Plain-English translation of NCT02725567 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 ivacaftor, a medication designed to help the lungs work better in very young children with cystic fibrosis who carry certain genetic mutations. Researchers studied how safe the medication was, how the body processes it, and how well it worked in infants and toddlers under 24 months old. The trial included two parts that tested different starting doses based on the child's age and weight.
Most cystic fibrosis treatments have only been studied in older children and adults. This medication had shown promise in helping people with specific genetic CF mutations, but researchers needed to find the right, safe doses for the youngest patients and make sure the treatment worked well for them.
Participants received the medication by mouth twice a day, with doses adjusted based on their weight and age. Part A involved a short 4-day dosing period to check how the body handled the medication. Part B involved taking the medication twice daily for 24 weeks. Families attended visits where researchers checked the child's health, ran blood tests, and monitored for any side effects throughout the study.
AI-generated summary from trial data · Oct 12, 2026 · Not medical advice
United States