Action of Epigenetic Modifiers in Cystic Fibrosis Treatment

Study Overview
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Brief Summary
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Epigenetic modifiers has been showed to rescue F508del-CFTR channel to apical membrane of epithelial cell lines. In this study, the investigators evaluate epigenetic modifiers effects firstly on CFTR rescue, then on secretion and synthesis of inflammatory factors (IL-8, LXA4 and SCGB1A1) and mucines (MUC5AC and MUC5B) in a dynamic epithelium model using an air-liquide interface culture of nasals cells from CF patients or controls.

Official Title
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Action of Epigenetic Modifiers in Cystic Fibrosis Treatment: ex Vivo Model of Nasal Epithelium of CF Patients

Conditions
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Cystic Fibrosis, Healthy Subjects

Intervention / Treatment
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* Other: Tests in vitro after sampling nasal cells of CF patients or controls


Participation Criteria
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Eligibility Criteria
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General inclusion criteria : informed consent benefit from disease insurance regimen men and women Inclusion Criteria for CF patients 2 severe CF mutations age superior or equal to 12 Inclusion Criteria for controls : age superior or equal to 18 no smoker (for 5 years) General exclusion criteria : participation to an other interventionnal study subject in exclusion period law protected subject pregnant and breast fooding Specific Exclusion Criteria: Xylocaine hypersensibility Porphyria severe hepatic failure Epilepsy Severe cardiac failure local anesthesic contra indication Specific Control subject Exclusion Criteria: respiratory disease cystic fibrosis acute infection < 6 weeks on treatment antibiotic treatment < 3 months

Ages Eligible for Study
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Minimum Age: 12 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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Accepts Healthy Volunteers

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Basic Science
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Cystic Fibrosis patients (CF)<br>Tests in vitro after sampling nasal cells of CF patients or controls are the intervention done on these subjects | Other: Tests in vitro after sampling nasal cells of CF patients or controls<br>* Nasal cells of CF patients or controls were collected by scratching of intermediate turbinate and cultured in vitro. Epigenetic modifiers treatment was applied to these ex vivo nasal epithelia. Then, mRNA, protein and secretions were quantified.<br>|
| Other: Control subjects (non CF)<br>Tests in vitro after sampling nasal cells of CF patients or controls are the intervention done on these subjects | Other: Tests in vitro after sampling nasal cells of CF patients or controls<br>* Nasal cells of CF patients or controls were collected by scratching of intermediate turbinate and cultured in vitro. Epigenetic modifiers treatment was applied to these ex vivo nasal epithelia. Then, mRNA, protein and secretions were quantified.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Mature CFTR protein percentage variation after in vitro epigenetics modifiers treatment |  | 24 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Pro-inflammatory cytokines number variations after epigenetic modifiers treatment |  | 24 months | 
| Anti-inflammatory cytokines number variations after epigenetic modifiers treatment |  | 24 months | 
| Mucin composition variations after epigenetic modifiers treatment |  | 24 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Cystic Fibrosis, nasal epithelia, mucins