Recruiting

Testing a new gene therapy treatment for cystic fibrosis lung disease

Plain-English translation of NCT05248230 on ClinicalTrials.gov ↗ · Source last updated March 2026 · Translation generated June 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 , an experimental gene therapy that is breathed in directly to the lungs. The study aims to find out if this new treatment is safe and whether it can help adults with cystic fibrosis whose lungs are affected by the disease. The treatment is being tested in people who either cannot take current CF medications or don't tolerate them well, as well as in people already on CF medications who are still experiencing frequent lung problems.

Current cystic fibrosis medications don't work for everyone — some patients can't take them due to side effects, and others continue to have serious lung problems even while on treatment. This trial explores whether a gene therapy approach might help patients who aren't responding well to existing options.

Do you qualify?

You likely qualify if…

you are 18 years or older
you have a confirmed diagnosis of cystic fibrosis with genetic testing showing CFTR gene mutations
you are unable to take or have stopped taking current CF modulator medications due to side effects (for the main study)
you have lung function between 50% and 90% of normal (or 40% to 70% for the sub-study), and your oxygen levels at rest are adequate
you have not previously received any gene therapy treatments

You likely don't qualify if…

you have an active mycobacterium infection or fungal infection of the lungs requiring treatment
you smoke, vape, or use cannabis, or have done so in the past 2 months
you need to take steroid or immunosuppressant medications regularly for another medical condition
you have poorly controlled diabetes or blood sugar levels that are unstable
you require continuous oxygen support or have advanced liver cirrhosis related to cystic fibrosis

What participation looks like

Participants will receive a single dose of the medication breathed in through inhalation, either as part of the dose-finding phase or the expansion phase of the study. You will be monitored for safety and effectiveness over a 24-month observation period with regular study visits to check your lung function, oxygen levels, and overall health. The study involves blood tests, lung function measurements, and assessments of how often you have lung infections or need antibiotics.

AI-generated summary from trial data · Jun 3, 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