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

Hôpital Necker-Enfants Malades

Status: Enrolling

Conditions: Cystic Fibrosis

Treatments: Procedure: Nasal swab; rectal biopsy.

Study type: Interventional

Funder types: Other

Identifiers

NCT02965326

ABCF-2016-01

2016-A00309-42 (Other Identifier)

Details and patient eligibility

About

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.

Full 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.

Enrollment: 75 estimated patients

Sex: All

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Basic Science

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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75 participants in 3 patient groups

Cystic fibrosis, treated

Other group

Description:

Cystic fibrosis patients treated either by Ivacaftor or by the association Ivacaftor-Lumacaftor

Treatment:

Procedure: Nasal swab; rectal biopsy.

Cystic fibrosis, non treated

Other group

Description:

Cystic fibrosis patients, non treated by a CFTR modulator

Treatment:

Procedure: Nasal swab; rectal biopsy.

Non-Cystic fibrosis

Other group

Description:

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

Treatment:

Procedure: Nasal swab; rectal biopsy.