CYSTIC FIBROSIS

This study tested a new tablet called brensocatib in 29 adults with cystic fibrosis. The goal was to see how the drug behaves in the body and if it is safe compared to a placebo over 4 weeks. Participants had stable lung function and continued their usual treatments.

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 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 if larg…

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 whether sildenafil, a drug often used for other conditions, can reduce inflammation in the lungs of people with cystic fibrosis (CF). Researchers measured changes in sputum and breath markers in 36 participants aged 12 and older with stable lung function. The go…

This study tested a new drug called CMTX-101 in 43 adults with cystic fibrosis who have a stubborn lung infection (pseudomonas). The drug is designed to break up the slimy shield bacteria build, so standard antibiotics can work better. The main goal was to see if single doses are…

This study tested a three-drug combination (dirocaftor/posenacaftor/nesolicaftor) in 41 adults with cystic fibrosis who have rare CFTR gene mutations not covered by existing treatments. Participants were selected based on lab tests using their own intestinal organoids to predict …

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

This study tested whether miglustat, a drug used for other conditions, can help restore salt and water balance in the lungs of adults with cystic fibrosis. Sixteen adults with a specific genetic mutation (F508del) received miglustat or a placebo in a crossover design. The main go…

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 see if the drug is safe and tolerable, and to measure its ef…

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

This study looked at 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 and continued treatment to monitor side effects and …

This early-stage study tested whether losartan, a blood pressure medication, could help clear mucus from the lungs of adults with cystic fibrosis who are not eligible for standard CFTR therapies. Thirteen participants took losartan for over 12 weeks. The goal was to see if it imp…

This study looked at 51 adults with cystic fibrosis to compare two IV antibiotics (tobramycin and colistin) used for lung infections. The goal was to see if a new, less frequent dosing schedule for colistin could reduce kidney damage while still fighting the infection effectively…

This pilot study tested whether adding a GLP-1 drug (semaglutide) to insulin helps overweight adults with cystic fibrosis-related diabetes lose weight and improve blood sugar control. Eight participants took the drug for 12 weeks. The study aimed to see if the drug is safe and to…

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 remains effective and safe outside of clinical trials, in everyday medical practice. They measured lung function, hospit…

This early-stage study tested an experimental drug called MRT5005, given as a mist through a nebulizer, in 42 adults with cystic fibrosis. The main goal was to check if the treatment is safe and tolerable. Researchers also looked at whether it could improve lung function. The stu…

This study looked at whether adding an inhaled sugar powder called Bronchitol (mannitol) can further improve mucus clearance from the lungs in 14 adults with cystic fibrosis who already take the triple-combination drug elexacaftor/tezacaftor/ivacaftor. Participants had moderate t…

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

This study followed 125 children aged 6-11 with cystic fibrosis (CF) who started taking a triple combination therapy (elexacaftor/tezacaftor/ivacaftor). Researchers measured changes in lung function, sweat chloride levels, and growth over 24 months to see how well the treatment w…

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 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 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 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 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 looked at 27 children aged 6 to 11 with cystic fibrosis to see if a treatment called CFTR modulators (KAFTRIO) improves their nose and sinus symptoms and quality of life. The researchers measured changes using a special questionnaire (SN-5 scale) before and one year af…

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 counseling. Researchers measured lung function, quality of life, and emotional well…

This study tested whether using a Fitbit fitness tracker helps adults with cystic fibrosis exercise more regularly and improve their exercise tolerance over one year. Forty adults with CF took part. The goal was to see if the tracker encourages more physical activity and leads to…

This study tested a new program called 'Improving Life with CF' at five cystic fibrosis (CF) centers. The program helps doctors regularly check for and treat common problems like pain or stress, and provides education for patients and families. Over 600 people with CF took part t…

This study tested a medical device called Simeox against a standard breathing technique (autogenic drainage) to see which works better for clearing mucus from the lungs in adults with cystic fibrosis. Thirty-one adults with stable cystic fibrosis took part. The researchers measur…

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 looked at whether adding relaxation sessions at home to usual medical and psychological care can help people with cystic fibrosis who have ongoing or frequent pain. Thirteen patients over age 10 took part. The goal was to see if this approach could reduce pain intensit…

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 via telehealth. The goal was to see if patients would join and stick with it, not to measure health i…

This study tested whether a home exercise program delivered through video calls could help children and teens with cystic fibrosis become more active. Ten participants aged 8 to 21 used an activity tracker and did supervised workouts from home. The goal was to see if this approac…

This study aimed to find DNA markers (called epigenetic biomarkers) in sputum that could predict how severe lung disease will become in people with cystic fibrosis. Researchers followed 50 participants aged 12 to 30 for 18 months, collecting sputum samples and measuring DNA methy…

This study aims to find risk factors for 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 how CFTR modulators affect these bone cells. The study i…

This study talked to 27 children (ages 6 and up) who have a parent with cystic fibrosis, a serious lung disease. Through interviews and small group discussions led by a psychologist, researchers aimed to understand the children's feelings, worries, and needs. The goal is to use t…

This study looked at how the antibiotic omadacycline behaves in the bodies of 9 adults with cystic fibrosis. Participants received the drug by IV and by mouth so researchers could measure peak levels, total exposure, and absorption. The goal was to gather information for better d…

This study looked at mucus samples from healthy people and those with cystic fibrosis (CF). Researchers tested whether certain sugar-like compounds could make CF mucus less sticky and disrupt harmful bacteria. The goal was to better understand how infections take hold in CF lungs…

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 compared cells from patients with and without chronic lung infections, and those on certain medications. The goal was to l…

This study looked at how bacteria travel through the nose, mouth, and lungs of infants with cystic fibrosis. Researchers collected swabs and mucus samples from 7 babies over their first year of life. The goal was to understand which germs are present and how they move, which coul…

This study compared ultra-low-dose CT scans and lung MRI to see how well they show lung damage in adults with cystic fibrosis. 185 adults took part, and researchers checked if MRI could give similar results to the standard low-dose CT. The goal is to find a safer way to monitor l…

This small study tested a device called the Volara System to help adults with cystic fibrosis produce mucus (sputum) samples during clinic visits. The goal was to find a better way to collect samples for lab testing in patients who have trouble coughing up mucus. Twenty adults to…

This study looked at how many people with cystic fibrosis (CF) have antibodies against the COVID-19 virus. Researchers followed 152 adults and children with CF for two years, testing their blood for antibodies and checking their health. The goal was to learn if CF patients are le…

This study looked at whether a test using cells from the nose can predict how well the drug Orkambi® will improve breathing in people with cystic fibrosis. 91 participants with a specific genetic mutation (F508del) took part. The goal was to see if the lab test could serve as a r…

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

This study looked at whether the 6-minute walk test can help predict survival or the need for a lung transplant in adults with cystic fibrosis. Researchers followed 291 patients and measured how far they could walk in 6 minutes, along with oxygen levels and quality of life. The g…

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

This study followed 58 adults with cystic fibrosis to see how their bodies respond to CFTR modulator treatments. Researchers collected blood, sputum, urine, and quality-of-life surveys before and after starting the drugs. The goal was to track changes in inflammation, lung bacter…

This study looked at whether a non-invasive breathing test called the lung clearance index (LCI) can detect early lung disease in young children with cystic fibrosis. Fifty-three children aged 3 to 6 years old took the test during their annual check-up. The goal was to see if LCI…

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 small group discussions and one-on-one interviews with 52 participants, including patients and their spouses. T…

This study followed 79 infants with cystic fibrosis under 6 months old to see how their pancreas function changed over 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 simply to un…

This study tested whether a voice journaling app called Kintsugi is practical and liked by teenagers with cystic fibrosis (CF). Twenty teens aged 14-18 used the app for three months. The goal was to see if they would use it regularly and find it helpful, and to measure any change…

This study looked at 72 adults with cystic fibrosis to see if chemical changes in DNA (called DNA methylation) help explain why lung disease gets worse at different rates. Researchers focused on people with a common CF mutation. The goal was to understand how environment and gene…

This study looked at why adults with cystic fibrosis (CF) may be less physically active, focusing on factors outside the lungs like nutrition, diabetes, bone health, and mood. Eighty adults with CF wore activity monitors for a week and completed questionnaires. The goal was to fi…

This early-stage study tested a new granule (powder) version of three cystic fibrosis drugs (vanzacaftor, tezacaftor, and deutivacaftor) in 34 healthy adults. The goal was to see how well the body absorbs the granule form, whether food changes its effects, and if different doses …

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

This study looked at sputum (mucus coughed up from the lungs) from 83 adults with cystic fibrosis who were hospitalized for a sudden worsening of their lung disease. Researchers used genetic testing to find which antibiotic-resistant bacteria were present and compared those resul…

This study looked at how starting the medication ORKAMBI changes anxiety, depression, quality of life, and treatment adherence in 19 adolescents and young adults (ages 12-20) with cystic fibrosis. Researchers compared those taking ORKAMBI to a similar group not on the drug, using…

This study aims to create better methods to measure how the gut digests and absorbs fats, proteins, and sugars in people with cystic fibrosis (CF). Poor digestion is a major cause of malnutrition in CF, leading to muscle weakness and lung problems. Researchers will test these met…

This study looked at whether electrical impedance tomography (EIT), a radiation-free imaging method, can detect lung damage in children and young adults with cystic fibrosis (CF). Researchers compared EIT results to CT scans to see if it can spot air trapping and lung changes. Th…

This study looked at whether people with cystic fibrosis (CF) have different immune responses to COVID-19. Researchers tested blood samples from 331 children and adults with CF in Europe over two years to see how many had COVID-19 antibodies. They also tracked symptoms, lung func…

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 tested a smartphone app called MuCopilot that lets people with cystic fibrosis do lung function and walking tests at home. 70 adults in France used the app and their results were compared to standard clinic tests. The goal was to see if the app provides accurate and re…

This study looked at whether people with cystic fibrosis (CF) have different rates of COVID-19 infection and antibody responses compared to the general population. Researchers followed 75 adults and children with CF across Europe for two years, testing their blood for SARS-CoV-2 …

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 56 children with cystic fibrosis, aged 6 to 18, to compare four non-invasive methods for measuring liver scarring (fibrosis). The methods tested were FibroTest, Fibroscan, ShearWave elastography, and magnetic resonance elastography. The goal was to see which …

This study looked at whether people with cystic fibrosis (CF) have different immune responses to COVID-19. Researchers tested blood samples from 113 CF patients in Europe over two years to check for antibodies from natural infection or vaccination. The goal was to understand how …

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 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 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 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…