NCT04301856

Brief 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.

Trial Health

43
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
600

participants targeted

Target at P75+ for all trials

Timeline
Completed

Started Jan 2020

Longer than P75 for all trials

Geographic Reach
1 country

1 active site

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

January 1, 2020

Completed
2 months until next milestone

First Submitted

Initial submission to the registry

February 21, 2020

Completed
18 days until next milestone

First Posted

Study publicly available on registry

March 10, 2020

Completed
5.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 1, 2025

Completed
6 months until next milestone

Study Completion

Last participant's last visit for all outcomes

January 1, 2026

Completed
Last Updated

March 22, 2021

Status Verified

March 1, 2021

Enrollment Period

5.5 years

First QC Date

February 21, 2020

Last Update Submit

March 19, 2021

Conditions

Cystic Fibrosis in Children

Outcome Measures

Primary Outcomes (4)

  • Lung Imaging

    Lung structural injury assessed by Low Dose CT, as part of routine care

    at initiation, as part of national guidelines

  • Lung Imaging

    Lung structural injury assessed by Low Dose CT, as part of routine care

    at 1 year, as part of national guidelines

  • Lung Imaging

    Lung structural injury assessed by Low Dose CT, as part of routine care

    at 3 years, as part of national guidelines

  • Lung Imaging

    Lung structural injury assessed by Low Dose CT, as part of routine care

    at 5 years, as part of national guidelines

Secondary Outcomes (25)

  • weight in kilogrammes

    longitudinal monitoring of assessments carried out as part of routine care during 5 yrs

  • height in meters

    longitudinal monitoring of assessments carried out as part of routine care during 5 yrs

  • pubertal evolution

    longitudinal monitoring of assessments carried out as part of routine care during 5 yrs

  • bronchial infectious exacerbations

    longitudinal monitoring of assessments carried out as part of routine care during 5 yrs

  • Forced Expiratory Volume in 1 second(FEV1)

    longitudinal monitoring of assessments carried out as part of routine care during 5 yrs

  • +20 more secondary outcomes

Study Arms (1)

CF children treated with CFTR modul

Cystic fibrosis patients under 18 years treated with CFTR modulators according to french health recommendations observational cohort study

Drug: CFTR Modulators

Interventions

CFTR ModulatorsDRUG

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

Also known as: Orkambi, Kalydeko...
CF children treated with CFTR modul

Eligibility Criteria

AgeUp to 18 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)
Sampling MethodNon-Probability Sample
Study Population

Children with cystic fibrosis under the age of 18 eligible for CFTR modulator thérapies according to french national recommandations

You may qualify if:

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

You may not qualify if:

  • Patients with cystic fibrosis without indication for CFTR modulator therapy
  • Patients over the age of 18
  • Pregnant or lactating women

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Sermet-Gaudelus Isabelle

Paris, 75015, France

RECRUITING

Related Publications (1)

  • Sermet-Gaudelus I, Letierce A, Berteloot L, Bonnel AS, Chen Y, Makani P, Kelly-Aubert M, Kanoun F, Penalva L, Bouleghem N, Bihouee T, Bui S, Corvol H, Houdouin V, Mittaine M, Tatopoulos A, Weiss L, Wizla N, Kapel N, Bessou A, Tiddens HAWM, Caudri D, Reix P, Marguet C; MODUL-CF study group. Effect of elexacaftor-tezacaftor-ivacaftor on bronchial dilatations in adolescents with cystic fibrosis: a multicentre prospective observational study. Lancet Respir Med. 2026 Jan;14(1):38-48. doi: 10.1016/S2213-2600(25)00248-6. Epub 2025 Oct 22.

    PMID: 41138737DERIVED

Biospecimen

Retention: SAMPLES WITHOUT DNA

Excess bronchial secretions and serum

MeSH Terms

Interventions

lumacaftor, ivacaftor drug combination

Study Officials

  • Isabelle Sermet-Gaudelus, MD PhD

    Société Francaise de la Mucoviscidose

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Stéphane Mazur, PhD

CONTACT

0033427855043stephane.mazur@chu-lyon.fr

Anne-Sophie Bonnel, MD

CONTACT

OO33144494887asbonnel@ch-versailles.fr

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Professor Isabelle Sermet-Gaudelus

Study Record Dates

First Submitted

February 21, 2020

First Posted

March 10, 2020

Study Start

January 1, 2020

Primary Completion

July 1, 2025

Study Completion

January 1, 2026

Last Updated

March 22, 2021

Record last verified: 2021-03

Locations

FR(1)