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Surrogate Markers of Response to New Therapies in Cystic Fibrosis Patients (BIO-CFTR)

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ClinicalTrials.gov Identifier: NCT02965326
Recruitment Status : Recruiting
First Posted : November 16, 2016
Last Update Posted : December 5, 2017
Sponsor:
Collaborators:
Association Mucoviscidose-ABCF2
Vaincre la Mucoviscidose
Information provided by (Responsible Party):
Isabelle Sermet-Gaudelus, Hôpital Necker-Enfants Malades

November 11, 2016
November 16, 2016
December 5, 2017
May 2016
March 2020   (Final data collection date for primary outcome measure)
Correlation between biological markers and clinical and physiological outcome [ Time Frame: 6 months ]
Same as current
Complete list of historical versions of study NCT02965326 on ClinicalTrials.gov Archive Site
Correlation between biological markers and clinical and physiological outcome [ Time Frame: 12 months ]
Same as current
Not Provided
Not Provided
 
Surrogate Markers of Response to New Therapies in Cystic Fibrosis Patients
Personalized Therapy of Cystic Fibrosis: Set-up of Response Markers
The purpose of this study is to determine which biological marker, or association of biological markers, best predict clinical response of cystic fibrosis patients to CFTR modulators.
This study is based upon the hypothesis that clinical response of cystic fibrosis patients to CFTR modulators is correlated to in vitro responses to these drugs of epithelial cells derived from the patients, as assessed by CFTR-dependent Chloride secretion. Epithelial cells will be derived either from nasal or rectal epithelia, and consist both of cultured cells and organoids. The drugs tested will be Ivacaftor, or Lumacaftor/Ivacaftor, according to patient's treatment. Results of these assays will be compared with response to treatment at 6 and 12 months, assessed by clinical response and in vivo assay of CFTR function.
Interventional
Not Applicable
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Basic Science
Cystic Fibrosis
Procedure: Nasal swab; rectal biopsy.
Nasal epithelial cells will be obtained by nasal swabs from patients of the three arms; intestinal epithelial cells will be obtained, by rectal biopsy, only from patients treated by CFTR modulators.
  • Cystic fibrosis, treated
    Cystic fibrosis patients treated either by Ivacaftor or by the association Ivacaftor-Lumacaftor
    Intervention: Procedure: Nasal swab; rectal biopsy.
  • Cystic fibrosis, non treated
    Cystic fibrosis patients, non treated by a CFTR modulator
    Intervention: Procedure: Nasal swab; rectal biopsy.
  • Non-Cystic fibrosis
    Patients in whom cystic fibrosis diagnosis has been suspected, but excluded by physiological and genetic investigations
    Intervention: Procedure: Nasal swab; rectal biopsy.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
75
Same as current
October 2020
March 2020   (Final data collection date for primary outcome measure)

Inclusion Criteria:

  • Cystic fibrosis patients treated by CFTR modulators (Ivacaftor or the association Ivacaftor-Lumacaftor)
  • Cystic fibrosis patients non treated by CFTR modulators
  • Patients in whom cystic fibrosis diagnosis has been suspected, but excluded by physiological and genetic investigations

Exclusion Criteria:

  • pregnant or lactating women
  • contraindication to nasal swab
  • contraindication to rectal biopsy
Sexes Eligible for Study: All
Child, Adult, Older Adult
No
Contact: Isabelle Sermet, MD, PhD 33 1 44 49 48 87 isabelle.sermet@aphp.fr
Contact: Jean-Louis Pérignon, MD, PhD jean-louis.perignon@aphp.fr
France
 
 
NCT02965326
ABCF-2016-01
2016-A00309-42 ( Other Identifier: ANSM, French national agency for drug safety )
No
Not Provided
Plan to Share IPD: No
Isabelle Sermet-Gaudelus, Hôpital Necker-Enfants Malades
Hôpital Necker-Enfants Malades
  • Association Mucoviscidose-ABCF2
  • Vaincre la Mucoviscidose
Not Provided
Hôpital Necker-Enfants Malades
December 2017

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP