CYSTIC FIBROSIS

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 any improve…

This study tests a new inhaled medicine called RCT2100 for people with 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 multiple doses in people with cystic fibrosis to see if it is safe and …

This study tests an experimental mRNA medicine called ARCT-032 in 33 adults with cystic fibrosis who cannot take standard CFTR modulators. The goal is to see if the drug is safe and can improve lung function and quality of life. Participants receive multiple doses, and researcher…

This early-stage study tests a new investigational drug called VX-828, alone or combined with other CF medicines, in healthy adults and people with cystic fibrosis. The main goal is to check safety and how the drug moves through the body. About 255 participants will take part to …

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 tests a triple combination medicine (VX-121/tezacaftor/deutivacaftor) in children aged 1 to 11 with cystic fibrosis who have a specific gene change. The goal is to see if the drug is safe and how it works in young bodies. About 210 kids will take part, and the study is…

This study tests whether metformin, a standard diabetes medicine, 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 a…

This study tests a new drug called VX-522 in adults aged 18 and older with cystic fibrosis whose genetic mutations do not respond to current CFTR modulator therapies. The goal is to see if the drug is safe and can improve lung function. About 39 participants will receive the drug…

This study tests whether a structured diet and exercise program can help adults with cystic fibrosis increase their lean muscle mass. Participants are already taking Trikafta, a medication that improves lung function and weight but can lead to new issues like overweight. The prog…

This study tests whether certain CF drugs, already approved for some people with cystic fibrosis, can help those with less common CFTR 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 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 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 if a smartphone app can listen to your breathing and detect lung problems like asthma, COPD, or cystic fibrosis. Researchers will compare the app's results to standard breathing tests. If successful, this could make lung health checks easier and more accessible, …

This study aims to see if a blood test from the mother can accurately diagnose genetic disorders in the fetus, such as sickle cell disease or cystic fibrosis, as early as 9 weeks into pregnancy. The test uses fetal DNA found in the mother's blood, avoiding the miscarriage risk of…

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 with constipation, in 25 adults with cystic fibrosis who also have constipation. Participants take one 50 mg tablet twice daily for 4 weeks and fill out questionnaires and daily diaries to track bowe…

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 s…

This study is testing a new type of MRI that uses a special gas (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 this imaging method can b…

This study looks at how much adults with cystic fibrosis move and sit during their daily lives. Researchers want to understand which patients are inactive or spend too much time sitting, especially those taking newer medications. The goal is to find people who could benefit from …

This study follows about 200 people with cystic fibrosis, ages 6 and up, who are starting a new triple therapy (VTD) after experiencing mental health issues or liver injury from a previous drug (ETI). Researchers will closely monitor for any return of mental health symptoms or li…

This study tests two new types of lung MRI (using special gases) to see if they can better show lung problems in people with cystic fibrosis or asthma. About 30 people aged 8 and older, including healthy volunteers, will have scans to compare lung structure and function. The goal…

This study is collecting detailed information from 470 adults with chronic inflammatory lung diseases like asthma, COPD, and cystic fibrosis, as well as healthy volunteers. The goal is to identify different disease subtypes and shared features, which could lead to more personaliz…

This study collects blood, nasal, and intestinal cells from 500 people with cystic fibrosis (CF) who have rare gene mutations not covered by current therapies. The cells are stored and shared with researchers to test potential new drugs in the lab. Participants must be at least 1…

This study aims to find out how many adults with cystic fibrosis smoke or vape. Many people assume they don't, but there is little data. The results will help doctors improve prevention and understand how smoking or vaping might affect newer treatments like Kaftrio. About 100 adu…

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 and a breathing test called LCI to see what happens when patients stop and restart their usual airway clea…

This study is creating new, kid-friendly exercise tests for children with cystic fibrosis, sickle cell disease, or long-term effects of COVID-19. Current tests are designed for healthy athletes and don't work well for these children. Researchers will track 240 kids over 3-4 years…

This study looks at how estrogen and hormonal birth control affect bone health in adolescent and young adult women with cystic fibrosis (CF). It has two parts: an observational study that follows participants for a year, and a small sub-study where volunteers use estrogen skin pa…

This study looks at how well CFTR modulator drugs work in people with cystic fibrosis. Researchers will measure CFTR function using sweat chloride tests and other methods in 500 participants. The goal is to better understand the drugs' effects in real-world use.

This study compares a continuous glucose monitor (CGM) worn on the skin for 10 days to the standard 2-hour oral glucose test for detecting diabetes in people with cystic fibrosis. Researchers want to see if the CGM is easier and just as accurate for screening. The study involves …

This study watches how newer cystic fibrosis (CF) medicines impact 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 help with sinus and …

This observational 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 are on the therapy and those who are not. Participants pr…

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 learn why teens with CF may have weaker bones, which could help improv…

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

This study follows 146 new parents with cystic fibrosis to see how parenthood impacts their lung function and mental health over five years. Participants complete surveys about stress and depression, and researchers track their medical records. The goal is to understand how to be…

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 18-45 with cystic fibrosis will take part. No treatment is given; the goal is to learn more about t…

This study looks at whether people with cystic fibrosis can collect sputum (mucus) samples at home and mail them in to check for lung infections, instead of coming to the clinic. About 150 adults will provide both home and clinic samples so researchers can compare how well each m…

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 drug is being developed for cystic fibrosis, but this trial does not involve people with the disease. The goal is to gather safety informat…

This study looks at how genes play a role in lung diseases like cystic fibrosis, asthma, and pulmonary fibrosis. Researchers will analyze DNA from up to 3,500 people, including healthy volunteers, to find genetic differences linked to lung problems. A small optional part of the s…

This study aims to understand why many people with cystic fibrosis develop diabetes. Researchers will measure how gut hormones called incretins affect insulin release in 45 adults with cystic fibrosis and pancreatic insufficiency. The goal is to uncover the mechanisms behind cyst…

This observational study uses leftover tissue from planned surgeries to grow epididymal cells in the lab. Researchers want to see how the CFTR gene is organized and regulated in these cells, which may help explain male infertility in cystic fibrosis. About 20 men with or without …

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. The goal is to find inherited or immune system proble…

This study looks at whether sinus surgery improves lung function and quality of life in adults with cystic fibrosis who also have chronic sinusitis. About 170 participants will be followed, including some who have surgery and some who receive only medical treatment. The goal is t…

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 use regular check-ups, imaging, and tests to see how the disease chang…

This study looks at how well lungs work in adults with cystic fibrosis using a special MRI that uses an inhaled gas. Researchers will track 30 people over a year to see how their lungs change, especially during a flare-up. The goal is to better understand lung problems, not to tr…

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…