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

NCT ID: NCT05599230

Last Updated: 2022-10-31

Basic Information

• Recruitment Status: UNKNOWN
• Total Enrollment: 20 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2022-09-01
• Study Completion Date: 2022-11-30

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 i) i) 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.

Conditions

Cystic Fibrosis

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: PROSPECTIVE

Study Groups

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

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

Intervention Type: DRUG

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

Interventions

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.

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

• Cystic fibrosis diagnosis
• At least 12 years old
• Carrier of at least one copy of F508del mutation
• Ability to perform reliable and reproducible spirometry
• Medical and psychological stability
• written consent
• owning a smartphone

Exclusion Criteria

• Lung transplant
• FEV1 > 120 % pr (%GLI) at the inclusion test
• Pulmonary exacerbation

• Minimum Eligible Age: 12 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

CHC Montlegia

OTHER

Sponsor Role: lead

Responsible Party

Lebecque Patrick

Principal investigator, Clinical Professor

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Patrick Lebecque, PhD

Role: PRINCIPAL_INVESTIGATOR

CHC Montlegia

Locations

Lebecque

Liège, , Belgium

Countries

Belgium

Other Identifiers

ML001

Identifier Type: -

Identifier Source: org_study_id