Real-world Efficacy and Safety of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Modulator Therapy in Adult Patients With Cystic Fibrosis (CF)
- Conditions
- Cystic Fibrosis
- Interventions
- Diagnostic Test: CFQ-R questionnaire, SNOT-22 questionnaire, fecal elastase measurement
- Registration Number
- NCT05526027
- Lead Sponsor
- Universitair Ziekenhuis Brussel
- Brief Summary
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.
- Detailed 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.
Recruitment & Eligibility
- Status
- NOT_YET_RECRUITING
- Sex
- All
- Target Recruitment
- 85
- eligible for ETI (i.e. age above 18 years and CFTR genotype F508del/any) based on reimbursement criteria in Belgium
- inability to perform lung function testing
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description ETI CFQ-R questionnaire, SNOT-22 questionnaire, fecal elastase measurement pwCF which are CFTR-modulator naive and pwCF previously treated with a CFTR-modulator (i.e. tezacaftor-ivacaftor or lumacaftor-ivacaftor) will undergo standard-of-care examinations as well as examinations in the context of this trial (i.e. CFQ-R, PHQ-9, GAD-7 and SNOT-22 questionnaires, fecal elastase measurement)
- Primary Outcome Measures
Name Time Method body mass index (BMI) 2 years percent predicted forced expiratory volume in 1 second (ppFEV1) 2 years fecal elastase (microgram per gram of feces) 2 years the concentration of elastase (a pancreatic enzyme) in feces before and 6 months after start of CFTR modulating treatment, as a surrogate measure for pancreatic function
the amount of participants experiencing a treatment-related adverse event 2 years CFQ-R questionnaire 2 years Lung clearance index (LCI) 2 years Fractional excretion of nitric oxide (FeNO) 2 years annual acute exacerbation rate 2 years
- Secondary Outcome Measures
Name Time Method PHQ-9 questionnaire 2 years dosage of pancreatic enzyme replacement therapy 2 years GAD-7 questionnaire 2 years SNOT-22 questionnaire 2 years aerobic culture on sputum or cough swab sample 2 years