CYSTIC FIBROSIS

This study tests whether a special MRI scan can better measure how well a new triple therapy works for cystic fibrosis. 64 participants will have MRI scans before and after starting the therapy. The goal is to find a more sensitive way to track lung health and guide future treatm…

This phase 3 study is testing the long-term safety and effectiveness of a triple combination therapy (vanzacaftor/tezacaftor/deutivacaftor) in people with cystic fibrosis aged 1 year and older. The 174 participants have already completed a previous study of the same drugs. The go…

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 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 study tests whether empagliflozin, a diabetes drug, is safe and tolerable for overweight adults with cystic fibrosis-related diabetes (CFRD). Eight participants will receive either the drug or a placebo, then switch after a break. The goal is to gather early data for a large…

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 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 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 study looks at the long-term safety and effectiveness of a triple-combination medicine (elexacaftor/tezacaftor/ivacaftor) for people with cystic fibrosis who are at least 12 months old. Participants must have completed a previous related study. The main goal is to track side…

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 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 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 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 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 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 different types of Achromobacter bacteria affect the lungs of people with cystic fibrosis in Réunion Island. Researchers will track 17 patients to see if certain species cause more lung infections or lead to chronic colonization. The goal is to better unde…

This study looks at how different genes affect the severity of lung disease in people with cystic fibrosis. It is an observational study, meaning no treatment is given. Researchers will follow 600 participants with CF to understand why some have milder or more severe lung problem…

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 follows 285 pregnant women with cystic fibrosis to see how their lung function changes during pregnancy and for two years after giving birth. Researchers are focusing on those taking highly effective CFTR modulators. The goal is to better understand the effects of preg…