CYSTIC FIBROSIS

This study tested the long-term safety of a triple combination therapy (VX-445, tezacaftor, and ivacaftor) in 507 people with cystic fibrosis who have the F508del mutation. Participants had already completed a previous study of the same drug. The main goal was to see how safe and…

This study tested a new drug called CMTX-101 in 43 adults with cystic fibrosis who have a chronic lung infection. The drug is designed to break down the slimy coating bacteria make, helping standard antibiotics work better. The main goal was to see if it is safe and how the body …

This study tested a combination of two drugs, VX-661 and ivacaftor, in 40 adults with cystic fibrosis who have two copies of the F508del gene mutation. The goal was to see if the combination is safe and improves lung function over 12 weeks. Participants were randomly assigned to …

This study followed 852 people with cystic fibrosis (ages 12 and older) who were taking the drug Orkambi outside of a clinical trial. Researchers wanted to see how many stopped treatment, why, and how their lung function and nutrition changed over one year. The goal was to unders…

This study tested a new inhaled medicine called ETD001 in 57 adults with cystic fibrosis. The goal was to see if it is safe and if it can improve lung function. Participants took the drug twice a day for up to 28 days and had their breathing tested. The results will help decide i…

This study tested a quicker treatment plan for early MRSA lung infections in 37 people with cystic fibrosis (ages 2 to 45). The goal was to see if the streamlined approach could clear the bacteria better than standard care. Researchers tracked infection clearance at 28 days and m…

This study looked at the long-term safety and effectiveness of the triple-combination drug Trikafta (elexacaftor/tezacaftor/ivacaftor) in 71 children aged 2 and older with cystic fibrosis. All participants had already taken the drug in an earlier study. The main goal was to track…

This study tested the drug Trikafta in 42 people with cystic fibrosis who have rare gene changes not normally treated with this medication. Participants took Trikafta for about four weeks, and researchers measured lung function and sweat chloride levels. They also used skin or bl…

This study tested a single dose of a special virus mixture (bacteriophages) given intravenously to adults with cystic fibrosis who have chronic lung infections with Pseudomonas aeruginosa bacteria. The goal was to see if the treatment is safe and can reduce the amount of bacteria…

This study tested a new tablet called BI 1291583 in 22 adults with cystic fibrosis and bronchiectasis (damaged airways). Participants took either the study drug or a placebo once daily for 12 weeks. The main goal was to check safety and side effects, not to cure the disease. Resu…

This study tested a combination of three drugs (Dirocaftor, Posenacaftor, Nesolicaftor) in 41 adults with cystic fibrosis who have rare gene mutations not covered by existing treatments. Participants were selected based on lab tests using their own intestinal organoids to predict…

This study looked at 57 French cystic fibrosis patients aged 6 and older with a G551D mutation who were taking Ivacaftor. Researchers wanted to see if the drug remained effective and safe in everyday use after it was approved. They measured lung function, hospital stays, and anti…

This study tested whether the drug miglustat can help fix a key protein problem in people with cystic fibrosis who have the F508del gene mutation. Sixteen adults took either miglustat or a placebo, then switched, to see if the drug improved chloride movement in their nasal passag…

This study tested a triple-combination drug (elexacaftor/tezacaftor/ivacaftor) in 70 toddlers with cystic fibrosis who are 12 to 24 months old. The goal was to see how the drug moves through the body and to check its safety. All children had at least one specific gene mutation th…

This small early-phase study tested whether losartan, a blood pressure medication, could improve mucus clearance in the lungs of 13 adults with cystic fibrosis who were not eligible for standard CFTR therapies. Participants took losartan for over 12 weeks. The goal was to see if …

This study looked at how the drug combination Trikafta (elexacaftor/tezacaftor/ivacaftor) affects coughing and physical activity in 82 people with cystic fibrosis aged 12 and older. Participants wore activity trackers to measure cough frequency and step counts over 12 weeks. The …

This study looked at how well a triple combination therapy (elexacaftor, tezacaftor, and ivacaftor, or ETI) works in children aged 6 to 11 with cystic fibrosis (CF) who have a specific genetic mutation. Researchers measured changes in sweat chloride, lung function, and growth ove…

This study looked at whether adding an inhaled medication called Bronchitol (mannitol) can improve how well the lungs clear mucus in people with cystic fibrosis who already take a powerful CFTR modulator drug. Fourteen adults with moderate-to-severe lung disease took part. The go…

This study tested a new inhaled medicine called MRT5005 in 42 adults with cystic fibrosis. The goal was to see if it is safe and to measure how it affects lung function. The medicine was given as a mist through a nebulizer, and participants were monitored for side effects and cha…

This small pilot study tested a GLP-1 medication (semaglutide) added to insulin in 8 overweight adults with cystic fibrosis-related diabetes. The main goals were to see if the drug is safe and helps with weight loss and blood sugar control. Results will guide future research on m…

This study tested a new oral tablet called brensocatib in adults with cystic fibrosis. Researchers wanted to understand how the drug moves through the body and check its safety over a 4-week period. The trial involved 29 participants who took either the drug or a placebo, and it …

This study tested whether a noninvasive device called FibroScan can accurately measure liver scarring (fibrosis) in children, potentially replacing the standard but painful liver biopsy. Researchers compared the device's readings to biopsy results in 264 children with various liv…

This study tested four different non-invasive methods (FibroTest, Fibroscan, ShearWave elastography, and magnetic resonance elastography) to measure liver scarring in 56 children with cystic fibrosis aged 6-18. The goal was to see which test best detects early liver fibrosis, hel…

This study tested a new program to help people with cystic fibrosis (CF) feel better day-to-day by addressing symptoms and emotional needs. Over 600 patients at five CF centers took part. The program included regular check-ins and guides for doctors to provide extra support, aimi…

This study tested a program to help children aged 8-12 with cystic fibrosis and their mothers build life skills and psychological resilience. The program included activities like laughter yoga and support sessions. Researchers measured lung function, quality of life, and emotiona…

This study tested whether a home-based exercise program, supervised remotely via video calls, could improve fitness in children and teens with cystic fibrosis. Ten participants aged 8 to 21 used an activity tracker and did cycling tests to measure their oxygen use and daily activ…

This study tested whether starting a structured exercise program combined with behavioral counseling during a hospital stay is feasible for adults with cystic fibrosis. Three participants joined the program while hospitalized for a lung flare-up. The goal was to see if this appro…

This study tested whether a voice journaling app called Kintsugi is easy and acceptable for teens aged 14–18 with cystic fibrosis. Twenty participants used the app over three months to track their feelings. The goal was to see if it could help reduce anxiety and depression sympto…

This study tested whether a video-based exercise program is practical and acceptable for adults with cystic fibrosis after a flare-up. Nine participants tried the program at home. The goal was to see if people would join and stick with it, not to measure health changes.

This study tested a medical device called Simeox against a standard breathing technique (autogenic drainage) in 31 adults with cystic fibrosis. The goal was to see which method better clears mucus from the lungs and improves breathing. Participants used both methods in separate s…

This study looked at whether adding relaxation sessions at home to standard medical and psychological care can help reduce chronic pain in people with cystic fibrosis. Thirteen patients aged 10 and older with frequent or constant pain took part. The goal was to find a better way …

This study looked at whether CFTR modulator drugs improve nose and sinus symptoms in children aged 6 to 11 with cystic fibrosis. Researchers measured quality of life using a special questionnaire before and one year after starting treatment. The goal was to see if these drugs hel…

This study looked at whether using a Fitbit fitness tracker helps adults with cystic fibrosis exercise more regularly. Over one year, 40 participants used the device to track their activity. The goal was to see if this leads to better exercise tolerance and lung function.

This completed study looked at 62 adults with cystic fibrosis to see how their health literacy (understanding health information) relates to physical activity, anxiety, depression, and how well they follow airway clearance treatments. Researchers used surveys to measure these fac…

This study looked at 29 adults with cystic fibrosis who were hospitalized for a lung infection. Researchers collected blood, sputum, and urine to study how infection and inflammation affect recovery of lung function. The goal is to find better ways to personalize treatment and im…

This study looked at 85 women with cystic fibrosis to find out how many carry the HPV virus, which can lead to cervical cancer. Researchers checked for HPV and abnormal cervical cells using a Pap test and DNA testing. The goal is to help doctors better understand the need for reg…

This study looked at 72 adults with cystic fibrosis to see if chemical changes in their DNA (called DNA methylation) are linked to how well their lungs work. The goal was to understand why lung disease gets worse at different rates in different people. No treatment was given; ins…

This study involved 8 children and teens with cystic fibrosis who had a history of MRSA infection and were receiving the antibiotic ceftaroline. Researchers measured drug levels in the blood at different times to understand how the body processes the medicine. The goal was to fin…

This study aimed to understand the thoughts, expectations, and needs of adults with cystic fibrosis (CF) who are parents or planning to become parents. Researchers held group discussions and individual interviews with 52 participants, including patients and their spouses. The goa…

This study followed 58 adults with cystic fibrosis (CF) who were starting or switching to a new CFTR modulator medication as part of their regular care. Researchers collected blood, sputum, urine, and quality-of-life surveys before and after treatment to see how inflammation, lun…

This study followed 75 people with cystic fibrosis (CF) for two years to see how many developed antibodies from COVID-19 infection or vaccination. Researchers collected blood samples and health data to understand if CF patients have different immune responses or milder COVID-19 s…

This study looked at sputum (mucus) from 83 adults with cystic fibrosis who were hospitalized for sudden lung worsening (called a pulmonary exacerbation). Researchers used genetic testing to find which antibiotic-resistant bacteria were present and compared those results to how w…

This study looked at lungs removed from 74 people with cystic fibrosis during transplant to see how bacteria and chemistry differ from one area to another. Researchers took samples from each lobe and used DNA sequencing to identify which bacteria were present and how active they …

This study looked at blood neutrophils (a type of white blood cell) in 47 adults with cystic fibrosis to see how they differ from healthy people. Researchers checked how these cells work and change with chronic infection, CFTR modulator treatment, and sudden worsening of symptoms…

This study looked at whether people with cystic fibrosis (CF) have COVID-19 antibodies and how their bodies respond to the virus or the vaccine. Over two years, 152 adults and children with CF from European clinics gave blood samples to check for antibodies. The goal was to under…

This study looked at how the antibiotic omadacycline is processed by the body in 9 adults with cystic fibrosis. Participants received the drug both by IV and by mouth so researchers could measure levels in the blood. The goal was to understand dosing, not to treat the disease dir…

This study talked to 27 children (ages 6 and up) who have a parent with cystic fibrosis, a serious lung disease. Through interviews and group discussions led by a psychologist, researchers aimed to understand the children's feelings, worries, and needs. The goal is to use this in…

This study looked at 48 children (ages 6-18) to see if those with cystic fibrosis have different muscle endurance compared to healthy kids. Researchers measured core, leg, and breathing muscle strength and endurance. The goal was to better understand how CF affects physical fitne…

This study looked at how bacteria grow in the nose, mouth, and lungs of 7 infants with cystic fibrosis during their first year of life. Researchers collected swabs and mucus samples to map bacterial communities. The goal was to understand how germs move through the airway, which …

This study looked at what influences daily physical activity in 80 adults with cystic fibrosis (CF). While most research focuses on lung health, this study also examined nutrition, diabetes, bone health, anxiety, and heart function. Participants wore an activity monitor for a wee…

This study compared two types of lung scans—ultra-low-dose CT and lung MRI—to see how well they show lung damage in adults with cystic fibrosis. 185 participants received both scans, and doctors checked if the results matched those from a standard low-dose CT. The goal is to find…

This study tested a new granule (small particle) form of three cystic fibrosis medicines in 34 healthy adults. The goal was to see how well the body absorbs the medicine and whether food changes that. This is an early-stage study to gather information, not a treatment for patient…

This study looked at how often depression and anxiety occur in teenagers with cystic fibrosis (CF) and their parents. Researchers asked 108 participants to fill out questionnaires during a regular clinic visit. The goal was to understand how mental health issues might affect trea…

This study looked at whether people with cystic fibrosis (CF) have natural protection against COVID-19. Researchers tested blood samples from 331 children and adults with CF in Europe over two years to see if they had antibodies from past infection or vaccination. The goal was to…

This study looked at whether a test using cells from the nose can predict if the drug Orkambi will improve lung function in people with cystic fibrosis. 91 participants with a specific genetic mutation (F508del) took Orkambi for up to 48 weeks. The goal was to see if the lab test…

This study looked at people with diseases that make it hard to clear mucus from the lungs, like cystic fibrosis and primary ciliary dyskinesia. Researchers did tests and genetic analysis to better understand why these patients get repeated lung infections and to find the genes re…

This study looked at how a cystic fibrosis drug combination (vanzacaftor/tezacaftor/deutivacaftor) affects the way the body processes a common cholesterol drug called rosuvastatin. 18 healthy adults took both medications, and researchers measured drug levels and checked for side …

This study aims to find risk factors for developing osteoporosis in people with cystic fibrosis. Researchers will take a blood sample and study monocytes, which can turn into osteoclasts—cells that break down bone. They will also test whether CFTR modulators affect these bone cel…

This study followed 79 infants with cystic fibrosis (CF) under 6 months old to see how their pancreas function changed during the first year of life. Researchers measured a stool enzyme called fecal elastase-1 to track pancreatic health. No treatment was given; the goal was simpl…

This study tested a smartphone app called MuCopilot that lets people with cystic fibrosis measure their lung function and exercise ability at home. 70 adults in France used the app to perform tests like coughing and walking, and researchers compared the results to standard clinic…

This study looked at whether a 6-minute walk test can help predict survival or the need for a lung transplant in adults with cystic fibrosis. Researchers followed 291 patients to see how far they could walk and how their oxygen levels changed. The goal is to find a simple way to …

This study followed 113 people with cystic fibrosis (CF) for two years to see how many developed COVID-19 antibodies from infection or vaccination. Researchers collected blood samples and health data to understand if CF patients have different immune responses or milder COVID-19 …

This study tracked 19 teens and young adults with cystic fibrosis to see how starting the drug ORKAMBI changed their anxiety, depression, quality of life, and how well they stuck to their treatments. Researchers compared those taking ORKAMBI to a similar group not on the drug. Th…

This study aims to find DNA markers in sputum (spit) that can predict how severe lung disease will become in people with cystic fibrosis. Researchers will collect sputum samples from 50 participants aged 12 to 30 over 18 months and analyze changes in DNA methylation. The goal is …

This study created new methods to measure how well people with cystic fibrosis digest and absorb fats, proteins, and sugars. Researchers tested these methods in 30 adults and children with and without cystic fibrosis. The goal is to improve nutrition and enzyme treatments for bet…

This small study tested a device called the Volara System to help collect sputum (mucus) from adults with cystic fibrosis who have trouble coughing it up. The goal was to see if this method works better for getting a sample that can be tested for infections. Twenty adults took pa…

This study looked at whether a simple, non-invasive breathing test called the lung clearance index can detect early signs of lung disease in young children (ages 3-6) with cystic fibrosis. The goal is to find lung problems sooner so treatment can start earlier. Researchers compar…

This study collected sputum (mucus coughed up from the lungs) from healthy volunteers and people with cystic fibrosis. Researchers tested whether certain sugar-like compounds could reduce the stickiness of the mucus and block harmful bacteria from binding to it. The goal was to b…

This study tested whether electrical impedance tomography (EIT), a non-invasive imaging method, can detect lung changes in children and young adults with cystic fibrosis (CF). Researchers compared EIT to CT scans to see if it could identify air trapping and other lung problems. T…