Daily Monitoring of Respiratory Symptoms and Spirometry During ETI Treatment in Persons With Cystic Fibrosis.

Sponsor

CHC Montlegia (Other)

Overall Status

Active, not recruiting

CT.gov ID

NCT05599230

Collaborator

(none)

Enrollment

Location

Anticipated Duration (Months)

6.8

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

The goal of this observational study is to provide optimal monitoring and support when initiating ETI treatment in eligible persons with cystic fibrosis (aged 12 y +) and to document on a daily basis, from 72 hours before the start of treatment and then for 14 days

1. FEV1 changes (home spirometry), ii) ii) respiratory symptoms changes, iii) any possible side effects. Through a dedicated electronic platform, these data will be monitored every day by the medical team, which will be fully available for any questions or concerns patients may have.

Condition or Disease	Intervention/Treatment	Phase
Cystic Fibrosis	Drug: Elexacaftor 100 MG / Ivacaftor 75 MG / Tezacaftor 50 MG, 2 tablets each morning + Ivacaftor 150 mg one tablet each evening

Study Design

Study Type:

Observational

Actual Enrollment :

20 participants

Observational Model:

Cohort

Time Perspective:

Prospective

Official Title:

Daily Monitoring of Respiratory Symptoms and Spirometry During ETI Treatment in Persons With Cystic Fibrosis.

Actual Study Start Date :

Sep 1, 2022

Anticipated Primary Completion Date :

Nov 30, 2022

Anticipated Study Completion Date :

Nov 30, 2022

Arms and Interventions

Arm	Intervention/Treatment
CF patients before (3 days) and during (14 days) ETI treatment Patients over 12 years old and eligible will start ETI treatment ( current standart treatment). Morning: Elexacaftor 100 mg, Tezacaftor 50 mg, Ivacaftor 75 mg, 2 tablets Evening: Ivacaftor 150 mg	Drug: Elexacaftor 100 MG / Ivacaftor 75 MG / Tezacaftor 50 MG, 2 tablets each morning + Ivacaftor 150 mg one tablet each evening Patients will daily perform home spirometry and complete a respiratory symptoms score before (3 days) and during (first 14 days) ETI treatment.

Arm

Intervention/Treatment

CF patients before (3 days) and during (14 days) ETI treatment

Patients over 12 years old and eligible will start ETI treatment ( current standart treatment). Morning: Elexacaftor 100 mg, Tezacaftor 50 mg, Ivacaftor 75 mg, 2 tablets Evening: Ivacaftor 150 mg

Drug: Elexacaftor 100 MG / Ivacaftor 75 MG / Tezacaftor 50 MG, 2 tablets each morning + Ivacaftor 150 mg one tablet each evening

Patients will daily perform home spirometry and complete a respiratory symptoms score before (3 days) and during (first 14 days) ETI treatment.

Outcome Measures

Primary Outcome Measures

FEV1 changes from baseline (3 days before treatment) to Day 14 under ETI [17 days]
Daily home spirometry with Spirobank device, between 14H-20H, before nebulization and / or physiotherapy

Secondary Outcome Measures

Respiratory symptoms score changes from baseline (3 days before treatment) to Day 14 under ETI [17 days]
Daily scoring unsing a respiratory questionnaire (8 questions); each item is scored 1 to 5 (worse score: 40)