Plain-English translation of NCT04058353 on ClinicalTrials.gov ↗ · Source last updated · Translation generated · How we translate trials
Read our Cystic fibrosis research guide →Phase 3 — Testing in thousands of people, comparing the treatment against what doctors currently use. This is the last big step before approval.
This trial tested elexacaftor combined with two other medications (tezacaftor and ivacaftor) in people with cystic fibrosis who had two specific types of genetic mutations. Researchers compared people taking the new three-drug combination to those taking standard treatments over an 8-week period to measure whether the new medication improved lung function and was well-tolerated.
Cystic fibrosis is a serious genetic condition that damages the lungs and digestive system. This trial examined whether combining three medications together could better correct the underlying genetic defect and improve breathing in people with certain cystic fibrosis mutations who had limited treatment options.
Participants first took standard cystic fibrosis medications for 4 weeks to establish a baseline. They were then randomly assigned to either continue their standard medications or switch to the new three-drug combination for 8 weeks. Throughout the trial, researchers measured lung function and monitored for any side effects.
AI-generated summary from trial data · Sep 21, 2026 · Not medical advice
United States