MODUCO: Effectiveness of CFTR Modulators According to Co-therapy

Sponsor

Hospices Civils de Lyon (Other)

Overall Status

Not yet recruiting

CT.gov ID

NCT05663255

Collaborator

(none)

1,990

Enrollment

Anticipated Duration (Months)

Study Details

Study Description

Brief Summary

Unless CFTR modulators are highly effective, the introduction of CFTR (Cystic fibrosis transmembrane conductance regulator) modulators could lead to concomitant reduction or discontinuation of respiratory co-therapies in real-life. Such reduction/discontinuation of respiratory co-therapies could lead to an overall decrease of the effectiveness of CF care.

MODUCO study aims: 1) to compare the clinical effectiveness on lung function and pulmonary exacerbation of CFTR modulator during the year of initiation, according to level of co-therapy among CF patients; 2) to describe the nature and level of respiratory co-therapies (azithromycin, RhDNase, inhaled antibiotics) in the year before the initiation of CFTR modulator; 3) to describe the changes in respiratory co-therapies during the first and the second year following the initiation of CFTR modulator and compare between the two CFTR modulators; 4) to describe adherence to CFTR modulator during the first and the second year following its initiation; 5) to study the association between the nature of respiratory co-therapies and adherence to CFTR modulator during the first and the second year following its initiation.

A national population-based comparative effectiveness study will be conducted, based on retrospective analysis of clinical and prescription data of the French CF registry linked with the French national health data system (SNDS).

Condition or Disease	Intervention/Treatment	Phase
Cystic Fibrosis	Drug: Discontinuation of respiratory co-therapy (azithromycin, RhDNase, or inhaled antibiotics)

Study Design

Study Type:

Observational

Anticipated Enrollment :

1990 participants

Observational Model:

Cohort

Time Perspective:

Retrospective

Official Title:

Is Effectiveness of CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) Modulators Impacted by Co-therapy? A Population-based Comparative Effectiveness Study Using Data From French Cystic Fibrosis Registry Linked to French National Healthcare Insurance Database

Anticipated Study Start Date :

Mar 1, 2023

Anticipated Primary Completion Date :

Sep 1, 2023

Anticipated Study Completion Date :

Dec 31, 2024

Arms and Interventions

Arm	Intervention/Treatment
Exposed group Patients treated with CFTR modulator with at least the discontinuation of one respiratory co-therapy (azithromycin, RhDNase, or inhaled antibiotics) during the year of initiation (A0) of CFTR modulator.	Drug: Discontinuation of respiratory co-therapy (azithromycin, RhDNase, or inhaled antibiotics) Discontinuation or reduction of at least one respiratory co-therapy (azithromycin, RhDNase, or inhaled antibiotics)
Control group Patients treated with CFTR modulator without any discontinuation of respiratory co-therapy (azithromycin, RhDNase, inhaled antibiotics) during the year of initiation (A0) of CFTR modulator.

Arm

Intervention/Treatment

Exposed group

Patients treated with CFTR modulator with at least the discontinuation of one respiratory co-therapy (azithromycin, RhDNase, or inhaled antibiotics) during the year of initiation (A0) of CFTR modulator.

Drug: Discontinuation of respiratory co-therapy (azithromycin, RhDNase, or inhaled antibiotics)

Discontinuation or reduction of at least one respiratory co-therapy (azithromycin, RhDNase, or inhaled antibiotics)

Control group

Patients treated with CFTR modulator without any discontinuation of respiratory co-therapy (azithromycin, RhDNase, inhaled antibiotics) during the year of initiation (A0) of CFTR modulator.

Outcome Measures

Primary Outcome Measures

Lung function [two years]
Change in the best forced expiratory volume in 1 second (FEV1) % predicted, measured during the year (A+1) compared to the year of initiation (A0) of CFTR modulator. The best FEV1 of the year is collected annually in the French CF Registry.

Eligibility Criteria

Criteria

Ages Eligible for Study:

6 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

- CF patients aged 6 years and more,
Treated at least 6 months by ivacaftor or lumacaftor/ivacaftor or elexacaftor/tezacaftor/ivacaftor,
Included in the French CF registry,
For whom probabilistic matching with the SNDS database has succeeded.