Could a common CF drug help those with rare mutations?

NCT ID NCT03506061

Completed Disease control Sponsor: Emory University Source: ClinicalTrials.gov ↗

First seen Jun 26, 2026 · Last updated Aug 12, 2026 · Updated 2 times

Summary

This study tested the drug Trikafta in 42 people with cystic fibrosis who have rare genetic mutations not currently approved for this treatment. 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 into airway cells in the lab, aiming to see if those cells could predict how well a person would respond to the drug.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: Trikafta (elexacaftor, tezacaftor, ivacaftor)
What this could lead to: If successful, this could expand Trikafta's approval to more people with cystic fibrosis who have rare mutations, offering them a treatment option.
What could go wrong: This is a small, early-phase study with only 42 participants, so results may not apply to everyone. The stem cell test is experimental and may not reliably predict real-world benefits.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

cystic fibrosis

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

CYSTIC FIBROSIS

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

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