Effectiveness of CFTR Modulators According to Co-therapy

Cystic Fibrosis Clinical Trial

— MODUCO  

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

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT05663255
• Other study ID #: 69HCL20_0066
• Status: Not yet recruiting
• Start date: March 1, 2023
• Est. completion date: December 31, 2024

Study information

• Verified date: December 2022
• Source: Hospices Civils de Lyon
• Contact: Isabelle DURIEU, MD
• Phone: 0033 (0)4 78 86 13 52
• Email: isabelle.durieu[@]chu-lyon.fr
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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).

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 1990
• Est. completion date: December 31, 2024
• Est. primary completion date: September 1, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 6 Years and older

Eligibility

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.

Exclusion Criteria:

• none

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis

Intervention

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)

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Hospices Civils de Lyon

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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