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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

Brief Summary:
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.

Condition or disease Intervention/treatment Phase
Cystic Fibrosis Procedure: Nasal swab; rectal biopsy. Not Applicable

Detailed Description:
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.

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 75 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Basic Science
Official Title: Personalized Therapy of Cystic Fibrosis: Set-up of Response Markers
Actual Study Start Date : May 2016
Estimated Primary Completion Date : March 2020
Estimated Study Completion Date : October 2020

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis

Arm Intervention/treatment
Cystic fibrosis, treated
Cystic fibrosis patients treated either by Ivacaftor or by the association Ivacaftor-Lumacaftor
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, non treated
Cystic fibrosis patients, non treated by a CFTR modulator
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.

Non-Cystic fibrosis
Patients in whom cystic fibrosis diagnosis has been suspected, but excluded by physiological and genetic investigations
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.




Primary Outcome Measures :
  1. Correlation between biological markers and clinical and physiological outcome [ Time Frame: 6 months ]

Secondary Outcome Measures :
  1. Correlation between biological markers and clinical and physiological outcome [ Time Frame: 12 months ]


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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02965326


Contacts
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

Locations
France
Necker Hospital Recruiting
Paris, France, 75014
Contact: SERMET Isabelle, Professor    01 44 49 48 87    isabelle.sermet@nck.aphp.fr   
Principal Investigator: SERMET Isabelle, Professor         
Sponsors and Collaborators
Hôpital Necker-Enfants Malades
Association Mucoviscidose-ABCF2
Vaincre la Mucoviscidose

Publications:

Responsible Party: Isabelle Sermet-Gaudelus, Professor, Hôpital Necker-Enfants Malades
ClinicalTrials.gov Identifier: NCT02965326     History of Changes
Other Study ID Numbers: ABCF-2016-01
2016-A00309-42 ( Other Identifier: ANSM, French national agency for drug safety )
First Posted: November 16, 2016    Key Record Dates
Last Update Posted: December 5, 2017
Last Verified: December 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Additional relevant MeSH terms:
Fibrosis
Cystic Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases