Cystic fibrosis is a genetic disease affecting about 40,000 people in the U.S., caused by mutations in the CFTR gene that makes mucus thick and sticky. Since 2012, CFTR modulator drugs have transformed treatment for many people with CF, improving lung function and lifespan. About 90% of people with CF now have at least one approved modulator option.
What's actually going on in research
Trials are testing next-generation CFTR modulators that work for more mutations, including rare variants currently without treatment. Researchers are also studying mRNA therapy to deliver working CFTR instructions, gene editing to fix the mutation in lung cells, and treatments for the inflammation and infections that damage CF lungs even when modulators are working.
mRNA and gene therapy
Inhaled mRNA treatments aim to deliver temporary CFTR instructions to lung cells, potentially working for any mutation. Gene editing approaches use tools like CRISPR to correct the CFTR mutation directly in airway cells.
Next-generation modulators
New modulator combinations are being tested for rare mutations not helped by current drugs. Some trials are also testing whether different modulator combinations work better than elexacaftor-tezacaftor-ivacaftor for people who already respond to it.
Infection prevention
Studies are testing inhaled antibiotics, anti-inflammatory drugs, and treatments targeting the thick mucus itself. The goal is to prevent lung infections and damage even in people whose CFTR function has improved with modulators.
What to know before you search
Eligibility usually depends on which CFTR mutations you have, current lung function, age, and whether you're already taking modulator drugs.
What types of trials are currently open
- CFTR modulator trials — Testing new drugs or combinations that help the faulty CFTR protein work better. These often measure lung function and sweat chloride levels.
- Gene therapy trials — Testing mRNA or gene editing treatments delivered by inhaler to fix or bypass the CFTR mutation in lung cells.
- Infection trials — Testing antibiotics, inhaled treatments, or other approaches to prevent or treat the chronic lung infections common in CF.
- Inflammation trials — Testing drugs that reduce airway inflammation, which damages lungs even when infection is controlled.
- Registry studies — Following people with CF over time to track how treatments affect lung function, infections, and lifespan.
Recently added Cystic Fibrosis trials
Autogenic Drainage Versus Mechanical Percussion in Children With Cystic Fibrosis
The goal of this clinical trial is to compare the effects of autogenic drainage and mechanical percussion on lung function, dyspnea, and quality of life in children with cystic fibrosis. The main questions it aims to answer are: * Does autogenic drainage improve lung function more effectively than mechanical percussion in children with cystic fibrosis? * Does autogenic drainage reduce dyspnea (breathlessness) more effectively than mechanical percussion? * Does autogenic drainage improve health-related quality of life more effectively than mechanical percussion? Researchers will compare autogenic drainage with mechanical percussion to determine which airway clearance technique provides greater benefits for children with cystic fibrosis. Participants will: * Be randomly assigned to receive either autogenic drainage or mechanical percussion therapy. * Participate in supervised treatment sessions five times per week for six weeks. * Undergo assessments of lung function using spirometry (FEV₁ and FVC), dyspnea using the Modified Borg Dyspnea Scale, and quality of life using the Cystic Fibrosis Questionnaire-Revised (CFQ-R) before and after the intervention period. The findings of this study may help identify the most effective airway clearance technique for improving respiratory health and quality of life in children with cystic fibrosis.
A Phase 1a/1b Study to Investigate the Safety, Tolerability, Microbiological Response, and Pharmacokinetics of Inhaled Rev-56 in Adult Healthy Volunteers and Adult Non-cystic Fibrosis Bronchiectasis (NCFB) Patients With Chronic P. Aeruginosa
This is a Phase 1a/1b study to test the safety, tolerability, microbiological response, and pharmacokinetics of inhaled Rev-56 compared to placebo in adult healthy participants and non-cystic fibrosis bronchiectasis (NCFB) patients with P. aeruginosa, respectively. Phase 1a (Part A) will evaluate single doses of inhaled Rev-56 in adult healthy participants and includes up to 5 cohorts. Eligible participants will be randomized to active treatment or placebo in a 3:1 randomization scheme. Phase 1b (Part B) will evaluate the safety, tolerability, microbiological response, and pharmacokinetics of multiple doses of inhaled Rev-56 in NCFB patients with P. aeruginosa and includes 2 cohorts evaluating once daily dosing and twice daily dosing, respectively. Eligible participants will be randomized to active treatment or placebo in a 2:1 randomization scheme.
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