Recruiting

Finding the right cystic fibrosis drug for your genes

Plain-English translation of NCT04580368 on ClinicalTrials.gov ↗ · Source last updated January 2026 · Translation generated June 2026 · How we translate trials

Read our Cystic Fibrosis research guide →

This study doesn't follow the usual testing phases — it may be an observational study or a different type of research.

What is this trial?

This trial helps find the best cystic fibrosis medication for people with rare genetic mutations. Researchers will grow cells from your nose in a lab and test how they respond to medications like ivacaftor, orkambi, symdeko, or trikafta. Then, if the lab results look promising, you'll try the medication yourself to see if it actually helps you breathe better.

Many people with cystic fibrosis have rare genetic mutations that doctors don't know much about, making it hard to predict which medications will work. This study bridges that gap by testing the medication on your own cells before you take it, so your doctor can be more confident it will help.

Do you qualify?

You likely qualify if…

you have cystic fibrosis with a rare CFTR gene mutation (one that affects fewer than 5% of people with CF)
you are at least 6 years old and within the approved age range for the medication your doctor is prescribing
your lung function is at least 50% of what is expected for your age
you have been on stable cystic fibrosis treatments for at least 28 days with no recent changes
your doctor has already decided to prescribe you a CFTR modulator medication

You likely don't qualify if…

you are pregnant, breastfeeding, or unwilling to use birth control during the study
you have serious infections like nontuberculous mycobacteria or Burkholderia growing in your lungs
you are taking certain medications that interfere with the study drug, such as voriconazole, fluconazole, or high-dose prednisone
you have advanced liver disease, kidney disease, or a history of organ transplant
you have very low blood cell counts or other serious lab abnormalities at screening

What participation looks like

You'll start with a 2-week preparation period, then a 4-week period without the medication to establish a baseline. Researchers will collect cells from your nose through a gentle brush swab and test how they respond to the medication in the lab. Based on those results, you'll take the medication your doctor prescribed for several weeks while the study team tracks how you're doing through visits and measures your lung function and symptoms.

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

Trial locations(1 site)

United States

CCHMC, Cincinnati, Ohio