Telomere syndrome

This study tests a new drug called elafibranor in people with primary biliary cholangitis (PBC), a rare liver disease that can lead to scarring and liver failure. Participants either did not get enough help from or could not take the standard medicine. The goal is to see if elafi…

This study tests a daily pill called elafibranor in about 18 Japanese adults with primary biliary cholangitis (PBC), a rare liver disease that can lead to scarring and liver failure. Participants had not responded well to standard treatment. The main goal is to see if the drug im…

This study tests whether elafibranor, a daily pill, can normalize alkaline phosphatase (ALP) levels in adults with primary biliary cholangitis (PBC) who did not respond well to or cannot take standard treatment. About 69 participants will receive either elafibranor or a placebo f…

This study tests two doses of K-808 (Pemafibrate) in 46 adults with primary biliary cholangitis (PBC) who haven't responded well to standard treatments. The main goal is to see if the drug lowers alkaline phosphatase (ALP) levels, a key marker of liver damage, after 12 weeks. Par…

This study tests a drug called saroglitazar magnesium in people with primary biliary cholangitis (PBC), a chronic liver disease. The goal is to see if the drug is safe and helps control the disease. About 102 adults who completed a previous PBC study will take the drug and be mon…

This study is testing the long-term safety of a drug called seladelpar in people with primary biliary cholangitis (PBC), a chronic liver disease. About 340 adults who have taken seladelpar before will receive either 5 mg or 10 mg daily for several years. Researchers will monitor …

This study tests a new drug (ADI-PEG 20) combined with chemotherapy and radiation before surgery for soft tissue sarcoma. The goal is to find the safest dose and see how well it shrinks tumors. Only 6 patients with intermediate or high-grade sarcoma of the trunk or limbs are taki…

This phase 2 study tests a new drug called CS0159 in 75 adults with primary biliary cholangitis (PBC), a chronic liver disease. Participants receive either CS0159 or a placebo for 12 weeks, followed by 40 weeks of open-label treatment. The goal is to see if CS0159 is safe and can…

This early-stage trial tests a single infusion of EXG34217 in 12 people aged 12 and older who have bone marrow failure due to telomere biology disorders. The main goal is to check safety and tolerability, while also looking at changes in telomere length and blood cell counts. Bec…

This study tests whether adding fenofibrate (a drug used for high cholesterol) to standard treatment with ursodeoxycholic acid can improve liver health in people with primary biliary cholangitis (PBC), a rare autoimmune liver disease. About 184 adults with PBC will receive either…

This study looks at whether adding prednisone to standard UDCA treatment helps people with primary biliary cholangitis (PBC) who also have moderate to severe interface hepatitis, a type of liver inflammation. About 244 adults will be randomly assigned to receive either UDCA alone…

This study is testing a reflex blood test to find primary biliary cholangitis (PBC) earlier in women aged 40 and older. When routine blood work shows certain liver enzyme elevations, the lab automatically checks for antibodies linked to PBC. The goal is to see if this automatic f…

This study tests the long-term safety of linerixibat, a drug for severe itching caused by primary biliary cholangitis (PBC). All 242 participants receive the drug openly. The goal is to see if it remains safe over time and improves quality of life.

This study at Stanford University is testing whether wearable devices like smartwatches can help measure fatigue, sleep, and daily activity in people with primary biliary cholangitis (PBC), a chronic liver disease. Researchers will compare data from the wearables with patient que…

This study looks at how a type of medication called PPAR agonists affects blood sugar levels in 30 adults with primary biliary cholangitis (PBC), a chronic liver disease. Researchers will measure changes in blood sugar and other metabolic markers over three months. The goal is to…

This study follows 250 Italian adults with genetic forms of pulmonary fibrosis to see how their lung function changes over time. Researchers will track breathing tests and compare patients who receive immune-modulating drugs with those who do not. The goal is to understand how in…

This observational study follows 1,222 adults with liver cirrhosis to understand why some develop serious complications like fluid buildup or bleeding. Participants have check-ups every six months, including questionnaires, physical exams, and imaging. The goal is to build better…

This observational study plans to enroll 120,000 people with various chronic diseases like pancreatitis, diabetes, and arthritis. Researchers will collect blood samples and health data to study how genetics and lifestyle affect disease progression. The goal is to find common dise…