Small Circulating RNA as Molecular Markers of Lung Disease in Cystic Fibrosis

Study Overview
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Brief Summary
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The aim of our study is to assess miRNAs expression profiles in the circuling blood of patients with cystic fibrosis and highlight signatures that could reflect the pulmonary status of patients

Detailed Description
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The objective of this project is to study the circulating miRNA profiles in 40 patients with cystic fibrosis (5 samples which are acquired through a secondary use) and 40 healthy individuals to assess whether these biomolecules could be used as markers of the pulmonary disease in cystic fifbosis. Moreover by comparing miRNAs expression level between Cystic fibrosis (CF) patients with severe (n=20) or moderate (n=20) pulmonary impairment, we want to assess whether some of these miRNAs may be used as markers for the severity of CF pulmonary disease. The identification of sensitive and early markers, from a non-invasive sampling could enable more effective and early treatment of CF patients.

Official Title
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Determination of Circulating miRNAs as Diagnostic Markers of Lung Disease in Cystic Fibrosis

Conditions
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Cystic Fibrosis

Intervention / Treatment
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* Other: miRNAs isolation from blood samples of patients and control


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Patients with Cystic fibrosis (CF) (MIM#219700) who are compound heterozygous or homozygous for CF causing mutations Healthy controls non -smokers and free pulmonary disease Exclusion Criteria: Participation or within the exclusion period of other clinicals trials Patients carrying mutations of clinical varying consequences or non CF-causing mutations smokers

Ages Eligible for Study
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Minimum Age: 12 Months
Maximum Age: 65 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: Diagnostic
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Other: Cystic fibrosis Patients<br> | Other: miRNAs isolation from blood samples of patients and control<br>* Blood sample collection in specific PAXGene tubes<br>|
| Other: Patients without fibrosis cystic<br> | Other: miRNAs isolation from blood samples of patients and control<br>* Blood sample collection in specific PAXGene tubes<br>|
| Other: Cystic fibrosis Patients (secondary use of samples)<br> | Other: miRNAs isolation from blood samples of patients and control<br>* Blood sample collection in specific PAXGene tubes<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Comparison of miRNAs expression between Cystic Fibrosis (CF) patients and healthy controls | Compare the distributions of miRNAs expression in blood samples of CF patients and to healthy controls | After blood collection: 2 years | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Assesment of miRNAs expression in Cystic Fibrosis Patients depending on the pulmonary status | Compare the distributions of miRNAs expression in blood samples of CF patients with mild lung disease and CF patients with severe lung disease | After blood collection 2 years | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Patient with Cystic Fibrosis, Patient without Cystic fibrosis