NCT02965326

Surrogate Markers of Response to New Therapies in Cystic Fibrosis Patients

Personalized Therapy of Cystic Fibrosis: Set-up of Response Markers

• Status: Recruiting
• Phase: N/A
• Study type: Interventional
• Enrollment: 75 (estimated)
• Sponsor: Hôpital Necker-Enfants Malades · Academic / Other
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

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.

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.

Conditions

• Cystic Fibrosis

Interventions

Type	Name	Description
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.

Timeline

• Start date: 2016-05-01
• Primary completion: 2026-03-01
• Completion: 2026-10-01
• First posted: 2016-11-16
• Last updated: 2024-03-12

Locations

1 site across 1 country: France