Stem cells may predict who benefits from CF drug
NCT ID NCT03506061
First seen Sep 30, 2025 · Last updated Apr 30, 2026 · Updated 22 times
Summary
This study tested the drug Trikafta in 42 people with cystic fibrosis who have rare gene changes not normally treated with this medicine. Participants took Trikafta for about four weeks, and researchers measured lung function and sweat chloride levels. They also took skin or blood samples to grow stem cells in the lab and see if those cells could predict who would respond to the drug. The goal was to find a faster way to match rare mutations with effective treatments.
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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Locations
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Emory Children's Center
Atlanta, Georgia, 30322, United States
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University of Alabama Cystic Fibrosis Research Center
Birmingham, Alabama, 35233, United States
Conditions
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