidney disease in 20 adults who are at high risk for kidney failure. Participants will follow the diet for one year and have regular blood tests, urine tests, and MRI scans to tra…

This study looks at a non-surgical procedure called renal embolization to shrink enlarged kidneys in 30 dialysis patients with autosomal dominant polycystic kidney disease (ADPKD). The goal is to reduce kidney size enough to make a future kidney transplant possible and improve co…

This study tests whether metformin, a drug used for diabetes, can slow the loss of kidney function in people with early-stage autosomal dominant polycystic kidney disease (ADPKD). About 1,174 adults aged 18-70 with mild to moderate kidney decline will take metformin or a placebo …

This study tests a 16-week nutritionist-led ketogenic diet program (Ren-Nu) with a medical food called KetoCitra in 20 adults with autosomal dominant polycystic kidney disease (ADPKD). The goal is to see if the diet can improve kidney function and other health markers while monit…

This study is testing a new blood test that can diagnose single-gene disorders in unborn babies using a sample from the mother. The test looks at fetal DNA found in the mother's blood, which is safer than traditional invasive methods that carry a small risk of miscarriage. Resear…

This pilot study explores whether digital neurotherapy or coaching with a wellness coach can help manage depressive symptoms and pain in people with autosomal dominant polycystic kidney disease (ADPKD). Participants will be assigned to one of these approaches or to an education-o…

This study looks at whether foam sclerotherapy can reduce pain and improve quality of life for people with autosomal dominant polycystic kidney or liver disease who have large, painful cysts. Researchers will treat 100 adults with 1-3 dominant cysts and follow them for 12 months.…

This early-stage trial is the first to test GV-100, an oral drug being developed for autosomal dominant polycystic kidney disease (ADPKD), in healthy volunteers. The study aims to assess the drug's safety, tolerability, and how the body processes it, including the effects of food…

This study creates a registry to collect health information from people with Autosomal Dominant Polycystic Kidney Disease (ADPKD), the most common genetic cause of kidney failure. While ADPKD was once thought to only affect adults, researchers now know it begins in childhood, but…

This study is creating a central database and tissue bank for rare diseases affecting the kidneys and liver, such as ARPKD and Joubert syndrome. Researchers will collect medical information, genetic samples, and tissues from 200 participants to help doctors and scientists better …

This study is tracking up to 2,000 people with autosomal dominant polycystic kidney disease (ADPKD) who are taking or considering tolvaptan. Researchers will collect real-world data on dosing, side effects, and kidney function over time. The goal is to understand how the drug is …

This observational study is collecting blood and urine samples from 250 people with early-stage polycystic kidney disease (PKD), their unaffected family members, and healthy volunteers. The goal is to create a biobank that researchers can use to find biological markers of disease…

This study aims to improve diagnosis for people with rare genetic diseases whose standard genetic tests came back negative. Researchers will take a small skin sample and analyze RNA to find hidden genetic changes. The study involves 105 participants, including healthy volunteers …

This study aims to understand how oxidative stress and the body's antioxidant defenses work in early stages of autosomal dominant polycystic kidney disease (ADPKD). Researchers will measure various biomarkers in blood and urine, and use MRI to measure kidney size. The goal is to …

This study is looking for markers in urine and blood that can show how severe autosomal dominant polycystic kidney disease (ADPKD) is in its early stages. Researchers will measure NOX4, mitochondrial DNA, and other substances in 60 people aged 15-40 with early ADPKD. The goal is …

This study aims to find out how common high blood pressure is in children and teenagers (ages 12-17) who are at risk for autosomal dominant polycystic kidney disease (ADPKD) because a parent has it. Researchers will use MRI scans and genetic tests to detect early signs of the dis…

This study aims to develop better MRI-based tools to measure kidney disease progression in people with Autosomal Recessive Polycystic Kidney Disease (ARPKD), a serious genetic condition with no approved treatments. Researchers will enroll 45 ARPKD patients and 15 healthy voluntee…

This study looks at pain in people with ADPKD, a kidney disease that often causes pain in the belly, side, and back. Researchers will use sensory tests and questionnaires to understand the pain better. They will compare results from 150 patients with those from healthy volunteers…

This study is building a large database of health information from people with autosomal dominant polycystic kidney disease (PKD), the most common inherited kidney disease. Researchers will collect data from 1,000 adults over time to better understand complications like high bloo…

This phase 2 trial is testing whether the diabetes drug empagliflozin changes how the kidneys handle calcium, phosphate, and magnesium in people with autosomal dominant polycystic kidney disease (ADPKD). Forty adults will take either empagliflozin or a placebo for two weeks, then…

This study is creating a registry and biobank of 350 adults with autosomal dominant polycystic kidney disease (ADPKD) in the Mid-Atlantic region. Participants will provide medical information, have kidney volume measured by MRI, and donate blood, urine, and DNA samples. The goal …