CYSTIC FIBROSIS

This study tests whether metformin, a standard diabetes medication, can improve how cells in the airways move salt and water in adults with cystic fibrosis-related diabetes (CFRD). Participants must already be taking a CFTR modulator drug. The trial will measure changes in gene a…

This study tests an experimental mRNA medicine called ARCT-032 in 12 adults with cystic fibrosis who cannot use or do not have access to standard CFTR modulator drugs. The goal is to see if the treatment is safe and can improve lung function and quality of life. Participants rece…

This study tests a triple combination medicine (VX-121/tezacaftor/deutivacaftor) in children aged 1 to 11 with cystic fibrosis who have a specific gene mutation. The goal is to see if the medicine is safe, tolerable, and works well in young kids. About 210 children will take part…

This study tests whether a structured diet and exercise program can increase lean muscle mass in adults with cystic fibrosis who are already taking the medication Trikafta. About 100 participants will be followed for 12 months. The goal is to improve muscle strength and overall h…

This study is testing a new, low-cost skin sensor that measures salt levels in sweat to diagnose cystic fibrosis (CF). Researchers will compare the sensor's results with standard lab tests in 30 adults—some with CF and some healthy volunteers. The goal is to see if this simple, w…

This study aims to see if a simple blood test from the mother can accurately diagnose genetic disorders like sickle cell disease, cystic fibrosis, and muscular dystrophy in unborn babies. The test uses fetal DNA found in the mother's blood and can be done as early as 9 weeks into…

This study is testing a smartphone app called AWARE that listens to your breathing to check for lung problems like asthma, COPD, and cystic fibrosis. Researchers want to see if the app can accurately measure lung function and spot disease flare-ups, just like standard hospital te…

This early-stage study is testing the safety of a new drug called VX-272 in 128 healthy adults. Participants will receive either the drug or a placebo, and researchers will monitor for side effects and measure how the drug moves through the body. The goal is to gather safety info…

This study looks at how the CFTR gene is organized and controlled in cells from the epididymis, a part of the male reproductive system. Researchers will collect leftover tissue from 20 men already scheduled for surgery, including those with cystic fibrosis or a condition causing …

This study looks at how well CFTR modulator drugs work in people with cystic fibrosis by measuring specific markers of CFTR function, such as sweat chloride and electrical signals in the nose and rectum. Researchers will follow 500 participants who are already taking these drugs …

This study aims to create better ways to measure how exercise affects children with cystic fibrosis, sickle cell disease, or long-term effects from COVID-19. Researchers will track about 240 kids over 3-4 years as they go through puberty, using advanced tools to measure their bod…

This study looks at how genes play a role in lung diseases like cystic fibrosis, asthma, and pulmonary fibrosis. Researchers will study the genes of up to 3,500 people, including healthy volunteers and those with lung disease. The goal is to find genetic differences that may expl…

This study follows 170 adults with cystic fibrosis and chronic sinusitis to see if sinus surgery improves lung function and quality of life compared to medical treatment alone. Participants either have surgery or continue with standard care. The goal is to learn which approach wo…

This study follows 400 people aged 12 and older who have or are at risk for noncirrhotic portal hypertension (NCPH), a liver condition that raises blood pressure in the liver without scarring. Researchers will use regular check-ups, imaging, and tests to see how the disease chang…

This study tests new ways to measure lung changes in people with cystic fibrosis (CF) aged 12 to 21. Researchers will use special MRI scans with a breathable gas (Xenon) and a lung function test called LCI to see what happens when patients stop and restart their daily airway clea…

This study aims to find out how many adults with cystic fibrosis smoke or vape, either actively or passively. Researchers will survey 100 adults who get regular care at a specialized center in France. The goal is to gather current data to improve tobacco prevention and understand…