CYSTIC FIBROSIS

This study looks at the long-term safety and effectiveness of the drug combination Trikafta (elexacaftor/tezacaftor/ivacaftor) in people with cystic fibrosis who have certain non-F508del gene mutations. About 297 participants who completed a prior study will continue taking the m…

This phase 3 study is testing the long-term safety and effectiveness of the drug combination elexacaftor/tezacaftor/ivacaftor (known as Trikafta) in 50 people with cystic fibrosis who are at least 12 months old. Participants previously completed a related study and will continue …

This study tests a triple-combination drug (VX-121/tezacaftor/deutivacaftor) in children aged 1 to 11 with cystic fibrosis who have a specific genetic mutation. The goal is to check how the medicine works in the body, its safety, and how well children tolerate it. Around 210 chil…

This early-stage study tests the safety and how the body handles a new drug called VX-828, alone or combined with other CF medicines. It involves about 165 healthy adults and people with cystic fibrosis. The goal is to see if VX-828 is safe enough for further testing.

This early-stage trial tests an inhaled mRNA therapy called VX-522, combined with an existing drug (ivacaftor), in 26 adults with cystic fibrosis who have genetic mutations that do not respond to current modulator treatments. The main goals are to check safety and tolerability, a…

This study tests a triple-combination drug (VX-121/TEZ/D-IVA) for people with cystic fibrosis, a genetic condition that affects the lungs and digestion. The goal is to see if the treatment is safe and works well over a long period. About 822 participants who completed earlier stu…

This phase 3 study tests the long-term safety and effectiveness of a triple combination drug (vanzacaftor/tezacaftor/deutivacaftor) in 174 people with cystic fibrosis aged 1 and older. Participants who completed a prior study will receive the drug to see if it helps control sympt…

This pilot study tests the safety and feasibility of empagliflozin (Jardiance) in 8 overweight or obese adults with cystic fibrosis-related diabetes (CFRD) who are already on insulin. Participants will receive either the drug or a placebo in a crossover design. The goal is to gat…

This study tests a special MRI technique that uses inhaled gas to create detailed images of lung function in people with cystic fibrosis. Researchers want to see how a new triple-combination therapy changes lung ventilation over time. The study involves 64 participants and aims t…

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 looks at whether stool-based tests (like the FIT test and Cologuard) can accurately detect colorectal cancer and precancerous polyps in adults with cystic fibrosis. Researchers will compare these non-invasive tests to the standard colonoscopy in 350 participants. The g…

This study looks at whether a bronchodilator (a drug that opens airways) can reduce shortness of breath and improve exercise ability in adults with cystic fibrosis. Twenty participants will receive either the drug or a placebo and then do exercise tests to compare their breathing…

This study looks at whether a home-based strength exercise program can improve muscle health in 48 children and teens with cystic fibrosis who are taking new CFTR modulator drugs. Participants are split into an exercise group and a control group. Researchers will measure muscle s…

This study follows 70 infants with cystic fibrosis to see if a specific germ (Porphyromonas catoniae) found in their body at 12 months old can predict whether they will get a serious lung infection with Pseudomonas aeruginosa by age 3. Researchers collect sputum, stool, and blood…

This study looks at how different types of Achromobacter bacteria affect the lungs of people with cystic fibrosis on Réunion Island. Researchers will analyze sputum samples from 17 patients to see if certain species cause more lung infections or lead to worse outcomes. The goal i…

This study planned to test whether a specific breathing technique called autogenic drainage helps clear mucus and improve lung function in people with cystic fibrosis. The study was withdrawn before enrolling any participants, so no results are available. It would have measured c…

This study follows 5 people with cystic fibrosis who previously received a single dose of BI 3720931 gene therapy or placebo. No new treatment is given. Researchers will monitor participants for up to 15 years to check for long-term side effects and track lung function. The goal …

This study aims to find the genes and other factors that explain why some people with cystic fibrosis develop diabetes while others do not. Researchers will analyze DNA from 1,500 people with cystic fibrosis and their parents. The goal is to better understand the causes of cystic…

This study is measuring body composition, muscle strength, and nutrition in 300 adults with cystic fibrosis. Researchers are comparing simple tests like grip strength and walking distance to a standard body scan. The goal is to find better ways to track health and guide future tr…

This study uses advanced MRI scans to track lung structure and function in people with cystic fibrosis who are taking CFTR-modulator therapy. Researchers will compare MRI results with standard breathing tests and quality-of-life measures. The study includes healthy volunteers, st…

This study tests a new, non-contrast MRI technique to map blood flow in the lungs of 26 children with cystic fibrosis (ages 6–21). The goal is to see if blood vessel damage happens early, before standard breathing tests show problems. Participants will be scanned at the start and…

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…

This study tests whether a research website helps people with cystic fibrosis (CF) feel more prepared to discuss lung transplant with their doctors. About 132 adults with CF and low lung function will use the website and attend Zoom sessions over 6 months. The goal is to increase…

This study aims to understand how small blood vessels in the lungs change as cystic fibrosis (CF) lung disease progresses. Researchers will use imaging to measure blood vessel volume in 86 people with CF aged 5-21. The goal is to learn more about the disease, not to test a new tr…

This study is testing a tiny camera called micro OCT that can take detailed pictures of cells inside the lungs and nose. Researchers want to see if it works well in people with cystic fibrosis and other lung conditions. The goal is to eventually use this imaging to detect early d…

This study follows 285 pregnant women with cystic fibrosis to see how CFTR modulators affect lung function during and after pregnancy. Researchers will measure breathing capacity before, during, and up to two years after delivery. The goal is to better understand how these medica…

This study aims to understand how nontuberculous mycobacteria (NTM) spread among people with cystic fibrosis (CF). NTM infections are hard to treat and common in CF, but their sources are unclear. Researchers will track 100 participants with CF and NTM, testing dust and water in …

This study looks at 5000 people with cystic fibrosis who are already taking approved CFTR modulator medicines. Researchers measure sweat chloride levels before and after treatment to see how the drugs affect the body. The goal is to better understand the link between sweat chlori…

This study looks at how CFTR modulators, a type of cystic fibrosis treatment, affect bone strength and muscle mass in adults with CF. Researchers will measure changes in bone density and lean body mass over 12 to 24 months. The goal is to understand whether these treatments help …

This study looks at genes, other than the CF gene, that might explain why some people with cystic fibrosis develop severe liver disease while others do not. Researchers are analyzing blood samples and medical records from 154 participants with CF who have serious liver problems. …

This study examines how different genes influence the severity of lung disease in people with cystic fibrosis. Researchers will analyze genetic data from 600 participants to identify gene variants linked to milder or more severe lung problems. The goal is to better understand the…