Could a common CF drug help those with rare mutations?
NCT ID NCT03506061
First seen Jun 26, 2026 · Last updated Jun 26, 2026
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.
Disclaimer
Read more
Show less
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.
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.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for CYSTIC FIBROSIS are added.
By submitting, you agree to our Terms of use
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
By submitting, you agree to our Terms of use
Locations
-
Emory Children's Center
Atlanta, Georgia, 30322, United States
-
University of Alabama Cystic Fibrosis Research Center
Birmingham, Alabama, 35233, United States