CYSTIC FIBROSIS

This early-phase trial is testing whether certain cystic fibrosis drugs, called CFTR modulators, can help people with rare gene mutations that are not currently approved for these medications. The study will enroll 20 participants aged 6 and older and measure changes in lung func…

This study looks at two ways to treat lung flare-ups in children with cystic fibrosis. One group starts antibiotics right away, while the other only takes them if symptoms don't improve. Researchers will track lung function over a year to see which approach works better. The goal…

This study looks at bone health in people with cystic fibrosis (CF) and tests a drug called denosumab to treat bone disease. Up to 100 participants, including both CF patients and healthy volunteers, will have bone scans and blood tests. A smaller group of 10 CF patients with bon…

This study is the first time the drug RCT2100 is being tested in humans. It aims to check the safety and tolerability of single and multiple doses in healthy adults and people with cystic fibrosis (CF). The trial will also see how the drug spreads in the body and gather early pro…

This study tests whether a weekly injection of dulaglutide, a GLP-1 drug used for diabetes, can improve early insulin release in adults with cystic fibrosis who have trouble processing sugar. About 30 participants will receive the drug or no drug in a crossover design. The goal i…

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 drug Trikafta for at least 6 months. The program combines dietary…

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 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 is testing a new blood test that can diagnose single-gene disorders in unborn babies using a sample from the mother. The test looks at fetal DNA found in the mother's blood, which is safer than traditional invasive methods that carry a small risk of miscarriage. Resear…

This study tests whether a smartphone app (AWARE) can accurately detect and monitor lung diseases like asthma, COPD, and cystic fibrosis by analyzing sound waves from the phone's speaker and microphone. Researchers will enroll 800 people aged 8-70, including healthy volunteers an…

This study is testing a special MRI scan that uses xenon gas to take detailed pictures of lung function. It includes 260 healthy volunteers and people with lung diseases like asthma, COPD, and cystic fibrosis. The goal is to see if this method can better detect and measure lung p…

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 tests tenapanor, a drug already approved for irritable bowel syndrome with constipation, as a treatment for constipation in people with cystic fibrosis. 25 participants will take a 50 mg tablet twice daily for 4 weeks and track their bowel movements and symptoms. The g…

This pilot 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. Participants will take the drug for two weeks alongside their usual laxatives. The goal is t…

This study aims to understand why many people with cystic fibrosis develop diabetes. Researchers will give two gut hormones, GLP-1 and GIP, to 45 adults with cystic fibrosis and measure how their pancreas releases insulin. The goal is to learn more about cystic fibrosis-related d…

This observational study looks at how CFTR modulator therapy affects the nose and sinuses in adults with cystic fibrosis. Researchers will compare bacterial communities, inflammation, sense of smell, and quality of life between patients on the therapy and those not on it. Partici…

This study is creating better ways to test exercise in children with cystic fibrosis, sickle cell disease, or long COVID. Current tests are designed for healthy athletes and may not be safe or useful for these kids. Researchers will track 240 children over 3-4 years, measuring ho…

This study looks at how much adults with cystic fibrosis move and sit during their daily lives. Researchers want to understand activity patterns, especially in those who are inactive and spend a lot of time sitting. The goal is to find patients who could benefit from personalized…

This study follows 200 people with cystic fibrosis, ages 6 and up, who are starting a new triple therapy drug (VTD) after having mental health or liver problems on a similar drug (ETI). Researchers will track how many need to stop or change VTD due to side effects, and how many h…

This study aims to find out how many adults with cystic fibrosis smoke or vape. Researchers will survey 100 patients at a French hospital to get current data. The goal is to improve tobacco prevention and understand how smoking or vaping might affect newer cystic fibrosis treatme…

This study looks at whether certain molecules can fix nonsense mutations in the CFTR gene, which cause cystic fibrosis. Researchers will take cells from the noses of 85 people with cystic fibrosis and test different molecules to see which ones can restore the function of the CFTR…

This observational study aims to understand how the CFTR gene is regulated in epididymal cells, which may help explain male infertility linked to cystic fibrosis. Researchers will collect leftover tissue samples from 20 men already scheduled for surgery. No extra procedures are n…

This study uses MRI scans to measure bone marrow fat and bone density in adolescents with cystic fibrosis compared to healthy teens. Researchers want to understand why bone problems occur in cystic fibrosis. The study involves 36 participants aged 13-20 and does not test any drug…

This study follows 80 children aged 2-8 with cystic fibrosis to see how highly effective CF drugs (Kalydeco or Trikafta) impact sinus disease and sense of smell. Researchers will use MRI scans, smell tests, and quality-of-life surveys over two years. The goal is to understand whe…

This study looks at new ways to measure lung changes in people with cystic fibrosis (CF) aged 12 to 21. Researchers will use a special MRI with a breathable gas called Xenon, along with a lung function test called LCI, to see how stopping or restarting airway clearance treatment …

This study collects blood, airway, and urine samples from 2,000 healthy volunteers and people with lung diseases like COPD and IPF. Researchers aim to establish normal ranges for various measurements and learn how gene patterns in airway cells change in disease. The goal is to be…

This study follows 170 adults with cystic fibrosis and chronic sinusitis to see if sinus surgery improves lung function and reduces hospital stays. Some participants will have surgery, while others will receive only medical treatment. The goal is to understand the real-world impa…

This study looks at how hormone levels change after a mixed meal in people with cystic fibrosis. Researchers want to find links to blood sugar problems common in this condition. About 61 adults aged 18-45 with cystic fibrosis will participate. No treatment is given; this is an ob…

This study aims to learn more about non-tuberculous mycobacterial (NTM) infections by observing up to 1,000 people over time. Researchers will collect medical history, blood samples, and genetic data to understand why some people get these infections and how the disease progresse…

This study aims to learn how noncirrhotic portal hypertension (NCPH) develops and changes over time in people who have it or are at risk. Researchers will follow 400 participants aged 12 and older for years, using blood tests, imaging, and liver pressure measurements. No treatmen…

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

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 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 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 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 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 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 …