Testing an IV medication to treat stubborn lung infections in cystic fibrosis patients

Plain-English translation of NCT04294043 on ClinicalTrials.gov ↗ · Source last updated October 2025 · Translation generated June 2026 · How we translate trials

Phase 1 — Testing in a small group (usually 20–80 people) to find a safe dose and watch for side effects.

What is this trial?

This study is testing a medication called given intravenously (through an IV) to help treat a specific type of stubborn lung infection that affects some people with cystic fibrosis. The infection is caused by bacteria called nontuberculous mycobacteria, which can be hard to clear with standard antibiotics. Researchers want to see if this medication is safe and whether it can help fight these infections.

Some people with cystic fibrosis develop lung infections that either never respond to antibiotics or come back after treatment stops. This medication hasn't been tested yet in this group of patients, so the trial is exploring whether it could offer a new treatment option for people whose current antibiotics aren't working well enough.

Do you qualify?

You likely qualify if…

you are 18 years of age or older
you have been diagnosed with cystic fibrosis
you have had repeated positive cultures showing nontuberculous mycobacteria in your sputum or lung fluid
you either have never been treated for your current infection, or your previous treatment cleared the infection more than 2 years ago (treatment-naïve group) — OR you have been treated with standard antibiotics for more than 12 months without clearing the infection (treatment-refractory group)
you are able to cough up sputum for testing
you have lung function (FEV1) of at least 25% of predicted value

You likely don't qualify if…

you are unable to attend study visits or follow the study schedule
you have been hospitalized within the past 7 days
you are not able or willing to continue (or start) standard antibiotic therapy for your infection during the study
your health status has changed significantly in the past week
you are unable to provide informed consent or have your legal representative provide it

What participation looks like

If you join this study, you will receive the medication through an IV catheter as two separate 5-day treatment cycles. During each cycle, the medication will be infused continuously over 24 hours for 5 days in a row, which you can do at home using a portable infusion pump. You will need to attend multiple study visits for lung function tests, sputum samples, and blood work to check how the treatment is working and monitor for any side effects. The entire study will take several months, and you will need to be willing to stop taking azithromycin (a chronic antibiotic) while enrolled.

AI-generated summary from trial data · Jun 5, 2026 · Not medical advice

Trial locations(10 sites)

United States

University of Alabama at Birmingham, Birmingham, Alabama

National Jewish Health, Denver, Colorado

Johns Hopkins University, Baltimore, Maryland

The Minnesota Cystic Fibrosis Center, Minneapolis, Minnesota

Nationwide Children's Hospital, Columbus, Ohio