Inherited kidney disorder

This study tests an experimental drug called BAY 3401016 in 60 adults aged 18 to 45 with Alport syndrome, a rare genetic condition that damages kidneys, hearing, and eyes. The drug works by blocking a protein thought to cause kidney injury, with the goal of slowing the loss of ki…

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 a drug called daratumumab in 40 people with a rare kidney condition called PGNMID, where abnormal proteins damage the kidneys. Participants receive weekly injections for 8 weeks, then every two weeks for another 8 weeks. The goal is to see if the drug can reduce …

This phase 2 trial tests an experimental drug called SGB-9768 in adults with three related kidney diseases: IgA nephropathy, C3 glomerulopathy, and immune complex-mediated membranoproliferative glomerulonephritis. These conditions occur when the immune system deposits proteins in…

This phase 2 trial tests whether theophylline, a drug used for asthma, can help people with pseudohypoparathyroidism lose weight and improve blood sugar control. The study includes 29 obese participants aged 13 and older. Researchers will measure changes in body mass index and gl…

This study tests whether theophylline, a drug used for asthma, can help children and young adults with pseudohypoparathyroidism—a rare genetic condition causing early obesity, hormone problems, and short stature. Researchers will check for weight loss, better blood sugar control,…

This phase 3 trial tests sefaxersen, a drug that targets a part of the immune system, in 428 people with IgA nephropathy at high risk of kidney decline. Participants receive either sefaxersen or a placebo as a shot under the skin. The main goal is to see if the drug reduces prote…

This trial tests whether sodium butyrate, a short-chain fatty acid, can reduce protein in the urine and slow kidney function decline in people with IgA nephropathy, a kidney disease. Participants receive either sodium butyrate or a placebo for 12 weeks, then switch after a washou…

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 looks at the long-term safety of a drug called iptacopan for adults with a kidney disease called primary IgA nephropathy. Participants have already completed earlier studies and will continue taking the drug to monitor side effects and see if it helps slow kidney damag…

This UCLA study is testing whether the Autoimmune Protocol (AIP) diet — which avoids dairy, grains, and sugar — can reduce protein levels in the urine of people with IgA nephropathy, a kidney disease that can lead to kidney failure. Thirty adults aged 18 to 65 with active disease…

This phase 2 trial tests the drug setmelanotide in one person with pseudohypoparathyroidism type 1a (PHP1a), a rare genetic disorder that often causes severe obesity. The participant will receive daily injections of setmelanotide for six months to see if it leads to weight loss a…

This Phase 3 trial tested sibeprenlimab, a drug given as a shot under the skin, in 530 adults with IgA nephropathy, a kidney disease. The study compared the drug to a placebo to see if it could reduce protein in the urine and protect kidney function over time. Participants were a…

This early-stage study tests two drugs together—linvoseltamab and dupilumab—in just 6 adults with severe food allergies. The goal is to see if the combination is safe and can lower IgE antibodies that trigger allergic reactions. Participants must have a history of anaphylaxis to …

This study tests a drug called sparsentan for people who have protein in their urine after a kidney transplant due to IgA nephropathy or FSGS. About 20 adults will take the drug once daily for 36 weeks to see if it safely reduces protein levels and protects kidney function. The g…

This phase 3 trial tests ravulizumab, a drug that blocks part of the immune system, against a placebo in 579 adults with IgA nephropathy—a kidney disease that can lead to kidney failure. The goal is to see if ravulizumab reduces protein in the urine and preserves kidney function …

This study looks at the long-term safety and effects of atacicept in people with IgA nephropathy, a kidney disease. Participants have already completed a previous trial with atacicept. Researchers will monitor side effects and measure kidney function over time to see if the drug …

This was a planned early-stage trial to test whether anti-CD19 CAR-T cells could safely help children with multi-drug resistant nephrotic syndrome, a serious kidney condition. The study was withdrawn before any patients were enrolled, so no data on safety or effectiveness were co…

This phase 3 trial tests whether the drug atrasentan can slow kidney function loss in people with IgA nephropathy, a disease that can lead to kidney failure. About 404 participants receive either atrasentan or a placebo, along with standard care. The main goal is to see if atrase…

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 phase 2 study tests the drug atrasentan in 103 adults with various kidney diseases that cause protein leakage, including IgA nephropathy and FSGS. Participants take a daily tablet to see if it reduces protein in urine, a sign of kidney damage. The goal is to find a new way t…

This phase 3 trial tests whether the drug povetacicept can reduce protein in the urine and preserve kidney function in adults with IgA nephropathy, a kidney disease. About 605 participants will receive either the drug or a placebo by injection. The study is active but not recruit…

This study looks at the long-term safety of a drug called sibeprenlimab for people with IgA nephropathy, a kidney disease. About 600 adults who completed earlier trials of this drug will continue receiving it for up to 2 more years. Researchers will track side effects and measure…

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 2 trial tests theophylline, a drug used for asthma, in 34 children aged 2 to 12 with pseudohypoparathyroidism, a genetic condition causing obesity, short stature, and hormone resistance. The study aims to see if theophylline can help with weight loss, slow bone growth …

This Phase 3 trial tests sparsentan, a new drug, against irbesartan (a standard treatment) in 406 adults with IgA nephropathy, a kidney disease. The goal is to see if sparsentan better protects kidney function over about 2 years. Participants must have significant protein in thei…

This phase 2 trial is testing a daily pill called belzutifan in 50 people with VHL disease who have kidney cancer. The goal is to see if the drug can shrink tumors. Researchers are also monitoring how long any tumor shrinkage lasts and checking for side effects.

This study tests a drug called atacicept given once a month to people with IgA nephropathy, a kidney disease that can lead to kidney failure. The goal is to see if monthly dosing lowers harmful antibodies and reduces protein in the urine, which are signs of kidney damage. About 9…

This study tests the long-term safety and effectiveness of pegcetacoplan in 100 people with two rare kidney diseases, C3 glomerulopathy and IC-MPGN. Participants previously completed a one-year trial and saw benefit from the drug. The goal is to see if pegcetacoplan can keep redu…

This phase 3 study tests BION-1301 (Zigakibart) in 383 adults with IgA nephropathy, a kidney disease that can lead to kidney failure. Participants receive either the drug or a placebo as a shot every 2 weeks for 2 years. The main goal is to see if the drug helps preserve kidney f…

This study was designed to test whether ADV7103, a combination of potassium citrate and potassium bicarbonate, could help people with cystinuria by making their urine less acidic. The goal was to see if it could raise urine pH to 7.0 or higher, which might reduce kidney stone for…

This study aimed to see if the drug tolvaptan could help infants with a severe form of polycystic kidney disease (ARPKD) avoid needing dialysis or a kidney transplant in their first year of life. The trial was planned for babies between 28 days and 12 weeks old, but it was withdr…

This study tests a drug called telitacicept in 318 adults with IgA nephropathy, a kidney disease that can lead to kidney failure. Participants are at high risk of worsening despite standard blood pressure medications. The goal is to see if telitacicept can reduce protein in the u…

This pilot study tests a new diet for people on hemodialysis who have high potassium levels. Instead of cutting out all high-potassium foods like fruits and vegetables, the diet focuses on avoiding foods with potassium that is easily absorbed, such as additives, processed meats, …

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 program provides early access to pegcetacoplan for people aged 12 and older with C3 glomerulopathy or primary IC-MPGN, rare kidney diseases that can lead to kidney failure. Participants must have active disease with significant protein in their urine. The goal is to offer a …

This study tests a new drug called povetacicept in adults with certain autoimmune kidney diseases, including lupus nephritis and IgA nephropathy. The goal is to see if the drug is safe and can help control the disease. Participants receive the drug every 4 weeks for at least 6 mo…

This phase 3 trial tests atacicept, a weekly injection, in 376 adults with IgA nephropathy (Berger's disease). The goal is to reduce protein in the urine and slow the loss of kidney function. Participants receive either atacicept or a placebo for about a year. The study is active…

Researchers are building deep learning models that analyze digital images of kidney biopsy tissue to classify kidney diseases such as IgA nephropathy and membranous nephropathy. The study uses pathology images and clinical data from thousands of patients who had kidney biopsies a…

This study offers voluntary screening for newborns in North Carolina to detect a wide range of rare health conditions early. Using a small blood sample already collected at birth, the program tests for dozens of disorders, including spinal muscular atrophy, cystic fibrosis, and m…

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 whether eating a diet high in magnesium can help prevent low blood magnesium levels in women with ovarian cancer who are receiving carboplatin chemotherapy. Low magnesium is a common side effect of this treatment. The study enrolled 26 participants and will me…

This trial investigates whether a daily effervescent supplement containing calcium, magnesium, and citrate can prevent bone loss, magnesium deficiency, and kidney damage in adults who take proton pump inhibitors (PPIs) long-term for conditions like heartburn or GERD. Participants…

This study at the University of Michigan is testing whether ultrasound can monitor bone graft healing after tooth extraction, potentially replacing more invasive scans. About 140 adults needing a tooth extraction and bone graft for a future dental implant will have their healing …

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 how a specific gene change (AGXT) affects oxalate production in people who have had calcium oxalate kidney stones. Four participants will eat a low-oxalate diet for five days and then receive an intravenous dose of glycolate. Researchers will measure oxalate l…

This study aims to identify the bacteria and fungi living in the urinary tract of people with calcium oxalate kidney stones or kidney cancer. Researchers will compare these microbial communities to those in healthy volunteers. The goal is to better understand how these tiny organ…

This study looks at how obesity changes the way the body handles oxalate, a substance that can form kidney stones. Researchers will give 22 calcium oxalate stone formers a controlled low-oxalate diet and a special tracer to measure oxalate absorption, kidney handling, and natural…

This observational study explores whether levels of a protein called soluble CD163 in blood or urine can reflect how active IgA nephropathy is and how it might progress. Researchers will compare these levels among people with IgA nephropathy, those with a similar kidney condition…

This study was designed to see if a new method of measuring the visual field—by projecting vision zones on the ground—helps people with retinitis pigmentosa understand their remaining sight and improve their walking rehabilitation. The trial was withdrawn before enrolling any par…

This study follows 600 people with Albright hereditary osteodystrophy, a rare genetic condition affecting bones and hormones. Researchers are looking at how growth hormone treatment affects height and weight in those with a related hormone problem, and also studying thinking and …

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 looks at how older adults (65+) with cancer understand their illness and what they value in life before and after a major operation. Researchers will interview 100 patients to learn about their awareness, expectations, and decision-making. No new drug or treatment is b…

This study from Mayo Clinic looks at whether certain gene variants make people more likely to form calcium oxalate kidney stones. Researchers will compare 2,900 adults with and without a history of kidney stones, analyzing blood samples and lifestyle factors. The goal is to bette…

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 follows over 3,000 Black people with HIV in the UK to understand how genes and lifestyle affect their risk for kidney disease, heart disease, diabetes, and obesity. Researchers collect health data and blood/urine samples to find genetic markers that predict these condi…

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…