Evaluation of the Response to CFTR Modulators in Patients With Cystic Fibrosis Less Than 18 Years of Age

Status: Recruiting
Location: See location...
Intervention Type: Drug
Study Type: Observational
SUMMARY

CFTR modulators should improve the prognosis of Cystic Fibrosis. Identifying patients under the age of 18 responding to CFTR modulators as well as detecting possible toxicity is an important medical objective given the potential side effects and the high cost of these molecules. This observational follow-up cohort study is carried out as part of routine care. The main objective is to assess the evolution of pulmonary structural impairment by low-dose CF scan at the end of the first year of CFTR modulator therapy. The secondary objectives are to evaluate structural impairment at low dose scan at 3 years and 5 years of CFTR modulator treatment, the evolution of respiratory functional parameters, growth, puberty, lung infection, sweat test, quality of life and pancreatic function, as well as tolerance of modulators including liver toxicity.

Eligibility
Participation Requirements
Sex: All
Maximum Age: 18
Healthy Volunteers: f
View:

• Children with cystic fibrosis under the age of 18 under CFTR modulator therapy

Locations
Other Locations
France
Sermet-Gaudelus Isabelle
RECRUITING
Paris
Contact Information
Primary
Stéphane Mazur, PhD
stephane.mazur@chu-lyon.fr
0033427855043
Backup
Anne-Sophie Bonnel, MD
asbonnel@ch-versailles.fr
OO33144494887
Time Frame
Start Date: 2020-01-01
Estimated Completion Date: 2026-01-01
Participants
Target number of participants: 600
Treatments
CF children treated with CFTR modul
Cystic fibrosis patients under 18 years treated with CFTR modulators according to french health recommendations observational cohort study
Related Therapeutic Areas
Sponsors
Leads: Societe Francaise de la Mucoviscidose

This content was sourced from clinicaltrials.gov