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In this trial real-world data on the safety (side effects and medication interactions) and efficacy (evolution of lung function testing, chronic bacterial airway infection, quality of life and endo- and exocrine pancreatic function) will be collected in adult people with cystic fibrosis (pwCF) eligible for elexacaftor-tezacaftor-ivacaftor (ETI) up until 2 years after the start of this therapy.
Full description
PwCF group A: CFTR-modulator-naive pwCF eligible for ETI (based on age and CFTR genotype)
PwCF group B: pwCF already on CFTR modulating therapy (i.e. ivacaftor-lumacaftor or ivacaftor-tezacaftor) and switching to ETI.
Patients in both groups will undergo these examinations:
Physical examination (including weight) Anamnesis for current and recent medication use (including dosage of pancreatic enzymes) and for acute respiratory exacerbations Lung function testing including spiometry, multiple breath washout testing and fractional exhaled nitric oxide Blood sampling: liver function tests, creatine kinase, albumin, PT, red and white blood cell count, platelet count Sputum/cough swab sampling fecal elastase measurement Cystic fibrosis questionnaire-revised (CFQ-R) questionnaire Patient health questionnaire-9 (PHQ-9) questionnaire General anxiety disorder-7 (GAD-7) questionnaire Sino-nasal outcome test-22 (SNOT-22) questionnaire
These will be performed at baseline (prior to the start of ETI, on the same day of start of ETI), and every 3 months (+/- 7 days) thereafter. A blood sample will also be performed 14 (+/- 7 days) days after start of ETI (for safety). Fecal elastase measurement will only be performed at baseline if not available in the patient's medical record, and only 6 months after start of ETI.
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85 participants in 1 patient group
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Stefanie Vincken, M.D.; Eef Vanderhelst, M.D. Ph.D.
Data sourced from clinicaltrials.gov
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