Cystic fibrosis gene fix tested in nasal cells

NCT ID NCT05100823

Terminated Knowledge-focused Sponsor: University Hospital, Montpellier Source: ClinicalTrials.gov ↗

First seen Jun 26, 2026 · Last updated Jun 26, 2026

Summary

This study aimed to see if a new type of genetic therapy could fix a specific problem in the CFTR gene that causes cystic fibrosis. Researchers took nasal and rectal cell samples from 16 patients and tested the therapy in the lab. The study was terminated early, so we don't have clear results on whether it works.

What this could mean

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

Active substance: oligonucleotide blockers (ONB-CFTR)
What this could lead to: If successful, this approach could point toward a new way to treat cystic fibrosis caused by certain genetic mutations.
What could go wrong: This was a very early, small study that was terminated, so results are limited. The approach has only been tested in cells, not in patients, and may not work in the body.

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.

CFTR GENE MUTATION CYSTIC FIBROSIS

Contacts and locations

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Locations

Montpellier University Hospital

Montpellier, 34090, France

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