CYSTIC FIBROSIS

This study tests whether a structured diet and exercise program can help adults with cystic fibrosis increase their lean muscle mass. Participants must be 18 or older, have cystic fibrosis, and have been taking the medication Trikafta for at least 6 months. The program combines d…

This early-stage study tests a new experimental drug, VX-828, in healthy adults and people with cystic fibrosis (CF). The main goal is to check if the drug is safe and how the body processes it. Researchers will also test VX-828 together with other CF medicines. About 255 partici…

This study aims to find effective treatments for people with cystic fibrosis who have rare gene mutations not approved for current drugs. Researchers will test FDA-approved CFTR modulators on nasal cells in the lab, then confirm the best option in a personalized N-of-1 trial. The…

This study tests a triple combination medicine (VX-121/tezacaftor/deutivacaftor) in children aged 1 to 11 with cystic fibrosis who have a specific genetic mutation. The goal is to see how the drug moves through the body, how safe it is, and if it helps control the disease. About …

This study tests whether metformin, a standard diabetes medication, can improve airway cell function in adults with cystic fibrosis-related diabetes (CFRD). Participants must already be taking a CFTR modulator drug. The trial involves 30 people and measures changes in ion channel…

This early-stage study tests an inhaled gene therapy called KB407 in 20 adults with cystic fibrosis. The treatment uses a harmless virus to deliver a working copy of the CF gene directly to the lungs. The main goal is to check safety, but researchers will also measure if it impro…

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

This study tests a new drug called RCT2100 for people with cystic fibrosis. It is the first time this drug is being tested in humans. The study will check if the drug is safe and how it spreads in the body. It involves healthy volunteers and people with cystic fibrosis.

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 standard therapies. The goal is to see if the drug is safe and can improve lung function and quality of life. Participants receive multi…

This study tests whether certain drugs (CFTR modulators) that are already approved for some people with cystic fibrosis can help those with less common genetic mutations. About 20 participants aged 6 and older will take these drugs off-label to see if their lung function improves…

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 the drug denosumab for up to 5 years to see if it improves bone density. The goal is to better unde…

This study compares two ways of giving antibiotics to children with cystic fibrosis when they have a lung flare-up. About 430 kids aged 3 to 18 will be randomly assigned to one of two treatment plans. The goal is to see which approach better preserves lung function over a year an…

This study tests whether a continuous glucose monitor (CGM) worn on the skin for 10 days can detect diabetes and pre-diabetes in people with cystic fibrosis as accurately as the standard 2-hour oral glucose tolerance test (OGTT). The OGTT is time-consuming and often skipped, so a…

This study is testing a blood test that can check for genetic disorders in unborn babies without the risks of traditional invasive tests. The test looks at fetal DNA found in the mother's blood as early as 9 weeks into pregnancy. Researchers aim to make this safe, accurate diagno…

This study will test if a smartphone app called 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, to compare the app's results wit…

This study is testing a new, low-cost skin sensor that measures salt in sweat to diagnose cystic fibrosis. Researchers will compare the sensor's results to standard lab tests in 30 adults—some with CF and some healthy. The goal is to see if this wearable device can make diagnosis…

This study tests tenapanor, a drug already approved for irritable bowel syndrome, as a treatment for constipation in people with cystic fibrosis. About 25 adults will take one 50 mg tablet twice daily for 4 weeks. Researchers will track bowel movements and use questionnaires to s…

This study tests whether Maralixibat, a drug that increases bile acids in the colon, can improve stool consistency in 20 children with cystic fibrosis who have chronic constipation despite using standard laxatives. Each child takes Maralixibat for two weeks alongside their usual …

This observational study uses leftover tissue from planned surgeries to grow epididymal cells in the lab. Researchers aim to understand how the CFTR gene is organized and regulated in these cells, which may help explain male infertility linked to cystic fibrosis. About 20 men wit…

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, which could lead t…

This study looks at whether people with cystic fibrosis can collect sputum (mucus coughed up from the lungs) at home and mail it in to detect lung infections as accurately as samples taken in a clinic. The goal is to make infection monitoring easier, especially with the rise of t…

This study is gathering information from 470 adults with various inflammatory lung diseases, including asthma, COPD, and cystic fibrosis, as well as healthy volunteers. Researchers will analyze medical history, lung function, and other health data to identify different disease pa…

This early-stage study is testing a new medicine called VX-581 in 128 healthy volunteers to see if it is safe and how the body processes it. Participants will receive either the drug or a placebo, and researchers will monitor for side effects and measure drug levels in the blood.…

This study looks at how estrogen and hormonal birth control affect bone health in adolescent and young adult women with cystic fibrosis (CF). In one part, researchers observe bone changes over a year in women using different contraceptives. In another small part, a few women try …

This study aims to find out how many adults with cystic fibrosis smoke or vape, since there is very little data on this. Researchers will survey 100 adults at a French hospital to understand the current situation. The goal is to improve tobacco prevention and learn how smoking or…

This study looks at how a powerful cystic fibrosis treatment (CFTR modulator therapy) changes the bacteria and inflammation in the sinuses, and whether it improves smell and daily life. About 30 adults with CF will give nose samples, take smell tests, and fill out surveys. Resear…

This study looks at how much adults with cystic fibrosis move and sit each day. Researchers want to understand activity patterns, especially in those taking newer CF drugs. The goal is to find patients who are very inactive and may need extra support to stay healthy. Around 270 a…

This study follows 146 people with cystic fibrosis who recently became first-time parents to see how parenthood impacts their lung health and mental well-being. Participants complete surveys about stress, mood, and caregiving responsibilities over five years. The goal is to under…

This study looks at how well CFTR modulator drugs work in people with cystic fibrosis. Researchers will measure changes in sweat chloride, nasal potential, and intestinal current to see if the drugs improve cell function. About 500 patients already taking these drugs will partici…

This early-stage study tests whether VX-272 is safe and how the body processes it. About 128 healthy adults will receive either the drug or a placebo. The goal is to gather safety data before testing in people with cystic fibrosis.

This study looks at why many people with cystic fibrosis develop diabetes. Researchers will test how certain gut hormones (incretins) affect insulin production in the pancreas. The goal is to better understand cystic fibrosis related diabetes and find better ways to treat it. The…

This study collects blood, nasal, and intestinal cells from 500 people with cystic fibrosis caused by rare gene mutations. The cells are stored and shared with researchers to test potential new drugs in the lab. Participants must be at least 12 years old and have certain rare mut…

This study follows 200 people with cystic fibrosis, ages 6 and up, who are starting a new triple therapy drug after having mental health or liver problems with a similar medicine. Researchers will track how many stop the new drug due to side effects and whether mental health or l…

This study looks at whether sinus surgery improves lung function and quality of life in adults with cystic fibrosis who also have chronic sinusitis. Researchers will compare people who have surgery with those who only receive medical treatment. The goal is to understand the real-…

This study is testing a new MRI technique that uses a special gas called hyperpolarized xenon to take detailed pictures of lung function. It will include 260 healthy volunteers and people with various lung diseases like asthma, COPD, and cystic fibrosis. The goal is to see if thi…

This study tests two new types of lung MRI (using special gases) to see if they can better detect lung problems in people with cystic fibrosis and asthma compared to healthy volunteers. About 30 people aged 8 and older will have these scans to check how well the images show lung …

This study looks at how the amount of muscle and fat in the body affects health in people with cystic fibrosis (CF) aged 16 to 30. Researchers will measure body fat distribution, lung function, and blood sugar control in 60 people with CF and 30 healthy volunteers. The goal is to…

This study looks at how genes play a role in lung diseases like cystic fibrosis, asthma, and pulmonary fibrosis. Researchers will study the DNA of up to 3,500 people with and without lung disease to find genetic differences. The goal is to better understand what causes these dise…

This study is creating new ways to test exercise in children with cystic fibrosis, sickle cell disease, or long COVID. Current tests are designed for healthy athletes and don't work well for kids with these conditions. Researchers will track about 240 children over several years …

This study looks at new ways to measure lung changes in people with cystic fibrosis (CF). Researchers will use a special MRI gas called Xenon and a breathing test called LCI to see how stopping and restarting airway clearance treatment affects the lungs. About 60 people aged 12-2…

This study looks at how modern cystic fibrosis medicines affect sinus disease and sense of smell in children ages 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 help improve sinus and …

This study follows 900 people with bronchiectasis (damaged and infected airways) to learn why they get repeated lung infections. Researchers will collect blood, spit, and genetic samples from patients and their family members. No new treatments are tested; the goal is to find inh…

This study looks at how hormone levels change after a meal in people with cystic fibrosis. Researchers want to find links to blood sugar problems that often happen in this group. About 61 adults aged 18 to 45 with cystic fibrosis will take part. No treatment is given—only blood s…

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 typical warning signs. Researchers will track participants over many years using blood tests, ima…

This study looks at how well the lungs work in adults with cystic fibrosis (CF) using a special MRI that uses an inhaled gas. Over one year, 30 participants will have 3 to 5 visits to compare MRI images with standard breathing tests. The goal is to see if MRI can better detect ch…

This study aims to collect detailed health data from 400 people with cystic fibrosis who cannot take or do not take current CFTR modulator drugs. Researchers will track participants' lung function and quality of life over 12 months to better understand this specific patient group…