Clinical trial NCT05526027: Real-world Efficacy and Safety of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Modulator Therapy in Adult Patients With Cystic Fibrosis (CF)
Official title: Real-world Efficacy and Safety of CFTR Modulator Therapy in Adult Patients With Cystic Fibrosis
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: NA
Conditions: Cystic Fibrosis
Lead sponsor: Universitair Ziekenhuis Brussel
Sponsor class: OTHER
First posted: 2022-09-02
Enrollment: 85
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

Eligibility criteria: Inclusion Criteria:

* eligible for ETI (i.e. age above 18 years and CFTR genotype F508del/any) based on reimbursement criteria in Belgium

Exclusion Criteria:

* inability to perform lung function testing

Primary outcomes:
measure: percent predicted forced expiratory volume in 1 second (ppFEV1)
timeFrame: 2 years
measure: Lung clearance index (LCI)
timeFrame: 2 years
measure: Fractional excretion of nitric oxide (FeNO)
timeFrame: 2 years
measure: CFQ-R questionnaire
timeFrame: 2 years
measure: fecal elastase (microgram per gram of feces)
timeFrame: 2 years
measure: the amount of participants experiencing a treatment-related adverse event
timeFrame: 2 years
measure: annual acute exacerbation rate
timeFrame: 2 years
measure: body mass index (BMI)
timeFrame: 2 years
Secondary outcomes:
measure: SNOT-22 questionnaire
timeFrame: 2 years
measure: GAD-7 questionnaire
timeFrame: 2 years
measure: PHQ-9 questionnaire
timeFrame: 2 years
measure: aerobic culture on sputum or cough swab sample
timeFrame: 2 years
measure: dosage of pancreatic enzyme replacement therapy
timeFrame: 2 years
Other outcomes:
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