Cystic FibrosisEpiithelixNCT03652090
Primary Nasal Cell Culture as a Tool for Personalized Therapy in Cystic Fibrosis
An observational study of Cystic Fibrosis, run by Institut National de la Santé Et de la Recherche Médicale, France.
Trial summary
characterization of CFTR function and expression in nasal primary cells collected from patients with cystic fibrosis in comparison to their parents, healthy heterozygotes and healthy controls Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- cell sampling
- Type of treatment
- Surgery or procedure
- How many people
- 112 participants
- Where
- No sites listed yet
- Study dates
- 1 Sep 2010to 3 Mar 2016
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- Child, Adult, Older adult
Study population: patients with Cystic Fibrosis with 2 mutations in CFTR * healthy heterozygotes with 1 mutation in CFTR * healthy subjects with no familial history of Cystic Fibrosis and no symptoms compatibel with Cystic Fibrosis
Who may be able to join
- patients with Cystic Fibrosis with 2 mutations in CFTR
- healthy heterozygotes with 1 mutation in CFTR
- healthy subjects with no familial history of Cystic Fibrosis and no symptoms suggesting Cystic Fibrosis
Who cannot join
- smoking
About this study
Official title: Evaluation of the Primary Human Nasal Epithelial Cell Culture Model in the Context of Personalized Therapy in Cystic Fibrosis
characterization of CFTR function and expression in nasal primary cells collected from patients with cystic fibrosis in comparison to their parents, healthy heterozygotes and healthy controls
Read the detailed description
3 groups of subjects are enrolled CF subjects according to their genotypes (aiming to enroll patients carrying 2 CF causing mutations with no CFTR expression/function, and patients carrying at least 1 mutation with residual function, such R117H) Parents or siblings of the CF subjects, as healthy hétérozygotes healthy controls All these subjects experience nasal brushings. From these nasal brushings,nasal cells are expanded, and cultured in air liquid interface to obtain polarized epithelium. This epithelium is then studied in Ussing chamber experiments to characterize the level of cAMP dependant Chloride transport and Sodium reabsorption. Apical expression of CFTR is assessed by immunofluorescence.
Results will allow to define the variability of CFTR function and expression criteria in subjects with the same genotype. Such data are crucial for interpretation of the effect of CFTR modulators.
- Conditions
- Cystic Fibrosis
- Being tested
- cell sampling
- Keywords
- biomarker
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
cell samplingProcedure or surgery
nasal brushing to collect cells
| Study group | Role | What they receive |
|---|---|---|
| cystic fibrosis patients | Cystic fibrosis patients carrying to 2 CFTR mutations undergoing cell sampling Receives: Procedure: cell sampling | |
| healthy heterozygotes | healthy heterozygotes carrying 1 CFTR mutations undergoing cell sampling Receives: Procedure: cell sampling | |
| healthy control | subject with no evidence of any symptoms compatible with Cystic Fibrosis undergoing cell sampling Receives: Procedure: cell sampling |
What is being measured
The primary outcome is the main question the study is designed to answer.
variation in the short-circuit-current (Isc) after Forskolin (Forskolin)/IBMx and VX-770 (∆IscFsk/IBMx+VX-770)
The short-circuit-current (Isc) was measured under voltage clamp conditions. Inhibitors and activators were added after stabilization of baseline Isc. The sum of the change after Forskolin (Forskolin)/IBMx and VX-770 (∆IscFsk/IBMx+VX-770) served as an index of CFTR function.
Measured: 1 day
Secondary outcomes (1)
percentage of cells displaying apical staining
CFTR immuno-detection was performed as previously described 31. Apical CFTR staining was assessed semi quantitatively as the percentage of cells displaying apical staining multiplied by the average corrected apical fluorescence 32.
Measured: 1 day
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Aug 2018
- Study start1 Sep 2010
- Primary completion3 Mar 2016
- Study completion3 Mar 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- More trials run by Institut National de la Santé Et de la Recherche Médicale, FranceThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Cross-sectional
- Enrolment
- 112 participants (actual)
Sponsor and investigators
- Sponsor type
- Other government
- Collaborators
- ABCF2
- Responsible party
- Principal investigator, Isabelle Sermet-Gaudelus, Institut National de la Santé Et de la Recherche Médicale, France
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03652090
- Sponsor's protocol ID
- 2010-05-03 A3
- Acronym
- Epiithelix
- Status last verified
- Mar 2019
- First submitted
- 28 Aug 2018
- Last updated
- 12 Mar 2019
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 Mar 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evaluation of the Primary Human Nasal Epithelial Cell Culture Model in the Context of Personalized Therapy in Cystic Fibrosis
- ClinicalTrials.gov ID
- NCT03652090
- Acronym / sponsor ID
- Epiithelix / 2010-05-03 A3
- Conditions
- Cystic Fibrosis
- Keywords
- biomarker
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 112 participants (actual)
- Lead sponsor
- Institut National de la Santé Et de la Recherche Médicale, FranceOther government
- Collaborators
- ABCF2
- Study start
- 1 Sep 2010 (actual)
- Primary completion
- 3 Mar 2016 (actual)
- Study completion
- 3 Mar 2016 (actual)
- First posted
- 29 Aug 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
characterization of CFTR function and expression in nasal primary cells collected from patients with cystic fibrosis in comparison to their parents, healthy heterozygotes and healthy controls
Detailed description
3 groups of subjects are enrolled CF subjects according to their genotypes (aiming to enroll patients carrying 2 CF causing mutations with no CFTR expression/function, and patients carrying at least 1 mutation with residual function, such R117H) Parents or siblings of the CF subjects, as healthy hétérozygotes healthy controls All these subjects experience nasal brushings. From these nasal brushings,nasal cells are expanded, and cultured in air liquid interface to obtain polarized epithelium. This epithelium is then studied in Ussing chamber experiments to characterize the level of cAMP dependant Chloride transport and Sodium reabsorption. Apical expression of CFTR is assessed by immunofluorescence.
Results will allow to define the variability of CFTR function and expression criteria in subjects with the same genotype. Such data are crucial for interpretation of the effect of CFTR modulators.
Study design
- Observational model
- Cohort
- Time perspective
- Cross-sectional
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| cystic fibrosis patients | – | Procedure: cell samplingCystic fibrosis patients carrying to 2 CFTR mutations undergoing cell sampling |
| healthy heterozygotes | – | Procedure: cell samplinghealthy heterozygotes carrying 1 CFTR mutations undergoing cell sampling |
| healthy control | – | Procedure: cell samplingsubject with no evidence of any symptoms compatible with Cystic Fibrosis undergoing cell sampling |
Who may qualify
- Sex
- All
- Age groups
- Child, Adult, Older adult
- Study population
- patients with Cystic Fibrosis with 2 mutations in CFTR * healthy heterozygotes with 1 mutation in CFTR * healthy subjects with no familial history of Cystic Fibrosis and no symptoms compatibel with Cystic Fibrosis
Key inclusion criteria (3 listed)
- patients with Cystic Fibrosis with 2 mutations in CFTR
- healthy heterozygotes with 1 mutation in CFTR
- healthy subjects with no familial history of Cystic Fibrosis and no symptoms suggesting Cystic Fibrosis
Key exclusion criteria (1 listed)
- smoking
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| cell samplingnasal brushing to collect cells | Procedure or surgery | cystic fibrosis patients, healthy control, healthy heterozygotes |
Primary outcome
variation in the short-circuit-current (Isc) after Forskolin (Forskolin)/IBMx and VX-770 (∆IscFsk/IBMx+VX-770)
Time frame: 1 day
The short-circuit-current (Isc) was measured under voltage clamp conditions. Inhibitors and activators were added after stabilization of baseline Isc. The sum of the change after Forskolin (Forskolin)/IBMx and VX-770 (∆IscFsk/IBMx+VX-770) served as an index of CFTR function.
1 secondary outcome
percentage of cells displaying apical staining
Time frame: 1 day
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Mar 2019.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
An observational study of Cystic Fibrosis, run by Institut National de la Santé Et de la Recherche Médicale, France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 12 Mar 2019. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
variation in the short-circuit-current (Isc) after Forskolin (Forskolin)/IBMx and VX-770 (∆IscFsk/IBMx+VX-770) (time frame: 1 day). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Mar 2019.