CYSTIC FIBROSIS

This study tested a new medicine called BI 1291583 in 22 adults with cystic fibrosis bronchiectasis, a lung condition that causes coughing and infections. Participants took one tablet daily for 12 weeks, and the study checked if the medicine was safe and well-tolerated. The trial…

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

This study looked at how a triple combination therapy (elexacaftor, tezacaftor, and ivacaftor) affects children aged 6 to 11 with cystic fibrosis who have a specific genetic mutation. Researchers measured changes in sweat chloride, lung function, and growth over 24 months. The go…

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

This study tested a streamlined treatment plan for early MRSA (a hard-to-treat bacterial infection) in 37 people with cystic fibrosis. The goal was to see if the treatment could clear the infection faster and reduce lung flare-ups. While the approach aims to control the infection…

This study tests a new inhaled medicine called ETD001 in 57 adults with cystic fibrosis. The goal is to see if it is safe and can improve lung function. Participants take the drug twice a day for up to 28 days, and their breathing and health are closely monitored.

This study tested a triple-combination drug (elexacaftor/tezacaftor/ivacaftor) in 70 toddlers aged 12 to 24 months with cystic fibrosis. The goal was to see how the drug moves through the body, whether it is safe, and if it helps reduce sweat chloride levels. Participants had at …

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

This early-stage study tested an inhaled drug called MRT5005 in 42 adults with cystic fibrosis. The main goal was to check if the treatment is safe and tolerable. Researchers also looked at how it affected lung function. The study was completed, but results are not yet public.

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

This study looked at the long-term safety of the triple-combination drug Trikafta (elexacaftor/tezacaftor/ivacaftor) in 71 children with cystic fibrosis who were 2 years or older. All participants had already completed a previous study with the same drug. Researchers tracked side…

This study tested a new medicine called CMTX-101, given as a one-time IV dose alongside standard inhaled antibiotics, in 43 adults with cystic fibrosis who have a chronic lung infection with Pseudomonas aeruginosa. The main goal was to see if the combination is safe and how the b…

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 completed a previous study and continued treatment to monitor side effects and lung fun…

This study tested a three-drug combination (dirocaftor/posenacaftor/nesolicaftor) in 41 adults with cystic fibrosis who have rare genetic mutations not covered by current treatments. Participants were chosen based on lab-grown mini-organs (organoids) that predicted they would res…

This study looked at how well the drug Ivacaftor works and how safe it is for people with cystic fibrosis who have a specific genetic change (G551D mutation) in everyday life, not just in a controlled trial. Researchers followed 57 French patients aged 6 and older for one year af…

This study looked at whether adding an inhaled medication called Bronchitol (mannitol) can further improve mucus clearance in people with cystic fibrosis who have moderate to severe lung disease and are already taking a triple-combination drug (elexacaftor/tezacaftor/ivacaftor). …

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 who have the most common genetic mutation (F508del). Sixteen participants received miglustat and a placebo at different times…

This study tested a new tablet called brensocatib in 29 adults with cystic fibrosis to see how safe it is and how the body processes it. Participants took the drug or a placebo once daily for 4 weeks. The goal was to measure drug levels in the blood and check for side effects, no…

This study looked at whether a non-invasive breathing test called the lung clearance index (LCI) can find early signs of lung disease in young children with cystic fibrosis. 53 children aged 3 to 6 took the test along with standard lung scans and function tests. The goal was to s…

This study tested whether a noninvasive device called FibroScan can accurately measure liver scarring (fibrosis) in children, potentially replacing the need for a liver biopsy. Researchers compared FibroScan results with biopsy results in 264 children aged 6 months to 18 years wi…

This study tested whether a home-based exercise program, guided remotely by a therapist, could help children and teens with cystic fibrosis become more active and improve their fitness. Ten participants aged 8 to 21 used a video chat system to exercise at home. The goal was to se…

This study tested a medical device called Simeox against a standard manual breathing technique (autogenic drainage) in 31 adults with stable cystic fibrosis. The goal was to see which method better clears mucus from the lungs and improves breathing. Researchers measured lung func…

This study tested a new program called 'Improving Life with CF' at five cystic fibrosis (CF) centers. The program helps doctors regularly check in on patients' symptoms and quality of life, and provides guides for managing common problems. Over 600 people with CF took part to see…

This study tested a program designed 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 aimed to improve emotional well-being, quality of life, and lung function.…

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 took part while hospitalized for a lung flare-up. The goal was to see if this approach could…

This study looked at whether using a Fitbit fitness tracker helps adults with cystic fibrosis exercise more regularly and improve their exercise tolerance over one year. Forty participants wore the device and their activity and lung function were tracked. The goal was to see if s…

This study tested whether a video-based exercise program is practical and acceptable for adults with cystic fibrosis after a flare-up. Nine clinically stable patients participated. The goal was to see if patients would join and stick with the program, not to measure health outcom…

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 overall quality of life. The researchers measured symptoms before and one year after starting the drug using a spec…

This small study tested whether losartan, a common blood pressure medication, could help clear mucus from the lungs of adults with cystic fibrosis who cannot take standard CFTR therapies. Thirteen participants took losartan for over 12 weeks. The goal was to see if it improved lu…

This early-stage study tested how a new granule (powder) form of three cystic fibrosis drugs (vanzacaftor, tezacaftor, and deutivacaftor) is absorbed by the body. It involved 34 healthy adults and looked at the effects of food and different doses. The goal was to gather informati…

This study tested whether a safe, radiation-free imaging method called electrical impedance tomography (EIT) can detect lung damage in children and young adults with cystic fibrosis (CF). Researchers compared EIT results to CT scans and checked if EIT could track treatment respon…

This study followed 75 people with cystic fibrosis (CF) for two years to see how many developed COVID-19 antibodies from infection or vaccination. Researchers wanted to know if CF patients are naturally protected or just avoiding the virus. The study collected blood samples and h…

This study looked at how a cystic fibrosis medicine (vanzacaftor/tezacaftor/deutivacaftor) changes the way the body processes a common cholesterol drug (rosuvastatin). 18 healthy adults took both drugs, and researchers measured drug levels in the blood. The goal was to check for …

This study looked at how bacteria develop in the nose, mouth, and lungs of infants with cystic fibrosis during their first year. Researchers collected swabs and mucus samples from 7 babies to track bacterial growth over time. The goal was to understand how germs move through the …

This study talked to 27 children (ages 6 and up) who have a parent with cystic fibrosis. Through interviews and small group discussions led by a psychologist, researchers aimed to understand their feelings, worries, and needs. The goal is to use this information to create better …

This study tested whether a voice journaling app called Kintsugi is easy and acceptable for teens with cystic fibrosis (CF) to use. Twenty adolescents aged 14-18 with CF and a history of anxiety or depression symptoms participated. The goal was to see how often they used the app …

This study aimed to find biological markers in spit (sputum) that could predict how severe lung disease becomes in people with cystic fibrosis. Researchers followed 50 participants aged 12 to 30 over 18 months, collecting spit samples and measuring DNA changes. The goal was to de…

This study aimed to find risk factors for osteoporosis in people with cystic fibrosis. Researchers took blood samples to study monocytes, which can turn into osteoclasts—cells that break down bone. They also tested how CFTR modulators affect these bone cells. The study included 2…

This study looked at sputum (phlegm) from 83 adults with cystic fibrosis who were hospitalized for a sudden worsening of lung symptoms. Researchers checked the bacteria in the sputum for genes that make them resistant to antibiotics. The goal was to see if these gene patterns cou…

This study collected sputum (mucus coughed up from the lungs) from 114 people with cystic fibrosis and healthy volunteers. Researchers tested whether certain sugar-like compounds could break down the thick mucus that causes breathing problems in cystic fibrosis. The goal was to u…

This study looked at 29 adults with cystic fibrosis who were hospitalized for a sudden worsening of lung symptoms. Researchers collected blood, sputum, and urine samples to study how infection and inflammation affect recovery of lung function. The goal was to find patterns that c…

This study followed 58 adults with cystic fibrosis who were starting or switching to newer CFTR modulator drugs as part of their regular care. Researchers collected blood, sputum, urine, and quality-of-life surveys before and after treatment to see how inflammation, lung bacteria…

This study looked at how often women with cystic fibrosis carry the HPV virus, which can lead to cervical cancer. Researchers followed 85 women for up to 24 months, testing for HPV and checking for abnormal cervical cells. The goal was to understand infection rates and help docto…

This study followed 79 infants with cystic fibrosis under 6 months old to see how their pancreas function changed during the first year of life. Researchers measured a digestive enzyme called fecal elastase-1 over time. The goal was to understand the natural course of pancreatic …

This small study tested a device called the Volara System to help adults with cystic fibrosis produce sputum (lung mucus) for testing when they cannot cough it up on their own. Twenty participants used the device during a clinic visit. The goal was to see if this method could suc…

This study compared two types of imaging—ultra-low-dose CT 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 way to …

This study looked at how well adults with cystic fibrosis understand health information and how that connects to their physical activity, mood, and use of breathing treatments. Researchers measured health literacy, anxiety, depression, and activity levels in 62 adults. The goal w…

This study tested a smartphone app called MuCopilot that helps people with cystic fibrosis measure their lung function and exercise ability at home. Seventy adults in France used the app to perform voice tests, walking tests, and answer questions about their symptoms. The goal wa…

This study looked at how bacteria and their environment differ in various parts of the lungs of people with cystic fibrosis. Researchers examined lung tissue from 74 patients who had a lung transplant. They hope this information will lead to new ideas for treatments that target t…

This study involved 8 children and teens with cystic fibrosis who had a history of MRSA infection and were already receiving the antibiotic ceftaroline. Researchers measured drug levels in the blood at different times to see how well the current dosing worked. The goal was to gat…

This study looked at blood neutrophils (a type of immune cell) in 47 adults with cystic fibrosis. Researchers wanted to see how these cells behave differently in people with chronic infections or on certain treatments. The goal was to learn more about the disease, not to test a n…

This study looked at what influences daily physical activity in 80 adults with cystic fibrosis, focusing on factors outside the lungs like nutrition, diabetes, bone health, and mood. Participants wore an activity monitor for a week and completed questionnaires. The goal was to id…

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 looked at whether people with cystic fibrosis (CF) have COVID-19 antibodies in their blood over two years. Researchers wanted to see if CF patients get infected less often or have milder symptoms. About 331 children and adults with CF from Europe gave blood samples and…

This study looked at how the antibiotic omadacycline moves through the bodies of 9 adults with cystic fibrosis. Participants received the drug by mouth and through an IV to measure levels in the blood. The goal was to understand dosing, not to treat the disease.

This study looked at how often depression and anxiety occur in teenagers with cystic fibrosis (ages 14-17) and their parents. Researchers used simple screening questionnaires during regular clinic visits to measure mental health symptoms. The goal was to find out how common these…

This study aims to create new methods to measure how well 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 worse health. The researchers will test thes…

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. Researchers collected nasal cells from 91 participants before they started Orkambi® and measured how well the drug fixed the c…

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 why …

This study talked to 52 adults with cystic fibrosis (and some of their spouses) to learn what they think and feel about becoming parents. Through small group discussions and one-on-one interviews led by a psychologist, researchers collected their hopes, worries, and needs. The go…

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 …