Plain-English translation of NCT03224351 on ClinicalTrials.gov ↗ · Source last updated · Translation generated · 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.
This trial tested whether a new medication called , when combined with two existing cystic fibrosis drugs (tezacaftor and ivacaftor), could safely and effectively treat cystic fibrosis in adults with certain genetic mutations. The study examined whether this three-drug combination was better than existing treatments or placebo at improving lung function and overall health.
Many people with cystic fibrosis have genetic mutations that don't respond well to current approved medications. This trial investigated whether adding to an existing two-drug combination could help more patients control their disease and improve their quality of life.
The trial was divided into three parts, with participants receiving either the new three-drug combination at different dose levels, an existing two-drug treatment, or placebo. Participants took their assigned medications daily for 4 weeks, with some participants then entering a short washout period. The study involved multiple visits to the research clinic where participants had their lung function and other health measures tested to see how well the treatment worked.
AI-generated summary from trial data · Oct 12, 2026 · Not medical advice
United States