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

NCT05599230 | Unknown FDA Drug

• 1 other identifier: ML001
• Study Type: observational
• Target: 20
• Locations: 1 country
• Sites: 1

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

Keywords

elexacaftor; time course; FEV1; Respiratory symptoms

Outcome Measures

Primary Outcomes (1)

• FEV1 changes from baseline (3 days before treatment) to Day 14 under ETI

  Daily home spirometry with Spirobank device, between 14H-20H, before nebulization and / or physiotherapy

  17 days

Secondary Outcomes (1)

• Respiratory symptoms score changes from baseline (3 days before treatment) to Day 14 under ETI

  17 days

Study Arms (1)

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

Interventions

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

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

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

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

CF patients aged 12 y or more, carrying at least one copy of F508del mutation, in stable condition, reliably performing spirometry

You may qualify if:

• 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

You may not qualify if:

• Lung transplant
• Pulmonary exacerbation

Sponsors & Collaborators

• CHC Montlegia: lead

Study Sites (1)

Lebecque

Liège, 4000, Belgium

Location

MeSH Terms

Conditions

Cystic Fibrosis; Signs and Symptoms, Respiratory

Interventions

elexacaftor; ivacaftor; tezacaftor

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; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Study Officials

• Patrick Lebecque, PhD

  CHC Montlegia

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal investigator, Clinical Professor

Study Record Dates

• First Submitted: October 21, 2022
• First Posted: October 31, 2022
• Study Start: September 1, 2022
• Primary Completion: November 30, 2022
• Study Completion: November 30, 2022
• Last Updated: October 31, 2022

Record last verified: 2022-10

Locations

BE (1)