ABeta2M amyloidosis

This study tests whether semaglutide, a diabetes drug, is safe and tolerable for people with type 2 diabetes who are on dialysis. About 100 participants will either receive weekly semaglutide injections or standard care for 26 weeks. The goal is to see if patients can stick with …

This study tests a bone marrow transplant from a family donor for children under 21 with severe non-cancer blood disorders like sickle cell disease, bone marrow failure, or immune problems. The goal is to see if the transplant can replace the diseased cells with healthy donor cel…

This phase 3 trial tests whether AP301, a drug taken with meals, can safely lower high blood phosphate in people on kidney dialysis. About 264 participants will receive either AP301 or a low-dose version for 8 weeks, then all get AP301 for 24 weeks. The goal is to see if AP301 he…

This study tests whether applying an ice pack or a warm gel pad to the drain site before removal can lower anxiety, improve comfort, and stabilize vital signs in patients recovering from open urological surgery. 120 adults will be randomly assigned to ice, warm gel, or standard c…

This study looks at children with suspected or confirmed genetic and metabolic disorders to find new disease-causing gene mutations. Researchers will analyze blood samples for DNA and metabolites, and in some cases take a small skin sample. The goal is to better understand these …

This pilot trial is testing whether amyloidosis patients can use the MyFitnessPal smartphone app to track their daily food and supplement intake for 8 weeks. The goal is to see if this low-cost method is feasible for studying malnutrition and weight loss in this condition. Forty …

This study enrolls up to 15,000 children with known or suspected endocrine or metabolic disorders, along with their family members. Researchers will collect medical records, blood, saliva, and other samples to identify genetic changes linked to these conditions. The goal is to be…

This study is creating a large collection of blood, urine, stool, and heart tissue samples from 10,000 people with and without heart or metabolic conditions. The goal is to store these samples along with medical information to speed up future research into what causes these disea…

This study follows 100 obese patients starting dialysis to understand why they are less likely to receive a kidney transplant. Researchers will track health changes over two years and compare them to non-obese patients. The goal is to identify barriers and improve access to trans…

This study tests a new communication tool called 'Let's Get REAL' that helps children and teens (ages 8-17) and their families talk together about stem cell transplant or cellular therapy decisions. The goal is to see if the tool is easy to use and helpful for families. About 60 …

This study is observing 1,000 critically ill adults in Latin American ICUs who need dialysis for severe acute kidney injury. Researchers will track treatments, outcomes, and country differences to understand what works best. The goal is to gather data that can improve care and gu…

This study is creating a biobank and data registry for people with amyloidosis, a rare disease where abnormal proteins build up in organs. Researchers will collect blood, urine, tissue samples, and medical information from 505 participants over time. The goal is to store these ma…

This study uses MRI scans to look for brain differences in people with metabolic diseases compared to healthy volunteers. Researchers will track changes over time and link them to body fat and other health measures. About 126 adults will take part at Ulm University Hospital. No d…

This study is a large registry that will collect health information from up to 60,000 people with both heart and kidney disease. The goal is to better understand how these conditions interact and to find ways to predict and improve patient outcomes. No new treatments or procedure…

This study looks at whether a campaign called Choosing Wisely can safely reduce unnecessary tests and treatments in intensive care units (ICUs). About 10,000 adult patients in Swedish ICUs will take part. The goal is to see if this approach lowers costs and improves care without …

This study is creating a large collection of blood, urine, and tissue samples from up to 10,000 people with amyloid diseases (like multiple myeloma). The goal is to store these samples and health data for future research to better understand these conditions. Participants do not …

The SMILE study is tracking 10,000 adults aged 18 and older to see how sarcopenia (age-related muscle loss) and metabolic diseases like diabetes influence overall health and lifespan. Researchers will collect data from medical records, tests, and surveys over time. This is an obs…

This study follows 100 children under 18 who are already on a ketogenic diet for conditions like drug-resistant epilepsy, autism, chronic migraine, or brain tumors. Researchers want to see if the diet supports healthy growth, improves symptoms, and boosts quality of life. The die…