Action of Epigenetic Modifiers in Cystic Fibrosis Treatment

NCT01883284 | Completed

• 1 other identifier: 8870
• Study Type: interventional
• Target: 39
• Locations: 1 country
• Sites: 1

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.

Conditions

Cystic Fibrosis; Healthy Subjects

Keywords

Cystic Fibrosis; nasal epithelia; mucins

Outcome Measures

Primary Outcomes (1)

• Mature CFTR protein percentage variation after in vitro epigenetics modifiers treatment

  24 months

Secondary Outcomes (3)

• 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

Study Arms (2)

Cystic Fibrosis patients (CF)

EXPERIMENTAL

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

Control subjects (non CF)

OTHER

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

Interventions

Tests in vitro after sampling nasal cells of CF patients or controls OTHER

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.

Control subjects (non CF); Cystic Fibrosis patients (CF)

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• informed consent
• benefit from disease insurance regimen
• men and women
• severe CF mutations
• age superior or equal to 12
• age superior or equal to 18
• no smoker (for 5 years)

You may not qualify if:

• participation to an other interventionnal study
• law protected subject
• pregnant and breast fooding
• Xylocaine hypersensibility
• Porphyria
• severe hepatic failure
• Epilepsy
• Severe cardiac failure
• local anesthesic contra indication
• respiratory disease
• cystic fibrosis
• acute infection \< 6 weeks
• on treatment
• antibiotic treatment \< 3 months

Sponsors & Collaborators

• University Hospital, Montpellier: lead

Study Sites (1)

Respiratory Diseases Department

Montpellier, 34295, France

Location

MeSH Terms

Conditions

Cystic Fibrosis

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Study Officials

• Raphaël RC CHIRON, MD

  Arnaud de Villeneuve CHU Montpellier

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: BASIC SCIENCE
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 13, 2013
• First Posted: June 21, 2013
• Study Start: January 1, 2012
• Primary Completion: November 1, 2014
• Study Completion: November 1, 2014
• Last Updated: December 3, 2014

Record last verified: 2014-12

Locations

FR (1)