CYSTIC FIBROSIS

This study is checking the long-term safety and effectiveness of a three-drug combination (VX-121/TEZ/D-IVA) for people with cystic fibrosis. It follows 822 participants who previously took these medications in earlier studies, tracking side effects and lung function over time. T…

This small pilot study is testing if adding the diabetes drug empagliflozin to standard insulin therapy is safe and helps control blood sugar in overweight adults with cystic fibrosis-related diabetes (CFRD). The main goal is to gather initial safety and feasibility data to see i…

This study follows 297 cystic fibrosis patients to see how safe and effective a triple-combination drug remains over the long term. The treatment targets specific genetic mutations beyond the most common one. Participants had previously taken the medication in earlier studies and…

This study follows 5 people with cystic fibrosis for 15 years to monitor the long-term safety and lasting effects of a single-dose inhaled gene therapy they received in a previous trial. Participants do not receive new treatment but undergo regular health checks and lung function…

This study is observing children with cystic fibrosis (CF) to see how a powerful, already-approved triple-drug therapy affects their health over two years. It involves 180 kids, ages 6 to 11, who are starting this treatment. Researchers will measure changes in lung function, swea…

This study is testing the long-term safety and effectiveness of a three-drug combination therapy for people with cystic fibrosis aged 1 year and older. It follows participants who have already taken the medication in a previous trial to see how they do over a longer period. The m…

This small, early-stage study is testing whether losartan, a common blood pressure medication, can help improve mucus clearance in adults with cystic fibrosis who cannot take newer, targeted CFTR therapies. Researchers want to see if reducing inflammation with losartan for 14 wee…

This study is checking if standard cystic fibrosis (CF) treatments help improve bone density and muscle mass in adults with CF. Researchers are observing 63 adults over 12 to 24 months to see if these treatments can help prevent bone loss and muscle weakness. The goal is to bette…

This study is checking the long-term safety and effectiveness of a three-drug combination treatment (elexacaftor/tezacaftor/ivacaftor) for cystic fibrosis. It involves about 50 people aged 12 months and older who have already taken the drug in a previous trial. The main goal is t…

This study aims to see if two different at-home stool tests can work as well as a colonoscopy for finding colon cancer and precancerous growths in adults with cystic fibrosis. Researchers will compare results from the stool tests to the standard colonoscopy procedure in 350 parti…

This study is testing whether a special educational website can help people with advanced cystic fibrosis feel more prepared to talk with their doctors about lung transplant. Researchers want to see if learning about transplant options earlier can help patients make informed deci…

This early-stage study aims to understand how a new cystic fibrosis drug combination affects the body's processing of a common cholesterol medication called rosuvastatin. Researchers are testing this in 18 healthy volunteers to check for safety and measure drug levels in the bloo…

This study aims to understand how people with cystic fibrosis (CF) catch nontuberculous mycobacteria (NTM) lung infections, which are difficult to treat. Researchers will follow 100 people with CF to see if infections spread between patients in healthcare centers or come from sha…

This study aims to understand how a key sweat test measurement changes in people with cystic fibrosis who are taking approved modulator drugs. It will enroll about 5,000 participants who have been on their medication for at least 3 months for a single visit to collect a sweat sam…

This study aims to understand how cystic fibrosis affects the small blood vessels in the lungs. Researchers will compare lung scans from 86 children and young adults with cystic fibrosis to scans from people without lung disease. They will also track changes in some participants …

This study aims to understand how pregnancy affects lung function in women with cystic fibrosis who are taking newer, highly effective CFTR modulator medications. Researchers will follow 285 pregnant women with cystic fibrosis at 40 US sites, checking their lung function every 3 …

This study aims to understand if a specific bacteria found in babies with cystic fibrosis can predict who will develop a serious lung infection called Pseudomonas aeruginosa by age 3. Researchers will follow 70 infants from birth to age 3, collecting samples like mucus and stool …

This study aims to understand why some people with cystic fibrosis develop serious liver disease while others do not. Researchers are looking at genes other than the main CF gene that might influence this risk. They are analyzing blood samples and medical information from about 1…

This study aims to understand how genetics, lifestyle, and other factors influence the progression and severity of many common chronic diseases. Researchers will collect health records, genetic data, and samples from 120,000 participants with conditions like diabetes, arthritis, …

This study aims to understand how cystic fibrosis (CF) affects blood flow in the lungs. Researchers are using a new, safe MRI method that doesn't require injection dye to map blood flow in children with CF. They will compare these blood flow maps to breathing tests and blood mark…

This study aims to understand how body composition, muscle strength, and nutrition relate to lung function and overall health in adults with cystic fibrosis. Researchers will measure 300 participants using scans, strength tests, and questionnaires to create detailed health profil…

This study aims to understand why lung disease varies so much in people with cystic fibrosis (CF), even when they have the same genetic mutation. Researchers are analyzing the DNA of 600 CF patients to identify other genes that might make the lung disease milder or more severe. T…

This study is testing a special, radiation-free MRI scan that uses a safe inhaled gas to create detailed pictures of the lungs. The goal is to see if this scan can detect very early changes in lung health in children with cystic fibrosis, especially as they start new, highly effe…

This study is testing a new, high-resolution imaging tool to take detailed pictures inside the lungs and nose. The goal is to better understand diseases like cystic fibrosis, COPD, and COVID-19, which could help guide future treatments. Researchers are enrolling 60 adults and tee…