Cystic fibrosis is a genetic disease that causes thick mucus to build up in the lungs and digestive system, leading to lung infections and difficulty absorbing nutrients. About 40,000 people in the US have CF. CFTR modulators — drugs that fix the faulty protein causing CF — have transformed care over the past decade, with some people now living into their 60s and beyond.
What's actually going on in research
Trials are testing next-generation CFTR modulators that work for more mutations, including rare ones not covered by current drugs. Researchers are also pursuing gene therapy and mRNA treatments to address CF at the DNA level, anti-inflammatory drugs to reduce lung damage, and treatments for CF-related diabetes and liver disease. Some studies focus on restoring fertility and improving bone health in adults living longer with CF.
Next-generation modulators
Companies are testing CFTR modulators designed to work for people with rare mutations who don't respond to elexacaftor-tezacaftor-ivacaftor. Some aim to be more potent or require less frequent dosing than current drugs.
Gene and mRNA therapy
Several trials are testing one-time gene therapy delivered through inhalation to correct the CFTR gene in lung cells. Others use mRNA to produce working CFTR protein, potentially avoiding the need for daily pills.
Lung infection prevention
Studies are testing inhaled antibiotics, phage therapy that targets specific bacteria, and drugs that break up mucus to prevent the chronic infections that damage lungs. Some aim to replace or reduce the burden of daily airway clearance.
What to know before you search
Eligibility typically depends on specific CFTR mutations, lung function level, age, whether you're already taking modulators, and presence of certain infections like Burkholderia.
What types of trials are currently open
- Modulator trials — Testing new or improved CFTR modulator drugs, often in people with mutations that don't respond to current treatments or in young children.
- Gene therapy trials — Testing one-time treatments that deliver a working copy of the CFTR gene to lung cells, usually through inhalation.
- Infection prevention trials — Testing inhaled antibiotics, phage therapy, or mucus-thinning drugs to prevent or treat lung infections.
- Complication trials — Testing treatments for CF-related diabetes, liver disease, bone loss, or sinus disease.
- Long-term follow-up studies — Following people on CFTR modulators for years to track lung function, quality of life, and complications as they age.
Recently added Cystic Fibrosis trials
Share your medical data to help understand antibiotic dosing in cystic fibrosis
Cystic fibrosis (CF) is associated with major pharmacokinetic and pharmacodynamic alterations affecting antibiotic exposure, including changes in absorption, distribution, metabolism, and elimination. Historically, these alterations justified the use of higher antibiotic doses in CF patients in order to achieve therapeutic concentrations and improve pulmonary outcomes. The advent of highly effective CFTR modulators, particularly the triple combination elexacaftor/tezacaftor/ivacaftor (ETI), has substantially improved pulmonary function, nutritional status, inflammatory burden, and quality of life in patients with CF. ETI therapy also appears to modify respiratory microbiology and reduce the frequency of pulmonary exacerbations. These clinical and physiological improvements may alter antibiotic pharmacokinetics and pharmacodynamics in patients with CF, potentially making current high-dose antibiotic recommendations less appropriate for some patients. Since repeated exposure to high-dose antibiotics is associated with cumulative toxicities, particularly aminoglycoside-related ototoxicity and nephrotoxicity, reassessment of antibiotic dosing strategies is warranted. The PKCF study is a multicenter, prospective, observational, non-interventional study designed to characterize the pharmacokinetic profiles of intravenous antibiotics administered during pulmonary exacerbations in adolescents and adults with cystic fibrosis receiving ETI therapy.
Take an investigational therapy to help treat chronic airway disease
This is a phase III, single-arm, open-label, multicenter study to assess the safety of HSK31858 in non-cystic fibrosis bronchiectasis (NCFBE) participants.
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