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New molecules aim to correct cystic fibrosis gene errors in lab tests

NCT ID NCT03670472

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
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 Results may not be published yet.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
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This status has not been confirmed recently, so it may be out of date.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 27, 2026 · Last updated Jun 27, 2026

Summary

This study looks at whether certain molecules can fix nonsense mutations in the CFTR gene, which cause cystic fibrosis. Researchers will take cells from the noses of 85 people with cystic fibrosis and test different molecules to see which ones can restore the function of the CFTR protein. The goal is to learn which mutations can be corrected and how, paving the way for future treatments.

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.

Participants

About 85 people

The number the study aims to enrol. It can still change while the study runs.

Started

Feb 2016

Expected to finish

Jan 2030

An estimate. End dates often move.

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.

Who is studied

Patients with cystic fibrosis and carry a nonsense mutation on the 2 alleles of the gene coding for the CFTR channel.

Ages

8 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: * Male / female adults and minors aged 8 years and over * Patients with cystic fibrosis and carry a nonsense mutation on the 2 alleles of the gene coding for the CFTR channel. * Patients whose genotype of patients concerning the CFTR gene is known. * Patients with social security * Major patients who have given their consent * Minor patients with parental authorization Exclusion Criteria: * Patients who have a mutation other than nonsense in the CFTR gene * Patients whose CFTR gene was not sequenced on the 2 alleles * Patients not wishing to participate in this study or persons not giving or not able to give consent. * Pregnant or lactating women * Patients under curatorship or guardianship

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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.

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    8 sites. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Aphm Hopital La Timone - Marseille

    RECRUITING

    Marseille, France

  • Camsp Chu Amiens

    RECRUITING

    Amiens, France

  • Chu Montpellier

    RECRUITING

    Montpellier, France

  • Cmp Enfants Aphp Robert Debre - Paris

    RECRUITING

    Paris, France

  • Hopital Femme Mere Enfant - Hcl - Bron

    RECRUITING

    Bron, France

  • Hopitaux Universitaires de Strasbour

    RECRUITING

    Strasbourg, France

  • Hu Paris Centre Site Cochin Aphp - Paris 14

    RECRUITING

    Paris, France

  • Hôpital Calmette,CHU

    RECRUITING

    Lille, France

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