Effectiveness of CFTR Modulators According to Co-therapy

NCT05663255 | Unknown

• 1 other identifier: 69HCL20_0066
• Study Type: observational
• Target: 1,990
• Locations: 0 countries
• Sites: N/A

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).

Conditions

Cystic Fibrosis

Keywords

Cystic Fibrosis; CFTR modulators; co-therapy; SNDS

Outcome Measures

Primary Outcomes (1)

• Lung function

  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.

  two years

Study Arms (2)

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)

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.

Interventions

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

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

Exposed group

Eligibility Criteria

• Age: 6 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Exhaustive on the recruitment of the all french CF patients meeting with eligibility criteria.

You may qualify if:

• \- 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.

You may not qualify if:

• none

Sponsors & Collaborators

• Hospices Civils de Lyon: lead

MeSH Terms

Conditions

Cystic Fibrosis

Interventions

Azithromycin; dornase alfa

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Intervention Hierarchy (Ancestors)

Erythromycin; Macrolides; Polyketides; Lactones; Organic Chemicals

Central Study Contacts

Isabelle DURIEU, MD

CONTACT

0033 (0)4 78 86 13 52; isabelle.durieu@chu-lyon.fr

Marie VIPREY, MD

CONTACT

0033 (0)4 72 11 51 39; marie.viprey@chu-lyon.fr

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 15, 2022
• First Posted: December 23, 2022
• Study Start: March 1, 2023
• Primary Completion: September 1, 2023
• Study Completion: December 31, 2024
• Last Updated: December 23, 2022

Record last verified: 2022-12