CYSTIC FIBROSIS

This study followed 852 cystic fibrosis patients in France for one year to see how well the Orkambi drug combination works in everyday treatment. Researchers tracked whether patients continued taking the medication, monitored side effects, and measured lung function and overall h…

This study tested how well a three-drug combination (elexacaftor/tezacaftor/ivacaftor) works for people with cystic fibrosis. It specifically measured if the treatment could reduce daily coughing and increase physical activity in patients aged 12 and older. Researchers used weara…

This study followed cystic fibrosis patients aged 2 and older to see how safe and effective a three-drug combination therapy is over the long term. Participants had previously taken these medications in an earlier study and continued treatment to monitor their response. The resea…

This study aimed to find the best dose of the antibiotic ceftaroline for children and teens with cystic fibrosis who have a history of a specific hard-to-treat lung infection (MRSA). Researchers measured how the drug moved through the bodies of 8 young patients to see if standard…

This early-stage study tested a new drug, CMTX-101, designed to help standard inhaled antibiotics work better in adults with cystic fibrosis who have a chronic lung infection called Pseudomonas aeruginosa. The main goal was to check if a single dose given through an IV was safe a…

This study tested whether a three-drug combination (Dirocaftor/Posenacaftor/Nesolicaftor) could help adults with cystic fibrosis who have rare genetic mutations not covered by existing treatments. Researchers first used patients' own lab-grown intestinal tissue samples to predict…

This study tested a new inhaled medication called ETD001 for people with cystic fibrosis. The first part checked if the medication was safe to take for a week. The second part measured whether taking it twice daily for 28 days could improve how well the lungs work, compared to a …

This small pilot study tested if adding a weekly GLP-1 drug (semaglutide) to insulin therapy is safe and tolerable for overweight adults with cystic fibrosis-related diabetes (CFRD). The main goal was to gather initial safety data to plan a larger future trial. Researchers also l…

This study tested a three-drug combination therapy (elexacaftor/tezacaftor/ivacaftor) in very young children with cystic fibrosis, aged 12 to 24 months. The main goals were to see how the medicine behaves in the body and to check if it is safe and well-tolerated in this age group…

This early-stage study tested a new granule form of a cystic fibrosis drug combination in healthy adults. Researchers wanted to see how the body absorbs the granules, if taking them with food makes a difference, and if different doses work as expected. The main goal was to gather…

This early-stage study tested a new inhaled gene therapy called MRT5005 in 42 adults with cystic fibrosis. The main goal was to check if the treatment was safe and tolerable when breathed in through a nebulizer. Researchers also looked for early signs that it might improve lung f…

This study tested a two-week antibiotic treatment to clear a dangerous bacteria called MRSA from the lungs of people with cystic fibrosis. Thirty-seven participants aged 2 to 45 received oral antibiotics, a nasal ointment, and mouthwash. Researchers checked if the treatment succe…

This study tested a new medicine called BI 1291583 for adults with cystic fibrosis who also have a lung condition called bronchiectasis. For 12 weeks, participants took either the new tablet or a placebo (dummy pill) to see how safe it was and if it could reduce a harmful enzyme …

This study followed 57 French cystic fibrosis patients with a specific genetic mutation (G551D) who took the drug Ivacaftor for at least one year. Researchers wanted to see if the drug remained effective and safe in everyday use after it was officially approved. They tracked lung…

This study looked at whether adding an inhaled powder called Bronchitol could help clear sticky mucus from the lungs of adults with moderate-to-severe cystic fibrosis who were already taking a highly effective triple-drug therapy (E/T/I). Researchers wanted to see if the extra tr…

This early-phase trial tested the safety and initial effectiveness of a single-dose, intravenous therapy made from special viruses called bacteriophages. The goal was to see if these viruses could safely target and reduce levels of Pseudomonas aeruginosa, a harmful bacteria that …

This study tested a new oral drug called brensocatib in adults with cystic fibrosis. The main goals were to see how the drug moves through the body and to check its safety over 4 weeks. A small group of 29 participants took either the drug or a placebo (a pill with no medicine) o…

This study followed 507 people with cystic fibrosis to check the long-term safety and effectiveness of a three-drug combination therapy. It focused on patients with a specific genetic mutation (F508del) that causes most CF cases. Researchers monitored lung function, weight, quali…

This study tested the safety and effectiveness of a two-drug combination (VX-661 and ivacaftor) for people with cystic fibrosis who have two copies of the most common genetic mutation (F508del). Forty participants were randomly assigned to receive either the drug combination or a…

This study tested whether a drug called miglustat could help fix the faulty protein that causes cystic fibrosis in adults with a specific genetic type of the disease. Sixteen participants received either miglustat or a placebo in a crossover design. Researchers measured changes i…

This study tested a new imaging technique called electrical impedance tomography (EIT) to see if it can monitor lung changes in children and young adults with cystic fibrosis. Researchers compared EIT to standard CT scans to see if it could detect areas of trapped air and lung da…

This study tested a smartphone app called MuCopilot, which lets people with cystic fibrosis measure their cough, breathing, and walking ability at home. Researchers wanted to see if the app's results matched those from standard tests done in a clinic. The goal is to create a reli…

This study tested a new, gentle scanning method (called elastometry) to measure liver scarring (fibrosis) in children. The goal was to see if this scan could be as accurate as a standard liver biopsy, which is invasive and painful. If successful, this scan could help doctors moni…

This study tested two newer, lower-radiation scanning methods (an ultra-low-dose CT scan and a lung MRI) to see how well they could assess lung damage in adults with cystic fibrosis. Researchers compared these newer scans to the standard low-dose CT scan that is currently used. T…

This study aimed to find better, painless ways to detect liver scarring (fibrosis) in children with cystic fibrosis. Researchers compared four different non-invasive tests—two types of ultrasound scans, a special MRI, and a blood test—to see how well they could identify liver dam…

This study tested a 12-week exercise program delivered by video call for adults with cystic fibrosis. The goal was to help participants start and stick with regular exercise to improve their health. Researchers provided personalized workouts and coaching to see if this remote app…

This study tested whether a medical device called Simeox® helps clear mucus from the lungs of adults with cystic fibrosis better than a standard manual breathing technique. Researchers compared how well the two methods improved breathing function and reduced symptoms like shortne…

This study tested whether starting a structured exercise program combined with behavioral counseling during a hospital stay could help people with cystic fibrosis. The goal was to see if beginning during this time made it easier for patients to stick with exercise long-term to he…

This small pilot study tested whether a voice journaling app called Kintsugi is easy and acceptable for teenagers with cystic fibrosis (CF) to use. Researchers wanted to see if using the app for three months could help with feelings of anxiety and depression, which are common in …

This study tested a new support program designed to improve the quality of life for people with cystic fibrosis (CF) and their caregivers. The program involved regular check-ins to identify and address common problems like pain, stress, and emotional distress. It was implemented …

This study looked at whether CFTR modulator drugs, which are known to help lung function in cystic fibrosis, also improve sinus symptoms and quality of life in children aged 6 to 11. Researchers followed 27 children for a year, using a special questionnaire to track changes in th…

This study tested whether children with cystic fibrosis could safely follow a live-streamed exercise program from home. Ten young patients aged 8-21 participated in a six-week program using video calls and wearable monitors. The goal was to see if this convenient, home-based appr…

This study tested whether adding a relaxation technique called sophrology to standard pain treatment could help people with cystic fibrosis who have frequent or constant pain. Thirteen patients received the relaxation sessions at home along with their usual medications and psycho…

This study aimed to see if a laboratory test on patients' own nasal cells could predict how well they would respond to the cystic fibrosis drug Orkambi. Researchers tested 91 patients with a specific genetic form of cystic fibrosis who were starting Orkambi treatment. They compar…

This study aimed to understand how bacteria move and grow in the airways of infants with cystic fibrosis (CF). Researchers followed 7 babies with CF from birth, taking regular swab samples from their mouth, nose, and throat over two years. By analyzing these samples, they hoped t…

This study aimed to see if a simple 6-minute walk test could help doctors predict the future health of adults with cystic fibrosis. Researchers followed 291 patients for three years to see if their walking distance or oxygen levels during the test were linked to their chance of n…

This study aimed to understand what factors influence how physically active adults with cystic fibrosis are in their daily lives. Researchers tracked 80 stable adult patients for a week using an armband monitor to measure their steps and activity levels. They also collected detai…

This study explored what it's like for children to have a parent with cystic fibrosis (CF). Researchers interviewed 27 children aged 6 and older to understand their experiences, feelings, and needs. The goal was to gather information to create better support programs for these fa…

This study aimed to understand how people with cystic fibrosis (CF) respond to COVID-19. Researchers followed 75 children and adults with CF for two years, checking their blood for COVID-19 antibodies from infection or vaccination. The goal was to see how common infections were, …

This study aimed to better understand several rare genetic conditions that make it hard for the lungs to clear mucus, leading to frequent infections. Researchers examined 87 participants, including healthy adults and patients with these conditions, using various tests to look at …

This study aimed to understand how common depression and anxiety are in teenagers with cystic fibrosis (CF) and their parents. Researchers surveyed 108 participants during routine clinic visits to measure these feelings. The goal was to see if depression or anxiety are linked to …

This study aimed to understand why people with cystic fibrosis (CF) are at higher risk for fragile bones (osteoporosis). Researchers took blood samples from 25 adult CF patients to study specific cells that can break down bone. They also tested in the lab whether CFTR modulator d…

This study aimed to understand how people with cystic fibrosis (CF) respond to COVID-19. Researchers followed 152 children and adults with CF in Europe for two years, checking their blood for COVID-19 antibodies at regular clinic visits. The goal was to see how many got infected,…

This study aimed to listen to and understand the experiences, needs, and concerns of people with cystic fibrosis (CF) who are parents. Researchers held small group discussions and individual interviews with 52 CF patients and their partners. The goal was to gather their real-life…

This study looked at how starting the cystic fibrosis medication ORKAMBI affects the mental health and daily life of teenagers and young adults. Researchers followed 19 young people with CF for one year, comparing those taking ORKAMBI to those not taking it. They measured changes…

This study aimed to understand if children with cystic fibrosis (CF) have different muscle endurance compared to healthy children. Researchers measured the strength and staying power of breathing muscles, core (torso) muscles, and leg muscles in 48 children. The goal was to gathe…

This study aimed to understand how a specific type of white blood cell, called a neutrophil, behaves in people with cystic fibrosis (CF). Researchers compared blood samples from 47 CF patients to samples from healthy donors and patients with other inflammatory diseases. They anal…

This study aimed to create better tools for measuring how people with cystic fibrosis (CF) digest and absorb nutrients from food. Researchers tested 30 participants, including both adults and children with CF and healthy volunteers, using special tracking methods. The goal was to…

This study followed 331 people with cystic fibrosis in Europe for two years to understand their COVID-19 infection rates and immune responses. Researchers regularly tested blood samples for COVID-19 antibodies and tracked participants' health to see if infection affected their cy…

This study aimed to understand how bacteria live and behave in different parts of the lungs of people with late-stage cystic fibrosis (CF). Researchers examined lung tissue removed from 74 adult CF patients during transplant surgery. They mapped where different bacteria were loca…

This study aimed to understand how people with cystic fibrosis (CF) respond to COVID-19. Researchers followed 113 children and adults with CF in Europe for two years, checking their blood for COVID-19 antibodies at regular clinic visits. The goal was to see how many got infected,…

This study aimed to find new ways to thin the thick, sticky mucus in the lungs of people with cystic fibrosis (CF). Researchers collected mucus samples from 114 people, including healthy volunteers and those with CF, to test in the lab. They tested if special sugar-like compounds…

This study tested whether a new lab method could predict if people with rare cystic fibrosis mutations would benefit from the drug Trikafta. Researchers gave Trikafta to 42 patients with specific rare mutations not normally approved for the drug. They collected skin or blood samp…

This study aimed to understand how cystic fibrosis affects the pancreas in babies during their first year of life. Researchers observed 79 infants who were not taking certain CF-specific medications. They measured a substance in stool samples over time to see how well the pancrea…

This study aimed to find biological markers in sputum (mucus coughed up from the lungs) that could help predict how severe lung disease might become in people with cystic fibrosis. Researchers studied 50 participants, aged 12 to 30, who have cystic fibrosis. They collected sputum…

This study aimed to understand how common HPV infection and cervical cell changes are in women with cystic fibrosis. Researchers followed 85 women for two years, performing regular Pap smears and HPV tests. The goal was to gather data to improve gynecological care and screening g…