CYSTIC FIBROSIS

This study looks at the long-term safety and effectiveness of a triple combination drug (vanzacaftor/tezacaftor/deutivacaftor) for people with cystic fibrosis aged 1 and older. Participants who completed a prior study are invited to continue treatment. The goal is to manage the d…

This study tests whether empagliflozin, a diabetes medication, 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 to pl…

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 early-stage study tests a new medicine called VX-522 in 26 adults with cystic fibrosis whose genetic mutations do not respond to existing CFTR modulator drugs. The main goal is to check if the drug is safe and tolerable, and to see if it can improve lung function. Participan…

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 the long-term safety and effectiveness of the drug combination Trikafta (elexacaftor/tezacaftor/ivacaftor) in people with cystic fibrosis who have certain gene mutations. About 297 participants who completed a previous study will take the drug and be monitored…

This early-stage study tests VX-828, a new drug for cystic fibrosis (CF), in about 165 healthy adults and CF patients. The main goal is to check safety and how the drug moves through the body, both alone and in combination with other CF medicines. Results will help decide if furt…

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 looks at how different types of Achromobacter bacteria affect the lungs of people with cystic fibrosis. Researchers in Réunion Island will follow 17 patients to see if certain bacteria cause more lung infections or lead to long-term colonization. The goal is to better …

This study looks at 5,000 people with cystic fibrosis who are already taking approved CFTR modulator drugs. Researchers measure sweat chloride levels before and after treatment to see how they relate to health outcomes. The goal is to better understand how these medications work …

This study aims to understand how small blood vessels in the lungs are affected in people with cystic fibrosis (CF). Researchers will use imaging to measure blood vessel changes in 86 participants aged 5-21 with CF. The goal is to learn more about how CF lung disease progresses, …

This study follows 70 babies with cystic fibrosis to see if a germ called Porphyromonas catoniae, found in the mouth at 12 months old, can predict if they will get a serious lung infection with Pseudomonas aeruginosa by age 3. Researchers collect spit and stool samples over time …

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 aims to find out how nontuberculous mycobacteria (NTM) infections spread among people with cystic fibrosis (CF). Researchers will collect dust and water samples from clinics and patients' homes, and map watersheds to identify possible sources. The goal is to better und…

This study looks at whether a new type of MRI, using a special gas, can detect early lung changes in people with cystic fibrosis who are taking a new triple-combination therapy. About 64 participants will be scanned over a year to see how their lungs respond. The goal is to find …

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…