Tailored CF drug tests offer hope for rare mutations

NCT ID NCT04580368

First seen Nov 01, 2025 · Last updated May 06, 2026 · Updated 26 times

Summary

This study aims to find effective treatments for people with cystic fibrosis who have rare gene mutations not covered by current drugs. Researchers will test FDA-approved CF drugs on nasal cells in the lab, then confirm the best option in a personalized N-of-1 trial. Up to 50 participants aged 6 and older will be enrolled. The goal is to improve lung function by at least 5%.

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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

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • CCHMC

    RECRUITING

    Cincinnati, Ohio, 45203, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

Conditions

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