- completedPhase II
A two-part study to assess the effectiveness, safety, and blood levels of repeat doses of inhaled ETD001 in people with cystic fibrosis
This study looks at a new inhaled medicine called ETD001 for adults with cystic fibrosis. The treatment aims to loosen sticky lung mucus to help make breathing easier. Researchers want to see if the medicine is safe, well tolerated, and improves lung function when taken alongside usual daily care.
United Kingdom · England · Northern Ireland - completed
CF START: A national UK trial to determine whether taking an antibiotic (flucloxacillin) every day predisposes infants with cystic fibrosis (CF) to earlier infection with a bug, Pseudomonas aeruginosa, that is resistant to treatment
The CF START study looks at the best way to use antibiotics in babies with cystic fibrosis. It compares giving a daily antibiotic to prevent chest infections against only giving antibiotics when a baby shows signs of infection. The goal is to see which approach keeps babies healthiest.
United Kingdom · England - completedPhase IV
Real world health outcomes in people with cystic fibrosis after initiation of a new combination treatment
This study looks at how new combination medicines help people with cystic fibrosis in everyday life. Researchers across the UK and Ireland are following children and adults aged six and older for up to seven years to see how these treatments improve health and daily living over time.
United Kingdom · England · Northern Ireland - recruitingPhase I and Phase II (Integrated)- First administration to humans
A Phase 1/2, Multicenter Study Evaluating the Safety, Tolerability, and Biodistribution of RCT2100 with Single-Ascending Doses in Healthy Participants and Multiple-Ascending Doses and Proof-of-Concept in Participants with Cystic Fibrosis
This clinical trial tests a new medicine called RCT2100 for cystic fibrosis. Doctors want to check its safety and how the body handles it. The study involves both healthy volunteers and adults with cystic fibrosis to see if it causes side effects and if it can help improve lung function.
France · Netherlands - recruitingTherapeutic exploratory (Phase II)
A Phase IIb, Multicentre, Randomised, Double-Blind, Placebo-Controlled, Crossover Study to Evaluate the Efficacy and Safety of Dirocaftor/Posenacaftor/Nesolicaftor in Subjects with Cystic Fibrosis Aged 18 Years or Older (CHOICES)
This study is looking at new medications, Dirocaftor, Posenacaftor, and Nesolicaftor, for adults with Cystic Fibrosis. We want to see how well they improve breathing and if they are safe, comparing them to a dummy treatment.
France · Belgium · Spain - recruitingPhase I and Phase II (Integrated)- First administration to humans
A seamless Phase I/II trial with an initial open-label dose escalation part and a subsequent randomised, double-blind, placebo-controlled expansion part to evaluate the safety, tolerability, and efficacy of a single dose of BI 3720931, an inhaled lentiviral vector gene therapy, in adult people with cystic fibrosis who are ineligible for CFTR modulators (LenticlairTM 1)
This study is testing a new inhaled gene therapy, BI 3720931, for adults with cystic fibrosis who can't use other treatments. It will check if the treatment is safe, how well people tolerate it, and if it improves lung function (FEV1pp).
Italy · Spain · Netherlands - recruitingTherapeutic confirmatory (Phase III)
A Phase 3 Open-label Study Evaluating the Long term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor Triple Combination Therapy in Cystic Fibrosis Subjects 2 Years and Older
This study is looking at the long-term safety and effectiveness of a new three-in-one medicine called Kaftrio (also known as Elexacaftor/Tezacaftor/Ivacaftor) for people aged 2 and older with Cystic Fibrosis. Researchers want to see how well it works and if it has any side effects over time.
Germany - recruitingTherapeutic exploratory (Phase II)
A phase II trial to assess the activity and tolerability of Thymosin alpha 1 in Cystic Fibrosis Patients
This study is looking at a new treatment called Thymosin alpha 1 (brand name ZADAXIN®) for cystic fibrosis. It wants to see if this treatment can reduce inflammation in the lungs and if it's safe for patients. Researchers will check levels of inflammation markers in sputum and monitor overall health.
Italy - recruitingTherapeutic exploratory (Phase II)
A Phase 2b, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate Nebulized Bacteriophage Treatment in Outpatient Adult Cystic Fibrosis (CF) Subjects with Chronic Pseudomonas aeruginosa (PsA) Pulmonary Infection
This study looks at a new mist treatment for adults with Cystic Fibrosis (CF) who have ongoing lung infections caused by Pseudomonas aeruginosa bacteria. We want to see if this treatment, called BX004, can reduce the amount of bacteria in their lungs after 8 weeks.
Germany · Ireland · France - recruitingTherapeutic exploratory (Phase II)
A Phase 2, Open-label, Multiple Ascending-Dose Study to Evaluate the Safety, Tolerability and Efficacy of ARCT-032 in People with Cystic Fibrosis
This study is testing a new medicine, ARCT-032, for people with cystic fibrosis. Researchers want to see if it's safe, how well people tolerate it, and if it helps improve their health, especially their breathing, by giving different doses.
Poland · Greece - completed
Detection of respiratory exacerbations in adults with cystic fibrosis using artificial intelligence
This UK study looks at whether a smart phone app can spot chest flare-ups early in adults with cystic fibrosis. By tracking everyday health checks at home, the app uses artificial intelligence to warn people before symptoms worsen, helping them get treatment sooner and stay well.
United Kingdom · England · Northern Ireland - completed
CFHealthHub Data Observatory
This UK study tests an online system called CFHealthHub for people aged 16 and over with cystic fibrosis. By connecting to a special nebuliser, the website tracks when you take inhaled medicines. It offers helpful tools to build daily routines, stay healthier, and reduce emergency hospital visits.
United Kingdom · England · Wales - completedPhase IV
Can people with cystic fibrosis safely stop taking some of their nebulised treatments if they are established on the new modulator therapy, Kaftrio?
The CF STORM trial is looking at whether people with cystic fibrosis who take the medicine Kaftrio can safely stop their daily mucus-clearing nebulisers. Researchers want to see if reducing these time-consuming treatments keeps lung function steady and makes day-to-day life easier without harming overall health.
United Kingdom · England · Northern Ireland - recruitingTherapeutic confirmatory (Phase III)
A Phase 3, Open-label Study Evaluating the Pharmacokinetics, Safety, and Tolerability of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis Subjects 12 to Less Than 24 Months of Age
This study is testing a new combination medicine for babies and toddlers aged 12 to 24 months with Cystic Fibrosis. Researchers want to ensure the medicine is safe and well-tolerated, and how it moves through the body.
Germany · Netherlands · Denmark - recruitingTherapeutic exploratory (Phase II)
A Randomised, Double-Blind, Placebo Controlled, Two-Part Study to Evaluate the Efficacy, Safety, Tolerability and Pharmacokinetics of a Repeat Dose of Inhaled ETD001 in People with Cystic Fibrosis
This study is testing a new inhaled medicine, ETD001, for people with cystic fibrosis. It aims to see if it's safe, how well it works, and how the body handles it. Some people will get the medicine, others a dummy treatment.
Germany · France · Italy - recruitingHuman Pharmacology (Phase I)- Bioequivalence Study
An open label, balanced, randomized, single dose, two treatment, two period, two sequence, crossover, bioequivalence study comparing Ivacaftor film coated tablets 150 mg of Sun Pharmaceutical Industries Ltd, India with Kalydeco (Ivacaftor) 150 mg film-coated tablets, Marketing Authorisation Holder: Vertex Pharmaceuticals (Ireland) Limited Unit 49, Block F2, Northwood Court, Santry, Dublin 9, D09 T665, Ireland, in healthy adult, human subjects under fed condition.
This study is comparing a new version of Ivacaftor with the current Kalydeco medicine. It looks at how the body uses these medicines in healthy adults to see if they work the same way. This is an early-stage study, not testing a new condition treatment.
Romania - recruitingPhase I and Phase II (Integrated)- First administration to humans
A Phase 1/2 Dose-escalation Study Evaluating the Safety, Tolerability, and Efficacy of VX-522 in Subjects 18 Years of Age and Older With Cystic Fibrosis and a CFTR Genotype Not Responsive to CFTR Modulator Therapy
This study is testing a new inhaled medicine, VX-522, for adults with cystic fibrosis (CF) whose current treatments aren't working well. Researchers want to see if it's safe and helpful. It's an early-stage trial, meaning it's one of the first times this medicine is being tested in people.
Germany · Italy · Netherlands - recruiting
Trikafta Exercise Study in Cystic Fibrosis
This study looks at how a cystic fibrosis (CF) drug called Trikafta affects shortness of breath and exercise ability. Researchers want to see if Trikafta helps people with CF exercise more easily and feel less breathless, which could improve their overall health.
Canada - recruitingPHASE2
Xenon-129 and Inert Fluorinated Gas Lung MRI: Study of Healthy Volunteers and Participants With Pulmonary Disease
This study uses special MRI scans with safe gases to look at lungs in people with cystic fibrosis and asthma, and healthy volunteers. The aim is to see how well these new scanning methods work and what they can tell us about lung health.
Canada - recruitingNA
Streamlined Treatment of Pulmonary Exacerbations in Pediatrics
This study looks at two ways to treat lung flare-ups (exacerbations) in children with Cystic Fibrosis. It compares immediate antibiotics to a 'watch and wait' approach to see which is safer and works better long-term, and if we can use fewer antibiotics.
United States · Canada - recruiting
Development and Validation of a Disease Specific PROM to Assess Abdominal Involvement in Patients With CF (CFAbd-Score)
Researchers are creating a new questionnaire to help people with Cystic Fibrosis (CF) describe their tummy troubles. This tool, called the CFAbd-Score, aims to better understand how CF affects the stomach and gut, from children to adults, to improve care.
Germany - recruiting
Establishment of a Primary Epididymal Cell Model From Epididymal Samples to Study CFTR Gene Regulation
This study looks at cells from a part of the male reproductive system called the epididymis. Researchers want to understand how a specific gene, CFTR, works in these cells. This could help us learn more about male infertility and cystic fibrosis.
France - active not recruitingPHASE1, PHASE2
A Phase 1/2 Study of VX-522 in Participants With Cystic Fibrosis (CF)
This study is testing a new medicine, VX-522, for adults with cystic fibrosis (CF) whose current treatments aren't effective. It aims to see if the medicine is safe, well-tolerated, and helpful for improving their condition. This is an important step in finding new ways to manage CF.
United States · Australia · Belgium - active not recruitingNA
Determinants of Daily Physical Activity Level in Patients With Cystic Fibrosis - Pilot Study Protocol
This study looks at why people with Cystic Fibrosis (CF) are more or less active. Participants will wear a small device for a week to measure their activity and fill out a questionnaire about their feelings and surroundings. This helps us understand how to encourage more activity.
France - active not recruitingPHASE3
Evaluation of VX-121/Tezacaftor/Deutivacaftor in Cystic Fibrosis (CF) Participants 1 Through 11 Years of Age
This study is testing a new medicine, VX-121/TEZ/D-IVA, for children aged 1 to 11 with cystic fibrosis. We want to see how safe and effective it is, how the body uses it, and if it helps improve their health.
United States · Australia · Canada - recruiting
Study of the Prevalence of Active/Passive Smoking and Vaping Among Patients With Cystic Fibrosis
This study looks at how many adults with cystic fibrosis in Strasbourg, France, smoke or vape. It aims to understand current trends, especially since treatments and quality of life for people with CF have improved a lot. The findings will help create better ways to prevent smoking and vaping.
France - recruiting
Modulate-CF: Cystic Fibrosis Transmembrane Regulator (CFTR) Biomarker Study to Evaluate the Rescue of Mutant CFTR in Patients With Cystic Fibrosis Treated With CFTR-modulators
This study looks at how new cystic fibrosis (CF) medicines, called CFTR modulators, change important body markers. We're checking how these drugs affect people with CF in real life by measuring things like intestinal currents, nasal activity, and sweat levels.
Germany - recruitingNA
A Usability Validation of the MuCopilot Mobile Application, a Digital Tool for the Unsupervised Objective Assessment of Cystic Fibrosis
This study looks at a new smartphone app, MuCopilot, for people with Cystic Fibrosis (CF). It helps check lung health and exercise at home. We want to make sure the app is safe and easy to use for everyone.
France - active not recruitingPHASE3
Study to Evaluate Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) Long-term Safety and Efficacy in Subjects Without F508del
This study looks at a new treatment called ELX/TEZ/IVA for cystic fibrosis, specifically for people who don't have a common gene change (F508del). It's checking if the treatment is safe and works well over a long time.
Austria · Belgium · Canada - active not recruitingPHASE2
A Phase 2 Study Evaluating Safety and Tolerability of RCT2100 (CFTR mRNA) in Healthy Participants and in Participants With CF
This study is testing a new inhaled medicine, RCT2100, for cystic fibrosis. It will first check if it's safe for healthy people, then for people with CF, sometimes alongside other CF treatments. We aim to understand its safety and how the body handles it.
United States · France · Netherlands - enrolling by invitationPHASE3
Evaluation of Long-Term Safety and Efficacy of Vanzacaftor/Tezacaftor/Deutivacaftor in Cystic Fibrosis Participants 1 Year of Age and Older
This study is looking at a new medicine called VNZ/TEZ/D-IVA for people with cystic fibrosis, aged one year and older. It wants to see how safe and effective it is over a long time for those who have already been in a related study.
United States · Australia · Canada - active not recruitingPHASE3
A Study Evaluating the Long-term Safety and Efficacy of VX-121 Combination Therapy
This study is looking at a new combination medicine, VX-121/TEZ/D-IVA, for people with cystic fibrosis. We want to understand how safe and effective it is over a longer period, especially for those who have been in previous studies with this medication.
United States · Australia · Austria - recruiting
Identification of Dysglycemia With Continuous Glucose Monitoring to Assess Clinical Evolution in Cystic Fibrosis
This study looks at how a small device, called a continuous glucose monitor (CGM), can help people with cystic fibrosis (CF) keep an eye on their sugar levels. We want to see if these monitors can spot early sugar problems and predict future health changes more easily than current tests.
Canada · France - recruiting
Canadian Observational Study Evaluating the Long-term IMPACT of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Modulators on People With CF
This study in Canada looks at how well Trikafta and similar cystic fibrosis (CF) medicines work over time for people with CF. Researchers want to see if these treatments keep people healthier in the long run and help find the best care for each person.
Canada - recruiting
Circadian Rhythm Disorders in Children With Cystic Fibrosis Under CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) Modulators
This study looks at sleep patterns, called chronotypes, in children with cystic fibrosis (CF) who are taking new CFTR modulator medicines like Kaftrio-Kalydeco. We want to see if these medicines affect their sleep and daily rhythms, which can impact their overall health and how well they feel.
France - active not recruitingPHASE4
Hyperpolarized Imaging for New Treatments
This study looks at new ways to check how well new cystic fibrosis (CF) medicines work. It uses special MRI scans with a harmless gas to see lung changes earlier and better, aiming for more personalised care for people with CF.
United States · Canada - active not recruiting
Analysis of Remote Monitoring/Virtual Clinic Data in Adult Patients With Cystic Fibrosis (Project Breathe)
This study looks at how home monitoring helps adults with Cystic Fibrosis. It checks if using devices like lung function testers and heart rate monitors at home can spot lung infections earlier and how people find using this technology.
United Kingdom - active not recruitingNA
Microbial Biomarkers of EArly Pseudomonas Aeruginosa Colonization in CHildren With Cystic Fibrosis
This study looks for early signs in babies with cystic fibrosis (CF) that might predict if they will get a common lung infection called Pseudomonas aeruginosa later. We are checking stool samples for certain bacteria at 12 months to see if it links to infections at 3 years old.
France - recruiting
Oral Health Status of Cystic Fibrosis Patients. An Online Survey in Collaboration With the Vaincre la Mucoviscidose Patient Association.
This study is a survey for adults in France with Cystic Fibrosis (CF) to understand their oral health better. It looks at how CF affects teeth and gums, especially since people with CF are living longer. The goal is to improve dental care for individuals with CF.
France - active not recruitingPHASE4
Real World Clinical Outcomes With Novel Modulator Therapy Combinations in People With CF (RECOVER)
This study, called RECOVER, looks at how well the medicine Kaftrio works for people with cystic fibrosis (CF) in real life. It checks many things like breathing, tummy problems, and how people feel overall, for two years, starting with those aged 12 and over.
Ireland · United Kingdom - authorisedTherapeutic exploratory (Phase II)
Exploratory study to evaluate the safety and tolerability of tamoxifen citrate in the treatment of cystic fibrosis in patients without mutations currently eligible for therapy with CFTR modulator drugs Protocol Code: CRCFC-TAMOXI063
This study is looking at a medicine called TAMOXENE for cystic fibrosis in adults who can’t use other modern treatments. We want to see if it’s safe and if it causes any side effects. We'll also check if it helps with breathing and other CF symptoms over 24 weeks.
Italy - authorisedTherapeutic use (Phase IV)
A low-intervention prospective-retrospective study to evaluate the pharmacokinetics of elexacaftor/tezacaftor/ivacaftor combination in a Cystic Fibrosis population
This study looks at how a cystic fibrosis medicine called Kaftrio works in the body. Researchers want to understand how much of the medicine gets into the blood and how long it stays there, to help make sure patients are getting the most benefit.
Italy - active not recruitingPHASE1, PHASE2
A Long-term Study to Monitor the Health Status of People With Cystic Fibrosis Who Took Part in a Previous Study With BI 3720931 (Lenticlair™-ON)
This study looks at the long-term health of people with cystic fibrosis who previously took part in a trial for a medicine called BI 3720931. Participants won't receive new medicine in this study, but their health will be monitored for many years to see how they are doing.
France · Italy · Netherlands - authorisedTherapeutic exploratory (Phase II)
A Phase 2, Randomized, Double-blind, Controlled Study to Evaluate the Safety and Efficacy of VX‑828/Deutivacaftor With and Without Tezacaftor in Subjects Aged 18 Years and Older With Cystic Fibrosis
This study is looking at a new drug combination, VX-828/Deutivacaftor, to see if it's safe and helps adults with cystic fibrosis (CF). Researchers will check how well people respond and if there are any side effects compared to other treatments or placebos. It's an early-stage study to gather important information.
Spain · Sweden · Germany - active not recruitingNA
YOGA-CF (Yoga Outcomes Get Assessed in Cystic Fibrosis)
This study looks at how yoga affects adults with Cystic Fibrosis (CF). Researchers want to see if doing yoga for 12 weeks improves their quality of life compared to just receiving standard CF care. It's about understanding if yoga can make a positive difference.
United Kingdom - active not recruitingPHASE3
Evaluation of Long-term Safety and Efficacy of ELX/TEZ/IVA in Cystic Fibrosis Participants 12 Months of Age and Older
This study looks at the long-term safety and how well a medicine called ELX/TEZ/IVA works for people with cystic fibrosis, aged 12 months and older. It continues to check on participants who have already been taking the medicine in a previous study.
Australia · Canada · Denmark - active not recruiting
COVID-19 Antibody Responses In Cystic Fibrosis
This UK study looks at how people with Cystic Fibrosis (CF) respond to COVID-19, including how many have antibodies and what impact the virus has on their health. It will follow adults and children with CF for two years, comparing natural infection with vaccine responses.
United Kingdom