Recruiting

Testing a new gene therapy inhaled treatment for cystic fibrosis lung disease

Plain-English translation of NCT05248230 on ClinicalTrials.gov ↗ · Source last updated July 2026 · Translation generated July 2026 · How we translate trials

Phase 2 — Testing in a bigger group (up to a few hundred people) to see if the treatment actually works and is still safe.

What is this trial?

This trial is testing a new gene therapy called , which is inhaled into the lungs to help treat cystic fibrosis. The medication is designed to help people whose bodies don't make working CFTR protein (which controls how salt and water move in and out of lung cells). Researchers want to see if this treatment is safe and whether it helps improve lung function and reduce breathing problems.

Many people with cystic fibrosis either cannot use current modulator medications, have stopped them because of side effects, or continue to have serious lung infections and breathing problems even while taking these medications. This trial exists to find out if this new gene therapy approach can help these patients who have limited treatment options.

Do you qualify?

You likely qualify if…

you are 18 years or older
you have a confirmed diagnosis of cystic fibrosis with confirmed CFTR gene mutations
your lung function (measured by breathing tests) falls within a specific range that researchers are studying
you are either ineligible for current CF medications, unable to tolerate them, or (for the sub-study) you are taking current CF medications but still having frequent lung infections or worsening lung function
your oxygen levels at rest are adequate without supplemental oxygen

You likely don't qualify if…

you have received any other gene therapy in the past
you have active infections or conditions that would interfere with how your body handles the treatment or steroid medications given during the trial
you smoke cigarettes, cigars, cannabis, or use vaping products
you have certain liver problems or blood abnormalities that show up in screening tests
you require ongoing oxygen supplementation at night or during the day

What participation looks like

You would receive a single inhalation dose of the medication during a visit. The trial lasts about 24 months, during which you will have regular check-in visits to monitor your lung function, breathing, blood work, and any side effects. Depending on which part of the trial you join, you may also receive corticosteroid medications to help your body tolerate the treatment, and researchers will carefully watch your blood sugar levels throughout the study.

AI-generated summary from trial data · Jul 18, 2026 · Not medical advice

Trial locations(20 sites)

United States

University of Alabama Child Health Research Unit, Birmingham, Alabama

The University of Arizona, Tucson, Arizona

University of California San Francisco, San Francisco, California

National Jewish Health, Denver, Colorado

University of Florida, Gainesville, Florida