Plain-English translation of NCT04509050 on ClinicalTrials.gov ↗ · Source last updated · Translation generated · How we translate trials
Read our Cystic Fibrosis research guide →The BEGIN Study is following young children with cystic fibrosis to understand how two medications—ivacaftor and elexacaftor/tezacaftor/ivacaftor—work in their bodies and affect their growth, lung function, digestion, and overall health. Researchers want to learn more about how these medications help children in the early years of life, when treatment might have the biggest impact. This information could help doctors better care for children newly diagnosed with cystic fibrosis.
These medications are newer and very effective at fixing the underlying cystic fibrosis protein problem, but doctors don't yet fully understand how they affect children's growth, nutrition, bone health, and long-term lung function when given early in life. This study aims to fill that gap so that doctors can better predict and support children's health as they grow.
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The study has two parts. In the first part, children not yet on these medications visit the clinic up to 8 times over 5 years for measurements like growth, lung function, and blood tests. In the second part, children who start the medication have a baseline visit within 30 days before starting treatment, then 8 follow-up visits over 5 years to track how the medication affects their growth, lung health, digestion, bone strength, and other body systems. The study takes place at multiple medical centers and lasts up to 10 years total.
AI-generated summary from trial data · Jul 18, 2026 · Not medical advice
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