MedTrace Logo

Response to CFTR Modulators in CF Patients Under 18 Years

NCT04301856 · Status: RECRUITING · Type: OBSERVATIONAL · Enrollment: 600

Last updated 2021-03-22

No results posted yet for this study

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.

Conditions

  • Cystic Fibrosis in Children

Interventions

DRUG

CFTR Modulators

CFTR modulators as recommended in routine care by the french health authorities

Sponsors & Collaborators

  • Societe Francaise de la Mucoviscidose

    lead OTHER

Principal Investigators

  • Isabelle Sermet-Gaudelus, MD PhD · Société Francaise de la Mucoviscidose

Eligibility

Max Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-01-01
Primary Completion
2025-07-01
Completion
2026-01-01

Countries

  • France

Study Locations

More Related Trials

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04301856 on ClinicalTrials.gov