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
The Effects of Yoga Practice on Exercise Capacity, Arterial Stiffness, Respiratory Function, Peripheral Muscle Strength, Posture and Quality of Life in Children With Cystic Fibrosis
The aim of this study is to investigate the effects of exercise training applied through telerehabilitation on exercise capacity, peripheral and respiratory muscle strength, arterial stiffness, posture and quality of life in children with cystic fibrosis.
Continuous Glucose Monitoring Parameter and Oral Glucose Tolerance Testing (OGTT) Parameters in Children and Young Adults With Cystic Fibrosis (CF)
As modulator therapies extend the lifespan of CF patients, the detection of complications like Cystic Fibrosis Related Diabetes (CFRD) becomes increasingly vital. The current gold standard for CFRD screening, the 2-hour OGTT, is cumbersome and suffers from low compliance. This study proposes that Continuous Glucose Monitoring (CGM) could offer a more comprehensive, easier, and potentially more compliant screening method. The research aims to correlate various CGM parameters (e.g., time in hyperglycemia) with traditional glycemic markers like OGTT glucose levels, GMI, average glucose, variability, and HbA1c. Additionally, the research seeks to understand patient and parent/guardian preferences for CFRD screening modalities. This prospective observational study will involve CF patients wearing a Dexcom CGM around the time of standard OGTT and HbA1c tests, followed by surveys, with the ultimate goal of paving the way for CGM to improve early detection and management of dysglycemia in CF children.
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