Recruiting

Testing a new inhaled medication for a specific type of cystic fibrosis

Plain-English translation of NCT06429176 on ClinicalTrials.gov ↗ · Source last updated May 2026 · Translation generated June 2026 · How we translate trials

Read our Cystic Fibrosis research guide →

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 is a Phase 2 study testing a new medication called for adults with cystic fibrosis who have a specific genetic mutation (3849+10 Kb C→T). Researchers want to learn whether is safe to use and whether it helps improve lung function. Some participants will receive the medication while others receive a placebo (a dummy inhaler with no medicine) so researchers can compare the two.

Many current cystic fibrosis treatments don't work for people with this particular genetic mutation. This trial is exploring whether this medication could offer a new treatment option for this group of patients.

Do you qualify?

You likely qualify if…

you have been diagnosed with cystic fibrosis and carry two copies of the specific 3849+10 Kb C→T mutation (or one copy plus another CF-causing mutation)
you are an adult with a healthy body weight (BMI of at least 17 kg/m²)
your lung function (FEV1) is between 40–90% of predicted values at screening
you have not smoked or used vaping products for at least 6 months before starting the study

You likely don't qualify if…

you are currently taking other CF modulator drugs like Kalydeco, Orkambi, Symdeko, or Trikafta (unless you're in a later group specifically testing combination therapy)
you have had a serious lung infection, pulmonary exacerbation, or respiratory illness within 2 weeks before the study starts
you have coughed up more than 30 mL of blood in the past 3 months or were hospitalized for coughing up blood
you have advanced liver disease with cirrhosis or portal hypertension
you have had an organ transplant

What participation looks like

You will visit the clinic approximately once a week for 9–12 weeks. At each visit, you'll receive an inhaled dose of either the study medication or a placebo, and the research team will perform check-ups and tests to monitor your safety and lung function. You won't know whether you're receiving the real medication or the placebo, and neither will the researchers, to keep the results fair and unbiased.

AI-generated summary from trial data · Jun 3, 2026 · Not medical advice

Testing a new inhaled medication for a specific type of cystic fibrosis

What is this trial?

Do you qualify?

What participation looks like

Trial locations