Cystic fibrosis treatment was transformed by CFTR modulators — especially Trikafta, which works for about 90 percent of patients and has dramatically improved lung function and life expectancy. Research is now focused on the remaining 10 percent and on preventing complications even with effective modulators.
What's actually going on in research
Trials are testing next-generation CFTR modulators for rare mutations, gene therapy and mRNA therapies that bypass the underlying gene problem, new antibiotics for resistant lung infections, treatments for CF-related diabetes and liver disease, and approaches to preserve fertility and pregnancy outcomes.
Modulators for rare mutations
About 10 percent of people with CF have mutations that do not respond to current modulators. New drugs and combinations are aimed specifically at these patients.
Gene and mRNA therapies
Inhaled mRNA and gene-editing approaches deliver instructions for working CFTR protein directly to lung cells. They could help patients regardless of mutation type.
Beyond the lungs
With longer life expectancy, CF-related diabetes, liver disease, and fertility are growing focuses. Trials are testing treatments for these complications.
What to know before you search
Eligibility often depends on specific CFTR mutations, age, lung function (FEV1), and current modulator use.
What types of trials are currently open
- Treatment trials — Testing CFTR modulators and other drugs to improve lung function and reduce CF complications.
- Gene and mRNA therapy trials — Testing inhaled or systemic therapies that deliver working CFTR instructions to cells.
- Antibiotic trials — Testing new antibiotics and inhaled therapies for resistant lung infections.
- Complication trials — Testing treatments for CF-related diabetes, liver disease, and bone health.
- Observational studies — Following people with CF to understand long-term outcomes with modulators.
Recently added Cystic Fibrosis trials
Proof of Principle Study for an Efficacy Trial of Linaclotide for Cystic Fibrosis
Linaclotide is a medicine used to treat constipation and irritable bowel syndrome with constipation (IBS-C). It works by acting on the surface of the gut lining, where it increases the movement of salt and water into the bowel. This softens stools, makes them easier to pass, and can also reduce gut pain One advantage of linaclotide is that, unlike some natural substances in the gut, it is stable and can act throughout the intestine. Studies in animals show that it has the strongest effect in the upper small intestine, but it may act in other parts of the bowel as well. In people, however, it is not yet clear whether linaclotide mainly works in the small intestine or in the large intestine (colon). Knowing this is important, because it could help the investigators understand whether linaclotide might also be useful in other conditions, such as cystic fibrosis, where the gut does not handle fluid properly. Linaclotide is taken as a capsule, but less than 1% is absorbed into the bloodstream. Instead, it stays in the gut, where it is broken down into smaller active parts. This means both the small intestine and colon may be exposed to its effects. Until now, it has been hard to study this because traditional methods only measure one part of the gut at a time. A team at the University of Nottingham has developed MRI scanning methods that can safely and non-invasively measure water content in the small intestine and colon. The aim of this pilot study is to use MRI in healthy volunteers to see exactly where linaclotide acts. This knowledge will help optimise future studies in conditions such as cystic fibrosis.
Exercises' Effect on Muscle Strength, Aerobic Capacity and Respiratory Functions in Cystic Fibrosis
This research aims to evaluate the effects of upper and lower limb exercises on muscle strength and pulmonary function in children diagnosed with CF. While aerobic training is a known component of CF management, resistance training focused on specific limb groups has gained attention for its additional benefits. Upper limb exercises may aid respiratory muscle endurance and thoracic mobility, enhancing pulmonary mechanics. In contrast, lower limb exercises (such as cycling or squats) are associated with improved oxygen consumption (VO₂ peak), enhanced mobility, and greater lower body strength. This randomized clinical trial will be conducted using a non-probability convenience sampling technique. The study will take place at the pediatric cystic fibrosis centers of Gulab Devi Chest Hospital and The Children's Hospital, Lahore. The targeted population includes children aged 6 to 18 years who have been diagnosed with cystic fibrosis and referred to the physiotherapy department. The study duration will be ten months following the approval of the synopsis. Eligible participants will be children aged between 6 and 18 years, clinically diagnosed with cystic fibrosis, currently stable with no history of hospitalization or significant lung infection in the past month. They should be physically able to participate in exercise and capable of following instructions, with informed consent obtained from their parents or guardians. Children will be excluded if they suffer from other severe lung diseases, cardiovascular or orthopedic conditions that restrict exercise, recent surgery or hospitalization within the last month, or if they are unable to understand instructions. Those dependent on oxygen therapy or ventilator support at all times will also be excluded. The primary outcome measures will include lung function assessed by spirometry (FVC, FEV1), sputum production recorded through a sputum diary, and aerobic capacity measured by the Incremental Shuttle Walk Test (ISWT). Muscle strength will be evaluated for both upper and lower limbs, using a handheld dynamometer for the upper limbs and the Sit-to-Stand Test for the lower limb
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