Cystic fibrosis drug repurposed: can a liver medication fix lung function?
NCT ID NCT02323100
First seen Apr 25, 2026 · Last updated Jun 19, 2026 · Updated 6 times
Summary
This early-phase trial tested whether glycerol phenylbutyrate (Ravicti), a drug already used for urea cycle disorders, could help restore chloride transport in the nasal cells of adults with cystic fibrosis. The study enrolled 16 participants and compared low-dose Ravicti to a placebo. However, the trial was terminated early, so the full results are not available.
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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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Contacts and locations
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Locations
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Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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Johns Hopkins University School of Medicine
Baltimore, Maryland, 21205, United States
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National Jewish Health
Denver, Colorado, 80206, United States
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
glycerol phenylbutyrate (Ravicti)
What this could lead to
If it worked, this could point toward a new way to improve CFTR function in people with cystic fibrosis who still produce some CFTR protein.
What could go wrong
The trial was terminated early with only 16 participants, so results are very limited. It was a small, early-phase study testing a repurposed drug, so even if promising, much more research would be needed.
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.