CYSTIC FIBROSIS

This study tests whether a structured diet and exercise program can help adults with cystic fibrosis increase their lean muscle mass. All participants are already taking the drug Trikafta (elexacaftor-tezacaftor-ivacaftor). The program combines dietary advice with adapted physica…

This early-stage study tests a new gene therapy called KB407, given as a mist through inhalation, in 20 adults with cystic fibrosis. The goal is to see if it is safe and can improve lung function by delivering a working copy of the CF gene to lung cells. Participants will receive…

This study tests whether certain drugs, already approved for some people with cystic fibrosis (CF), can help those with less common CF gene mutations. About 20 participants aged 6 and older will try these off-label medications to see if their lung function improves. The goal is t…

This study tests an experimental mRNA medicine called ARCT-032 in 33 adults with cystic fibrosis who cannot take or do not benefit from current CFTR modulators. The goal is to see if the drug is safe and can improve lung function and quality of life. Participants will receive mul…

This study tests an experimental drug called SPL84 in adults with cystic fibrosis who have a specific genetic mutation (3849+10kb C->T). The drug is inhaled weekly for 9 to 12 weeks. Researchers want to see if it is safe and if it can improve lung function. Some participants will…

This study tests whether metformin, a standard diabetes medicine, can improve airway cell function in adults with cystic fibrosis-related diabetes (CFRD). About 30 participants who are already taking CF modulator drugs will take metformin and have their nasal cells tested. The go…

This study tests whether a once-weekly injection of dulaglutide (a GLP-1 agonist) can improve early insulin release and blood sugar control in 30 adults with cystic fibrosis who have pancreatic insufficiency and abnormal glucose tolerance. Participants will receive the drug or no…

This study looks at bone health in up to 100 people with and without cystic fibrosis (CF) using scans and blood tests. A smaller group of CF patients with bone disease will receive denosumab, a drug that helps strengthen bones, for up to 5 years. The goal is to see if this treatm…

This study tests a new inhaled medicine called RCT2100 for cystic fibrosis. It is the first time this drug is being tested in humans. The study will first check safety in healthy adults, then test different doses in people with cystic fibrosis to see if it helps control the disea…

This study tests a new, low-cost skin sensor that measures salt in sweat to diagnose cystic fibrosis. Researchers will compare the device to the standard lab test in 30 adults—some with CF and some healthy. The goal is to see if the sensor is accurate enough for routine use.

This study is testing a new, safer blood test for pregnant women to diagnose genetic disorders like sickle cell disease and cystic fibrosis in their unborn babies. The test uses a small sample of the mother's blood, avoiding the miscarriage risk of traditional invasive procedures…

This study tests whether a smartphone app (AWARE) can accurately detect and monitor lung diseases like asthma, COPD, and cystic fibrosis by analyzing breathing sounds. Researchers will enroll 800 people aged 8-70, including healthy volunteers and those with airway conditions. The…

This study tests whether Maralixibat, a drug that increases bile acids in the gut, can improve stool consistency in 20 children with cystic fibrosis who have chronic constipation. Participants take the drug for two weeks alongside their usual laxatives. The goal is to see if it h…

This study tests tenapanor, a drug already approved for irritable bowel syndrome, to see if it can help with constipation in people with cystic fibrosis. About 25 adults will take one pill twice a day for 4 weeks and track their bowel movements and symptoms. The goal is to improv…

This study will count how many adults with cystic fibrosis smoke or vape. Researchers want to understand the real situation so they can improve prevention and see how smoking affects newer treatments. About 100 adults from one French center will take part.

This study compares a continuous glucose monitor (CGM) worn on the skin for 10 days to the standard 2-hour oral glucose tolerance test (OGTT) for detecting diabetes and early glucose problems in people with cystic fibrosis. The goal is to see if the CGM is easier and just as accu…

This study looks at how genes (the instructions in your cells) might cause or influence lung diseases like cystic fibrosis, asthma, and pulmonary fibrosis. Researchers will compare the genes of up to 3,500 people with and without lung disease. The goal is to better understand the…

This study watches 500 people with cystic fibrosis who are already taking CFTR modulator medicines. Researchers measure sweat chloride, nasal electrical activity, and rectal tissue responses to see how well the drugs improve CFTR protein function. The goal is to better understand…

This study looks at how newer cystic fibrosis medicines (called CFTR modulators) affect sinus disease and sense of smell in children aged 2 to 8. Researchers will use MRI scans, smell tests, and quality-of-life surveys over two years. The goal is to understand if these treatments…

This study looks at new ways to measure lung changes in people with cystic fibrosis (CF) when they stop and restart their usual airway clearance treatments. Researchers will use special MRI scans with a breathable gas called Xenon and a breathing test called LCI to see how the lu…

This study follows 900 people with bronchiectasis—a condition where airways are damaged and prone to infection—to learn why they get sick repeatedly. Researchers will collect medical history, lung function tests, and blood, urine, and sputum samples from patients and their family…

This study collects blood, nasal, and intestinal cells from 500 people with cystic fibrosis (CF) who have rare gene mutations. These cells are stored and shared with researchers to test potential new drugs in the lab. The goal is to find treatments that work for people whose rare…

This study looks at why many people with cystic fibrosis develop diabetes. Researchers will test how natural hormones called incretins affect insulin production in the pancreas. About 45 adults with cystic fibrosis and healthy volunteers will take part. The goal is to better unde…

This study is testing a new type of MRI that uses a special gas (xenon) to take detailed pictures of lung function. It includes healthy volunteers and people with various lung diseases like asthma or COPD. The goal is to see if this imaging method can help doctors better understa…

This study aims to see if a special MRI that uses an inhaled gas can better measure lung function in adults with mild cystic fibrosis. Researchers will compare MRI results to standard breathing tests over a year, and also track how the lungs change during a flare-up. The study in…

This study tests two new types of MRI scans that use special gases to take pictures of the lungs. Researchers want to see if these scans can find differences in lung function between people with cystic fibrosis, asthma, and healthy volunteers. The study involves 30 people aged 8 …

This early-stage study is testing the safety of a new drug called VX-272 in 128 healthy volunteers. Researchers want to see how the body handles the drug and if it causes any side effects. This is a first step before testing the drug in people with cystic fibrosis.

This study looks at whether sinus surgery improves lung function and reduces hospital stays for adults with cystic fibrosis who also have chronic sinusitis. About 170 people will take part, some having surgery and others getting only medical treatment. Researchers will track brea…

This study follows 200 people with cystic fibrosis, ages 6 and up, who are starting a new triple therapy drug (VTD) after experiencing mental health problems or liver injury from a previous drug (ETI). Researchers will track how many stop or change VTD due to mental health sympto…

This study looks at whether people with cystic fibrosis can collect sputum (mucus) samples at home and mail them in to detect lung infections as accurately as samples taken in a clinic. With newer treatments reducing mucus production, diagnosing infections has become harder. The …

This study is looking at how body fat and muscle affect the health of teens and young adults with cystic fibrosis (CF), ages 16 to 30. Researchers will measure body composition using MRI and track changes in lung function and blood sugar control over two years. The goal is to use…

This study is creating new ways to test how children with cystic fibrosis, sickle cell disease, or long COVID respond to exercise. Current tests are designed for healthy athletes and don't work well for kids with chronic conditions. Researchers will track 240 children over 3-4 ye…

This study looks at how hormones change after a meal in people with cystic fibrosis. Researchers want to find links to blood sugar issues common in this group. About 61 adults aged 18 to 45 with cystic fibrosis will participate. No treatment is given; it is an observation-only st…

This study measures how much adults with cystic fibrosis move and sit each day. Researchers want to understand activity patterns, especially in people taking newer medications. The goal is to identify those who are inactive and at higher health risk, so they can offer personalize…

This study looks at how a cystic fibrosis therapy (CFTR modulator) affects the nose and sinuses. Researchers will compare bacteria, inflammation, sense of smell, and quality of life between adults with CF who take the therapy and those who do not. About 30 participants will provi…

This study follows 400 people aged 12 and older who have or are at risk for noncirrhotic portal hypertension (NCPH), a liver condition that raises blood pressure in the liver without scarring. Researchers will monitor participants with regular check-ups, scans, and tests over man…

This study looks at bone health in teenagers with cystic fibrosis (CF) compared to healthy teens. Researchers will use MRI scans to measure bone marrow fat and other tests to check bone density. The goal is to understand why teens with CF may have weaker bones and how factors lik…

This study collects leftover epididymal tissue from men already scheduled for surgery to learn how the CFTR gene is regulated. Researchers aim to better understand male infertility linked to cystic fibrosis and related conditions. No extra procedures or health impacts are involve…

This study looks at how estrogen and other hormones affect bone development in teenage and young adult women with cystic fibrosis (CF). It has two parts: an observational study that tracks bone health in women using different types of birth control, and a small feasibility study …