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Cystic fibrosis gene fix tested in nasal cells

NCT ID NCT05100823

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

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early This study
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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.

Study facts

What this study's own registry entry says, in plain language.

Phase

Not a phased trial

Phase numbers describe drug development. The registry uses this when they do not apply, as it does for trials of devices, procedures or behaviour changes, and for observational studies.

Participants

16 people

The number who actually took part.

Started

Mar 2022

Finished

Feb 2025

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

12 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * The subject must have given their free and informed consent and signed the consent * The subject must be affiliated or beneficiary of a health insurance plan Women and men are included * The patient is at least 12 years old. * The patient has cystic fibrosis or a CFTR pathology and therefore carries two mutations (with at least one mutation affecting splicing) in the CFTR gene. * Patients who volunteer for rectal biopsy collection (only from Montpellier University Hospital) must be at least 18 years old. Exclusion Criteria: * The subject is in a period of exclusion determined by a previous study. * The subject is under judicial protection, under guardianship or under curatorship * The subject does not accept to sign consent * It turns out to be impossible to give informed information to the subject * The subject does not read the French language fluently * The subject is a pregnant or breastfeeding woman * The subject has porphyria, or has hepatic insufficiency, or suffers from epilepsy, or suffers from conduction disorders, or suffers from severe heart failure, has a cons-indication to the use of a local anesthetic spray. Specific non-inclusion criteria for rectal sampling: * the subject has thrombocytopenia * the subject has a bleeding disorder * The patient has severe inflammation of the rectum.

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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

Locations

  • Montpellier University Hospital

    Montpellier, 34090, France

More trials for these conditions

Other studies related to the condition(s) this trial covers.