Personalized drug trial offers hope for cystic fibrosis patients with rare gene mutations
NCT ID NCT03587961
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This early-phase trial is testing whether certain cystic fibrosis drugs, called CFTR modulators, can help people with rare gene mutations that are not currently approved for these medications. The study will enroll 20 participants aged 6 and older and measure changes in lung function. The goal is to see if personalized treatment can improve breathing in patients who have few other options.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
Symdeko (also known as Orkambi, Ivacaftor)
What this could lead to
If it works, this could expand treatment options for people with cystic fibrosis who have rare mutations not currently covered by approved drugs.
What could go wrong
This is a very early, small trial with only 20 participants, so results may not apply widely. The drugs are used off-label and may not work for all mutations, and there are risks like side effects or no improvement in lung function.
Disclaimer
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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.
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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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
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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University of Alabama at Birmingham
RECRUITINGBirmingham, Alabama, 35233, United States
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact Phone: •••-•••-•••• Email: •••••@•••••