New molecules aim to correct cystic fibrosis gene errors in lab tests
NCT ID NCT03670472
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.
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Study facts
What this study's own registry entry says, in plain language.
- Participants
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About 85 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Feb 2016
- Expected to finish
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Jan 2030
An estimate. End dates often move.
- Lead sponsor
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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
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8 years and older
- Sex
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Anyone
- Healthy volunteers
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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 Hide 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.
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The places running it
8 sites. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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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
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Aphm Hopital La Timone - Marseille
RECRUITINGMarseille, France
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Camsp Chu Amiens
RECRUITINGAmiens, France
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Chu Montpellier
RECRUITINGMontpellier, France
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Cmp Enfants Aphp Robert Debre - Paris
RECRUITINGParis, France
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Hopital Femme Mere Enfant - Hcl - Bron
RECRUITINGBron, France
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Hopitaux Universitaires de Strasbour
RECRUITINGStrasbourg, France
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Hu Paris Centre Site Cochin Aphp - Paris 14
RECRUITINGParis, France
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Hôpital Calmette,CHU
RECRUITINGLille, France
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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- Can a simple questionnaire reveal why some cystic fibrosis patients skip physiotherapy?
- Cystic fibrosis diabetes: do gut hormones and genes hold the key?
- Can a special lung scan catch cystic fibrosis damage earlier than standard tests?
- Can a pill replace the liquid? testing a new form of cystic fibrosis drug
- Triple therapy under the microscope: does it transform cystic fibrosis care?