Cystic fibrosis is a genetic disease that causes thick mucus to build up in the lungs and other organs, leading to infections and digestive problems. About 40,000 people in the US have CF. Treatment now includes CFTR modulators — drugs that fix the underlying protein defect — which have changed life expectancy and quality of life for most people with CF.
What's actually going on in research
Trials are testing next-generation CFTR modulators that work for more gene mutations, including rare ones not helped by current drugs. Researchers are also studying mRNA therapy to deliver working CFTR instructions, gene editing to correct the mutation permanently, and treatments for lung infections and inflammation that persist even with modulators. Anti-inflammatory drugs and mucolytics that break up thick mucus are in active testing.
Next-generation modulators
Newer CFTR modulators aim to help people with mutations that don't respond to elexacaftor-tezacaftor-ivacaftor (Trikafta). Some are designed as single pills or to work across a broader range of mutations.
mRNA and gene therapy
Trials are testing inhaled mRNA that could deliver working CFTR instructions directly to lung cells. Gene editing approaches using CRISPR aim to correct the CFTR mutation in cells permanently.
Infection and inflammation
Even with modulators, lung infections and inflammation remain problems. Trials are testing new antibiotics, anti-inflammatory drugs, and inhaled treatments to reduce mucus and infection.
What to know before you search
Eligibility typically depends on specific CFTR mutations, lung function (measured by FEV1), age, and whether you're currently taking modulator therapy.
What types of trials are currently open
- CFTR modulator trials — Testing new drugs that improve function of the defective CFTR protein, often targeting specific mutations or aiming to work better than current modulators.
- Gene therapy trials — Testing approaches that deliver working CFTR genetic instructions to cells, either through mRNA or gene editing techniques.
- Infection trials — Testing new antibiotics and inhaled treatments for chronic lung infections, including those caused by Pseudomonas and other bacteria.
- Anti-inflammatory trials — Testing drugs to reduce lung inflammation, which contributes to lung damage even when infections are controlled.
- Mucolytic trials — Testing treatments that break up or thin the thick mucus characteristic of CF, making it easier to clear from airways.
Recently added Cystic Fibrosis trials
Try a high-intensity exercise program to improve breathing and symptoms
The aim of our study is to investigate the effect of high-intensity interval exercise (HIIE) on the symptoms of bronchiectasis in patients with the disease. The main questions it aims to answer are: 1. Does HIIE improve respiratory function in patients with bronchiectasis? 2. Does HIIE increase functional capacity, peripheral muscle strength, and quality of life in patients with bronchiectasis? 3. Does HIIE reduce the severity of dyspnea, cough symptoms, and fatigue in patients with bronchiectasis? The study was designed as a randomized controlled trial. Participants were divided into two groups: intervention and control. Participants will: The intervention group will receive HIIE training three days a week along with airway clearance techniques. The control group would receive only airway clearance techniques. Airway clearance techniques will be provided clinically (three days a week) and at home, according to the needs of the individuals each day of the week.
Try a breathing technique to improve lung health in 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.
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