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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Contacts and locations

Locations

  • Emory Children's Center

    Atlanta, Georgia, 30322, United States

  • University of Alabama Cystic Fibrosis Research Center

    Birmingham, Alabama, 35233, United States

Conditions

Explore the condition pages connected to this study.