Cystic fibrosis drug repurposed: can a liver medication fix lung function?

NCT ID NCT02323100

Terminated Disease control Sponsor: National Jewish Health Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

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.

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.

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

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States
Johns Hopkins University School of Medicine

Baltimore, Maryland, 21205, United States
National Jewish Health

Denver, Colorado, 80206, United States

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