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 looked at whether children with cystic fibrosis (CF) who are taking CFTR modulator drugs and have regained normal pancreatic function can safely stop taking pancreatic enzyme replacement therapy (PERT). Seventeen children were followed for six months after stopping enz…

This study tested whether sildenafil (Revatio), a drug often used for erectile dysfunction, can reduce inflammation in the lungs of people with cystic fibrosis. Thirty-six participants took increasing doses over six weeks. Researchers measured changes in sputum elastase, a marker…

This study tested a single dose of CMTX-101, an antibody designed to break up bacterial biofilms, in 43 adults with cystic fibrosis who have chronic lung infections with Pseudomonas aeruginosa. The goal was to see if adding CMTX-101 to standard inhaled antibiotics is safe and tol…

This small study tested whether losartan, a blood pressure medicine, can help clear mucus from the lungs of people with cystic fibrosis who cannot take newer CFTR therapies. Thirteen adults with mild to moderate lung disease took losartan for over 12 weeks. Researchers measured c…

This study tested two common IV antibiotics, tobramycin and colistin, in 51 adults with cystic fibrosis to find the safest and most effective way to treat lung infections. Researchers compared a new, less frequent dosing schedule for colistin to standard care, aiming to reduce ki…

This study followed 125 children aged 6-11 with cystic fibrosis who were prescribed a triple therapy (elexacaftor, tezacaftor, and ivacaftor). Researchers measured changes in sweat chloride, lung function, and weight over 24 months to see how well the treatment works in real life…

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 tested an experimental pill called brensocatib in 29 adults with cystic fibrosis. The main goals were to see how the drug behaves in the body and whether it is safe compared to a placebo over 4 weeks. Participants took the pill once daily, and researchers measured drug…

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 previously taken the drug in an earlier study. Researchers tracked sid…

This study tested a two-week course of antibiotics (TMP/SMX or minocycline) given twice to people with cystic fibrosis who had early MRSA lung infections. The goal was to see if this approach could clear the bacteria better than standard care. The trial included 37 participants a…

This early-stage trial tested an inhaled drug called MRT5005 in 42 adults with cystic fibrosis. The goal was to check safety and see if it could improve lung function. Participants received either the drug or a placebo by nebulizer. The study is completed, and results will help d…

This pilot study tested the drug semaglutide (a GLP-1 receptor agonist, like Ozempic) in 8 overweight or obese adults with cystic fibrosis-related diabetes who were already using insulin. The goal was to see if adding semaglutide could safely help with weight loss and improve blo…

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 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 followed 852 cystic fibrosis patients aged 12 and older who started taking Orkambi outside of a clinical trial. Researchers tracked how many stopped treatment, why, and measured lung function and nutrition over one year. The goal was to see how the drug performs in rea…

This study tested an inhaled drug called ETD001 in 57 adults with cystic fibrosis. The trial had two parts: one to check safety over 7 days, and another to see if the drug improves lung function over 28 days. Participants took the drug or a placebo twice daily. The goal is to fin…

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

This study tested whether miglustat, a drug that blocks certain sugar processing, can improve salt movement in the noses of adults with cystic fibrosis who have the F508del gene mutation. Sixteen participants took miglustat for two weeks and a placebo for two weeks, with a washou…

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 check how the drug moves through the body, its safety, and any side effects. The trial has finished, and results will help dete…

This study tested a combination drug (elexacaftor/tezacaftor/ivacaftor) in 82 people with cystic fibrosis aged 12 and older. Researchers used wearable devices to measure how much the drug reduced coughing and increased physical activity over 12 weeks. The goal was to see if the t…

This phase 3 study tested the long-term safety and effectiveness of the drug combination elexacaftor/tezacaftor/ivacaftor (Trikafta) in 507 people with cystic fibrosis who have the F508del mutation. Participants had already completed a previous study and continued taking the medi…

This study tested a combination of two oral 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 drugs are safe and can improve lung function over 12 weeks. Participants were randomly assigned to…

This study tested the drug Trikafta in 42 people with cystic fibrosis who have rare genetic mutations not currently approved for this treatment. Participants took Trikafta for about four weeks, and researchers measured lung function and sweat chloride levels. They also used skin …

This study tested a new way to diagnose cystic fibrosis in unborn babies using a simple blood sample from the mother. Instead of using a needle to take fluid from around the baby, researchers used advanced DNA sequencing to read the baby's genes from tiny bits of fetal DNA floati…

This study looked at whether a simple saliva test could help doctors quickly assess the current health status of people with cystic fibrosis. Researchers measured markers of inflammation in saliva from 273 participants aged 6 and older. The goal was to develop a point-of-care dia…

This study compared two imaging methods—ultra-low-dose CT and lung MRI—for assessing lung damage in adults with cystic fibrosis. Researchers wanted to see if MRI could provide similar information to standard CT but without radiation. The study involved 185 participants and used t…

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 wearing a Fitbit activity tracker can help adults with cystic fibrosis exercise more regularly and improve their exercise tolerance over one year. Forty participants were enrolled and followed for 12 months. The goal was to see if a simple wearable devic…

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 a device called Simeox, which helps clear mucus from the lungs, against a standard manual breathing technique (autogenic drainage) in 31 adults with cystic fibrosis. The goal was to see which method better improves lung function and eases symptoms like cough and…

This study tested a new ventilator mode called NAVA for people with chronic obstructive pulmonary disease (COPD) who need a breathing machine. NAVA uses the patient's own diaphragm signals to control the machine, aiming to improve comfort and reduce time on the ventilator. The st…

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 tested whether starting a structured exercise program combined with behavioral counseling during a hospital stay for cystic fibrosis (CF) is feasible. Three adults with CF who were hospitalized for a lung flare-up took part. The goal was to see if this approach could h…

This study tested whether sophrology, a relaxation method, could help people with cystic fibrosis who have chronic pain. Thirteen patients received sophrology sessions at home in addition to their usual care. The goal was to see if this approach could reduce pain intensity and th…

This study tested whether a voice journaling app called Kintsugi is easy and acceptable for teens with cystic fibrosis to use. Twenty teens aged 14-18 with a history of anxiety or depression tried the app for three months. The goal was to see if it could help improve their emotio…

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 looked at 27 children aged 6 to 11 with cystic fibrosis who were starting treatment with CFTR modulators (Kaftrio). The goal was to see if the drug improves nose and sinus symptoms and quality of life after one year. Researchers used a special questionnaire called SN-5…

This small study tested whether a telerehabilitation program could help children and teens with cystic fibrosis exercise more at home. Ten participants used an online exercise program while wearing activity trackers. The goal was to see if this approach could improve their daily …

This study explores whether analyzing the chemicals in exhaled breath can detect early changes in children with cystic fibrosis who start taking a medication called Kaftrio. Researchers will collect breath samples from 50 children under 12 before and after they begin treatment, l…

This completed study looked at people with genetic conditions that affect how the lungs clear mucus, such as cystic fibrosis and primary ciliary dyskinesia. Researchers examined 87 participants, including healthy volunteers, to understand why these patients get repeated lung infe…

This study investigates whether tiny molecules in the blood, called miRNAs, can act as markers of lung disease in people with cystic fibrosis. Researchers will compare miRNA patterns between cystic fibrosis patients and healthy volunteers, and also between patients with mild vers…

This completed study followed 5 people with cystic fibrosis over two years to track COVID-19 antibodies from infection or vaccination. Researchers collected blood samples every six months to see how antibodies develop and last. The goal was to understand if people with CF have di…

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 collected leftover tissue, fluid, and bacteria from 400 cystic fibrosis patients after their routine medical tests. Researchers will use these samples to study lung diseases and explore new treatments. No genetic testing was done on the samples.

This completed study looked at how health literacy (understanding health information) relates to physical activity, anxiety, depression, and how well adults with cystic fibrosis follow their airway clearance routines. Researchers enrolled 62 adults aged 18-65 and used questionnai…

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 small pilot study tested whether a breathing device called the Volara System could help adults with cystic fibrosis produce sputum (mucus from the lungs) for lab testing. The study included 20 people who had not been able to produce a sputum sample in the past year. Particip…

This completed study followed 360 adults and children with chronic respiratory and other medical conditions to see how their immune systems responded to COVID-19 vaccines over 18 months. Researchers measured antibody, T cell, and B cell levels before vaccination and every 3 month…

This study followed 29 adults with cystic fibrosis who were hospitalized for a lung infection. Researchers collected blood, sputum, and urine samples to study how the body's immune response and bacteria affect recovery. The goal is to understand why some patients' lung function d…

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 sputum from 83 adults with cystic fibrosis during lung flare-ups to find genetic markers of antibiotic-resistant bacteria. Researchers compared these markers to standard lab tests and tracked how well patients responded to treatment. The goal is to develop fa…

This study looked at whether a blood test can measure beta cell death in people with cystic fibrosis, which may help predict diabetes. About 40 participants with cystic fibrosis and healthy controls gave blood samples to check for a specific DNA marker. The goal was to understand…

This study looked at 72 adults with cystic fibrosis to see if chemical changes in DNA, called 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; instead, rese…

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. Researchers tested 53 children aged 3 to 6 years and compared the results with CT scans and other lung function t…

This completed Phase 1 study tested how a cystic fibrosis drug combo (vanzacaftor/tezacaftor/deutivacaftor) affects the body's processing of the statin rosuvastatin. 18 healthy adults took both medications to check for interactions and safety. The goal was to see if the drug comb…

This study aims to find DNA markers (epigenetic biomarkers) in sputum 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 methylat…

This study looked at how the lungs move chloride, sodium, and bicarbonate in 42 patients with idiopathic bronchiectasis (widened airways of unknown cause). Researchers measured electrical signals in the airway lining during bronchoscopy and took nasal and bronchial samples. The g…

This completed study looked at how common depression and anxiety are in teenagers with cystic fibrosis and their parents. Researchers used questionnaires during routine clinic visits to screen for symptoms. The goal was to better understand mental health needs in this group.

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 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 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 bacteria travel through the nose, mouth, and lungs in infants with cystic fibrosis. Researchers collected swabs and mucus samples from 7 babies over their first year of life. By analyzing the bacterial communities at each site, they hope to understand how…

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 block bacterial and fungal proteins from binding to mucus, and whether these compounds could make t…

This study looked at whether people with cystic fibrosis (CF) have COVID-19 antibodies from infection or vaccination. Researchers followed 152 adults and children with CF for two years, taking blood samples every six months. They aimed to understand how many had antibodies, how l…

This study followed 113 people with cystic fibrosis (CF) over two years to see how many developed antibodies against COVID-19, either from infection or vaccination. Researchers collected blood samples and health data to understand if CF patients have a lower infection rate and ho…

This study compared muscle endurance in 48 children with cystic fibrosis (CF) and healthy children aged 6-18. Researchers measured core, respiratory, and leg muscle endurance to see if CF affects these abilities. The goal was to gather knowledge about physical differences, not to…

This completed study followed 79 infants with cystic fibrosis during their first year of life to see how well their pancreas works over time. Researchers measured a substance called fecal elastase-1 in stool samples to track pancreatic function. The goal was to learn more about t…

This study tested a smartphone app called MuCopilot that helps people with cystic fibrosis measure their lung function and exercise ability at home. 70 adults in France used the app to perform tests like speaking into the phone and timed walks, and researchers compared those resu…

This study looks at whether a simple 6-minute walk test can help predict how long cystic fibrosis patients will survive without needing a lung transplant. Researchers followed 291 adults with advanced cystic fibrosis, measuring how far they walked and their oxygen levels. The goa…

This study looked at how many girls and young women aged 11 to 20 with chronic diseases (like diabetes or immune conditions) got the HPV vaccine, compared to those without chronic illness. Researchers reviewed records of 223 participants from a hospital in France. The goal was to…

This study compared four non-invasive methods—FibroTest, Fibroscan, ShearWave Elastography, and Magnetic Resonance Elastography—to measure liver fibrosis in 56 children with cystic fibrosis aged 6-18. The goal was to see which test best detects early liver damage, helping doctors…

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 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 looks at why some adults with cystic fibrosis are more physically active than others, focusing on factors outside the lungs like nutrition, diabetes, bone health, and mood. Eighty stable adults will wear an activity monitor for a week and complete questionnaires. The g…

This study looked at what cystic fibrosis (CF) patients and their spouses think and feel about becoming parents. Researchers held small group discussions and one-on-one interviews led by a psychologist to gather their views. The goal was to identify common themes and needs, so be…

This study followed 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. T…

This study tested whether electrical impedance tomography (EIT), a non-invasive imaging technique, 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 identify air trapping and other lung cha…

This study examined lungs removed from 74 people with cystic fibrosis during transplant. Researchers looked at how bacteria and chemicals differ across various parts of the lung. The goal is to better understand why infections persist and how to target treatments more precisely.

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 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 absorption, and if different doses work as expec…

This study looked at how common human papillomavirus (HPV) infection and abnormal cervical cells are in women with cystic fibrosis (CF). Researchers tested 85 women using Pap smears and HPV DNA tests. The goal was to understand infection rates and how long the virus lasts, so doc…

This study looked at blood neutrophils (a type of immune cell) in 47 adults with cystic fibrosis. Researchers compared these cells between patients and healthy donors, and also examined how chronic infection, CFTR modulator drugs, and lung flare-ups affect neutrophil behavior. Th…

This study followed 230 people with cystic fibrosis to understand why some develop diabetes and why it sometimes reverses. Researchers tracked genetics, lung health, infections, diet, and physical activity over a year. The goal is to improve how diabetes is screened and treated i…