Action of Epigenetic Modifiers in Cystic Fibrosis Treatment (Mod2EpiCF)
Primary Purpose
Cystic Fibrosis, Healthy Subjects
Status
Completed
Phase
Not Applicable
Locations
France
Study Type
Interventional
Intervention
Tests in vitro after sampling nasal cells of CF patients or controls
Sponsored by
About this trial
This is an interventional basic science trial for Cystic Fibrosis focused on measuring Cystic Fibrosis, nasal epithelia, mucins
Eligibility Criteria
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
Sites / Locations
- Respiratory Diseases Department
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Other
Arm Label
Cystic Fibrosis patients (CF)
Control subjects (non CF)
Arm Description
Tests in vitro after sampling nasal cells of CF patients or controls are the intervention done on these subjects
Tests in vitro after sampling nasal cells of CF patients or controls are the intervention done on these subjects
Outcomes
Primary Outcome Measures
Mature CFTR protein percentage variation after in vitro epigenetics modifiers treatment
Secondary Outcome Measures
Pro-inflammatory cytokines number variations after epigenetic modifiers treatment
Anti-inflammatory cytokines number variations after epigenetic modifiers treatment
Mucin composition variations after epigenetic modifiers treatment
Full Information
NCT ID
NCT01883284
First Posted
June 13, 2013
Last Updated
December 2, 2014
Sponsor
University Hospital, Montpellier
1. Study Identification
Unique Protocol Identification Number
NCT01883284
Brief Title
Action of Epigenetic Modifiers in Cystic Fibrosis Treatment
Acronym
Mod2EpiCF
Official Title
Action of Epigenetic Modifiers in Cystic Fibrosis Treatment: ex Vivo Model of Nasal Epithelium of CF Patients
Study Type
Interventional
2. Study Status
Record Verification Date
December 2014
Overall Recruitment Status
Completed
Study Start Date
January 2012 (undefined)
Primary Completion Date
November 2014 (Actual)
Study Completion Date
November 2014 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
University Hospital, Montpellier
4. Oversight
Data Monitoring Committee
No
5. Study Description
Brief Summary
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cystic Fibrosis, Healthy Subjects
Keywords
Cystic Fibrosis, nasal epithelia, mucins
7. Study Design
Primary Purpose
Basic Science
Study Phase
Not Applicable
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
39 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Cystic Fibrosis patients (CF)
Arm Type
Experimental
Arm Description
Tests in vitro after sampling nasal cells of CF patients or controls are the intervention done on these subjects
Arm Title
Control subjects (non CF)
Arm Type
Other
Arm Description
Tests in vitro after sampling nasal cells of CF patients or controls are the intervention done on these subjects
Intervention Type
Other
Intervention Name(s)
Tests in vitro after sampling nasal cells of CF patients or controls
Intervention Description
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.
Primary Outcome Measure Information:
Title
Mature CFTR protein percentage variation after in vitro epigenetics modifiers treatment
Time Frame
24 months
Secondary Outcome Measure Information:
Title
Pro-inflammatory cytokines number variations after epigenetic modifiers treatment
Time Frame
24 months
Title
Anti-inflammatory cytokines number variations after epigenetic modifiers treatment
Time Frame
24 months
Title
Mucin composition variations after epigenetic modifiers treatment
Time Frame
24 months
10. Eligibility
Sex
All
Minimum Age & Unit of Time
12 Years
Accepts Healthy Volunteers
Accepts Healthy Volunteers
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Raphaël RC CHIRON, MD
Organizational Affiliation
Arnaud de Villeneuve CHU Montpellier
Official's Role
Principal Investigator
Facility Information:
Facility Name
Respiratory Diseases Department
City
Montpellier
ZIP/Postal Code
34295
Country
France
12. IPD Sharing Statement
Learn more about this trial
Action of Epigenetic Modifiers in Cystic Fibrosis Treatment
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