Real-world Efficacy and Safety of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Modulator Therapy in Adult Patients With Cystic Fibrosis (CF)

Universitair Ziekenhuis Brussel

Status: Not yet enrolling

Conditions: Cystic Fibrosis

Treatments: Diagnostic Test: CFQ-R questionnaire, SNOT-22 questionnaire, fecal elastase measurement

Study type: Interventional

Funder types: Other

Identifiers

NCT05526027

BUN1432021000472

Details and patient eligibility

About

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.

Enrollment: 85 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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85 participants in 1 patient group

ETI

Other group

Description:

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)

Treatment:

Diagnostic Test: CFQ-R questionnaire, SNOT-22 questionnaire, fecal elastase measurement