Cystic fibrosis

This study tests a new medicine called ANG003 for people with cystic fibrosis who have trouble digesting food (exocrine pancreatic insufficiency). The medicine is a type of enzyme replacement therapy, similar to the current standard treatment Creon. About 113 participants will re…

This study tests an experimental drug called AZD0292 in 435 people aged 12 and older who have bronchiectasis and a long-term lung infection with Pseudomonas aeruginosa. The goal is to see if the drug can reduce the number of moderate-to-severe lung flare-ups compared to a placebo…

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 tracks 20 adults with cystic fibrosis who are starting Trikafta to see if it reduces shortness of breath and improves exercise performance over two years. Participants will do exercise tests and report their breathing symptoms. The goal is to understand how this medica…

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 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 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 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 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 phase 2 trial is testing whether metformin, a common diabetes drug, can improve airway function in 30 adults with cystic fibrosis-related diabetes who are already on CFTR modulator therapy. Researchers will measure changes in ion channel activity in nasal cells. The study ai…

This study tests a drug called dorzagliatin in 15 adults with cystic fibrosis who have trouble controlling their blood sugar. Participants take the drug or a placebo for 7 days each, in a crossover design. The goal is to see if dorzagliatin improves glucose tolerance and insulin …

This phase 1/2 trial tests a single inhaled dose of gene therapy called 4D-710 in 30 adults with cystic fibrosis who cannot take standard modulator drugs. The therapy delivers a working copy of the CFTR gene directly to lung cells. The main goal is to check safety and side effect…

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 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 aims to find effective treatments for people with cystic fibrosis who have rare gene mutations not covered by current drugs. Researchers will test FDA-approved CF drugs on nasal cells in the lab, then confirm the best option in a personalized N-of-1 trial. Up to 50 par…

This study tests whether a one-year program of remote exercise sessions and personalized diet advice can increase lean muscle mass in 100 adults with cystic fibrosis who are already taking the drug Trikafta. The program is designed to address new nutritional challenges, like weig…

This study is testing the safety of a drug called gallium nitrate in 40 adults with cystic fibrosis who have a difficult-to-treat lung infection called NTM. Participants will receive two 5-day cycles of the drug through an IV. The main goal is to see if the treatment is safe and …

This early-stage trial tests the safety of three experimental drugs (SION-451, SION-2222, and SION-109) given together to healthy adults aged 18–55. The study aims to see if the combinations are safe and how the body processes them. It is a first step toward possibly developing a…

This study is working on a new blood test for pregnant women that can check for serious genetic conditions like cystic fibrosis, spinal muscular atrophy, and sickle cell disease. Researchers will collect blood samples from 4,000 pregnant women who are at higher risk of passing on…

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 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 is testing a new 3D automated ultrasound system called ATUSA to see how well it can image suspicious breast lesions. Up to 800 women with suspicious findings on standard imaging will get an ATUSA scan before their biopsy. The goal is to collect images and compare them …

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 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 tests a smartphone program called NUDGE that acts like a health coach for teens and young adults (ages 13-25) with cystic fibrosis. Participants set activity goals, track progress, and get feedback through the app. The goal is to see if the app helps them stay active a…

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 tests whether upper and lower limb exercises can improve muscle strength, aerobic capacity, and lung function in children aged 6-18 with cystic fibrosis. Participants will do arm or leg cycling exercises. Researchers will measure changes in walking distance, lung funct…

This study tests whether tenapanor, a drug already approved for irritable bowel syndrome with constipation, can help people with cystic fibrosis who have constipation. Twenty-five adults will take a 50 mg tablet twice daily for 4 weeks and track their bowel movements and symptoms…

This study tests whether a self-help version of Acceptance and Commitment Therapy (ACT), delivered via smartphone, can reduce anxiety and depression in adults with cystic fibrosis. About 60 participants will use the toolkit and report their symptoms. The goal is to improve mental…

This pilot study tests a wellness program for adults with cystic fibrosis (CF) to see if it helps with fatigue, sleep, and physical activity. The program includes 8 coaching sessions on topics like balancing activity and rest, improving sleep, and managing stress. Researchers wil…

This study tests whether online group cognitive behavioral therapy (CBT) can reduce depression, anxiety, and other mental health symptoms in adults with cystic fibrosis and their caregivers. Participants will attend 13 sessions via Zoom and be compared to those receiving supporti…

This study is testing a smartphone app called OnTrackCF, designed to help adults with cystic fibrosis remember to take their CFTR modulator medication and track their symptoms. The app sends up to 3 daily support messages and shows users their adherence levels. Researchers want t…

This study tests a new MRI method that uses an inhaled gas to create detailed images of lung ventilation in people with cystic fibrosis. Researchers will track 30 adults with mild lung disease over a year, comparing the MRI results to standard breathing tests. The goal is to see …

This study looks at whether a Mediterranean diet can improve gut health in children with cystic fibrosis. Twenty kids aged 3 and older will follow this diet for 6 months. Researchers will check changes in gut bacteria and inflammation markers. The goal is to offer better diet adv…

This study looks for better ways to find harmful bacteria in the lungs of people with cystic fibrosis. Traditional sputum tests are becoming harder to use because newer CF treatments reduce mucus. Researchers will collect samples like saliva, blood, urine, and breath from 300 par…

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 explores a new type of MRI that uses an inhaled gas to create images of lung ventilation in children ages 6-17. Researchers will compare results between children with mild cystic fibrosis and healthy children to see if the technique is feasible and sensitive to early l…

This study looks at how mucus plugs in the lungs affect breathing in people with severe asthma. Researchers will use special MRI scans to see air flow in blocked areas. Only 5 adults with severe asthma will take part, and the goal is to better understand lung function, not to tes…

This study aims to learn more about non-tuberculous mycobacterial (NTM) infections, which cause repeated lung and other organ infections. Researchers will study up to 1,000 participants to understand the genetics, symptoms, and best treatments for these infections. The study also…

This study tracks 253 cystic fibrosis patients starting a new drug combination (elexacaftor/tezacaftor/ivacaftor). Researchers collect sputum, stool, and blood samples at the start, 6 months, and 1 year to see how the drug changes bacteria and inflammation in the lungs and gut. T…

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 looks at how the cystic fibrosis drug ETI affects the way the body handles antibiotics. Researchers want to see if people on ETI need lower antibiotic doses to treat lung infections, which could reduce harmful side effects like hearing loss or kidney damage. The study …

This study tests whether a low-added-sugar, high-fat diet can reduce diabetes risk in adults with cystic fibrosis (CF). Sixty participants will receive either a low-sugar or standard CF diet for 8 weeks. Researchers will measure changes in insulin production, body fat, and blood …

This study looks at how new cystic fibrosis drugs work in children ages 2 and older. Researchers want to find genetic clues that can tell us ahead of time who will respond well, so kids don't get unnecessary side effects or high costs from treatments that won't help them. About 1…

This study is observing 400 people aged 12 and older who have noncirrhotic portal hypertension (NCPH) or are at risk for it. NCPH is a liver condition that increases blood pressure in the liver's blood vessels without causing cirrhosis, and often has no early symptoms. Researcher…

This study collects blood, nasal, and intestinal cells from 500 people with cystic fibrosis caused by rare mutations. The cells are stored in a bank and shared with researchers to test potential new drugs in the lab. It is a first step toward finding treatments for those who cann…

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 study looks at how the amount of muscle and fat in the body affects glucose tolerance and lung function in people aged 16-30 with cystic fibrosis. Researchers will use MRI scans and glucose tests to track changes over two years. The goal is to use this information to improve…

This early-stage study is testing a new drug called VX-581 in 128 healthy volunteers to see if it is safe and how the body processes it. The study is double-blind and placebo-controlled, meaning some participants get the drug and some get a dummy pill. This is a first step toward…

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 is not testing a new drug or treatment. Instead, researchers want to make sure their lab tests and equipment give accurate and repeatable results when studying conditions like obesity, diabetes, heart disease, and bone loss. Up to 100 healthy volunteers and people with…

This study aims to see if analyzing the chemicals in a child's breath can help diagnose and monitor diseases like asthma, neurological disorders, and type 1 diabetes. Researchers will collect breath samples from up to 3,600 children and teens to find patterns linked to these cond…

This study looks at why people with cystic fibrosis often develop kidney disease. Researchers will test blood and urine samples for early signs of kidney damage, comparing CF patients with and without frequent hospital stays, as well as people without CF. The goal is to find bett…

This study looks at how linaclotide, a drug for constipation, affects fluid in the gut. Researchers will use MRI scans to see where the drug works in the small and large intestines. The goal is to learn more about its effects, which could help design future studies for conditions…

This study tests advanced lung imaging (MRI with xenon gas) and breathing tests to better understand lung function in people with cystic fibrosis. 60 participants will be followed over time. The goal is to see if these new methods can detect changes more precisely than standard t…

This study looks at how cystic fibrosis patients who take the drug combination Elexacaftor-Tezacaftor-Ivacaftor (ETI) breathe during exercise. Researchers will measure lung function while patients walk or step, aiming to find out how many have limited breathing capacity. The resu…

This study follows 170 adults with cystic fibrosis and chronic sinusitis to see if sinus surgery improves lung function and quality of life compared to medical treatment alone. Participants either have surgery or continue with standard care. Researchers will track changes in brea…

This Phase 1 trial tests the safety and how the body handles VX-272, an experimental drug for cystic fibrosis. About 128 healthy volunteers will receive single or multiple doses of the drug or a placebo. The study focuses on side effects and drug levels in the blood, not on treat…

This study looks at how cystic fibrosis affects children's breathing, muscle strength, bladder control, and daily life compared to healthy kids. Researchers will measure grip strength, lung function, and other health markers in 50 children aged 6-18. The goal is to identify probl…

This study watches 200 people with cystic fibrosis to learn how their bodies react to standard treatments for severe lung infections. Researchers track symptoms, lung function, and collect samples to understand why some people recover better than others. The goal is to use this k…

This study is working to create better exercise tests for 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…

This study is looking for proteins and other markers in exhaled breath that could help doctors monitor lung inflammation and infections in people with asthma or cystic fibrosis. Researchers will collect breath samples from 450 participants (including healthy volunteers) and compa…

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 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 follows 300 people with cystic fibrosis for one year to learn what triggers sudden worsening of symptoms, called exacerbations. Participants provide regular health data and samples, and some will also have extra clinic visits and home monitoring. The goal is to find ea…

This study is testing two new types of lung MRI that use special gases (xenon and perfluoropropane) to create detailed images of lung function. Researchers will compare these scans in 30 people with cystic fibrosis, asthma, and healthy volunteers to see if they can spot lung prob…

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 is gathering detailed information from 470 adults with chronic inflammatory lung diseases like asthma, COPD, and cystic fibrosis, as well as healthy volunteers. Researchers will analyze clinical, biological, and lifestyle factors to identify distinct disease subtypes. …

This study looks at how the cystic fibrosis drug combination ETI (elexacaftor-tezacaftor-ivacaftor) behaves in children aged 2 to 17. Researchers will measure drug levels in the blood and see how they relate to the drug's benefits and side effects, such as liver problems or mood …

This study looks at how often people with cystic fibrosis and diabetes develop complications like eye, kidney, and nerve problems. Researchers will also check blood pressure and cholesterol. The goal is to better understand these risks in a diverse group of 200 adolescents and ad…

This study is investigating whether germs called non-tuberculous mycobacteria (NTM) found in home water systems and dust can cause lung infections in people with cystic fibrosis. Researchers will collect samples from the homes of 120 CF patients—some with NTM infections and some …

This study follows 150 cystic fibrosis patients starting the drug combination Trikafta (ETI) to see how it affects their metabolism, DNA methylation, and gut bacteria. Researchers will measure blood fats, sugars, and immune markers, and link them to drug levels. The goal is to un…

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 study is surveying 100 adults with cystic fibrosis to find out how many smoke or vape. Many people assume that patients with cystic fibrosis don't smoke, but there is almost no data on this. The results will help doctors improve tobacco prevention and understand how smoking …

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 aims to find out how common acid reflux symptoms are in children with cystic fibrosis. Researchers will use a symptom questionnaire and, for some children, will stop their reflux medication to see if symptoms get worse. About 20 children aged 2 to 18 will take part.

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 looks at whether people with cystic fibrosis who are on highly effective modulator therapy can safely stop using nasal saline rinses and steroid sprays. Researchers will monitor sinus health, lung function, and quality of life in 64 adults. The goal is to see if these …

This study is testing whether wearable sensors that track things like heart rate and breathing can detect changes during a chest infection in adults with cystic fibrosis or bronchiectasis. Researchers will monitor 50 patients who are starting intravenous antibiotics for a lung in…

This study aims to better understand bronchiectasis, a chronic lung disease, in Chinese patients. Researchers will collect data from 1500 adults to identify different disease types and their underlying causes. The goal is to improve diagnosis and tailor treatments for each patien…

This study looks at how much physical activity and sitting time adults with cystic fibrosis have, especially those on the latest triple-combination drug. Researchers will ask 270 participants about their activity and use activity monitors on a smaller group. The goal is to unders…

This study collects blood, airway, and urine samples from 2,000 healthy volunteers and people with lung diseases like COPD and pulmonary fibrosis. Researchers will compare samples to establish normal ranges and learn how genes change in lung disease. The goal is to better underst…

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 …

This study follows 146 people with cystic fibrosis who recently became first-time parents. Researchers will track their lung function, mental health, and stress levels over five years using surveys and medical records. The goal is to understand how parenthood affects their health…

This study from the University of Florida is looking at how two genetic lung conditions—Alpha-1 antitrypsin deficiency and cystic fibrosis—affect immune cells called macrophages. Researchers will collect blood and measure lung function in 220 adults to see how well these cells wo…

This study follows 200 people with cystic fibrosis (ages 6 and up) who had mental health or liver issues while taking the drug elexacaftor/tezacaftor/ivacaftor (ETI) and had to stop or change it. They will now start a newer triple therapy called vanzacaftor/tezacaftor/deutivacaft…

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 60 women with cystic fibrosis (CF) who are planning a pregnancy, pregnant, or new parents, along with their babies. Researchers will track lung function, nutrition, mental health, and pregnancy outcomes, as well as infant health issues like liver function and l…

This study is for people with cystic fibrosis (CF) who do not take CFTR modulators. Researchers will collect health information and samples over 12 months from 400 participants aged 12 and older. The goal is to use this data to design better clinical trials and develop new treatm…

This study looks at whether adults with cystic fibrosis can collect sputum samples at home and mail them in for infection testing, compared to standard clinic collection. With 150 participants, researchers will compare how well home samples detect bacteria like Pseudomonas aerugi…

This study compares a continuous glucose monitor (CGM) worn on the skin to the standard oral glucose tolerance test (OGTT) for detecting diabetes in adults with cystic fibrosis. The OGTT is time-consuming and often skipped, so researchers want to see if the CGM is a simpler and e…