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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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).
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| 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. |
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
| Measure | Description | Time Frame |
|---|---|---|
| Respiratory symptoms score changes from baseline (3 days before treatment) to Day 14 under ETI | Daily scoring unsing a respiratory questionnaire (8 questions); each item is scored 1 to 5 (worse score: 40) | 17 days |
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Inclusion Criteria:
Exclusion Criteria:
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CF patients aged 12 y or more, carrying at least one copy of F508del mutation, in stable condition, reliably performing spirometry
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| Name | Affiliation | Role |
|---|---|---|
| Patrick Lebecque, PhD | CHC Montlegia | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Lebecque | Liège | 4000 | Belgium |
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| ID | Term |
|---|---|
| D003550 | Cystic Fibrosis |
| D012818 | Signs and Symptoms, Respiratory |
| ID | Term |
|---|---|
| D010182 | Pancreatic Diseases |
| D004066 | Digestive System Diseases |
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D007232 | Infant, Newborn, Diseases |
| D012816 | Signs and Symptoms |
| D013568 | Pathological Conditions, Signs and Symptoms |
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| ID | Term |
|---|---|
| C000629074 | elexacaftor |
| C545203 | ivacaftor |
| C000625213 | tezacaftor |
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