Testing a highly effective CF medicine in young children
Part of Digestive system, Genetic & congenital, Lungs & breathing clinical trials.
This study looks at how well a highly effective cystic fibrosis (CF) medicine works in infants and young children, and what biological changes it causes. It may help families understand whether the medicine improves CF function and related measures early in life.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Be under 10 years old at the first study visit
- Have a confirmed diagnosis of cystic fibrosis (CF)
- Have specific CF gene changes that match the FDA-approved “highly effective modulator” treatment (ivacaftor or elexacaftor/tezacaftor/ivacaftor)
- Your doctor must plan to prescribe ivacaftor or elexacaftor/tezacaftor/ivacaftor
- Did not take any study medicine within 28 days before the first visit
- Have not taken ivacaftor or elexacaftor/tezacaftor/ivacaftor in the 28 days before the first visit
- Have not used chronic by-mouth steroid medicine (oral corticosteroids) in the 28 days before the first visit
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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