Could a common CF drug help those with rare mutations?
NCT ID NCT03506061
First seen Sep 30, 2025 · Last updated Jun 16, 2026 · Updated 29 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 used skin or blood samples to grow stem cells in the lab, hoping to predict who might benefit from the drug.
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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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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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