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

Cystic Fibrosis Clinical Trial

Official title: Real-world Efficacy and Safety of CFTR Modulator Therapy in Adult Patients With Cystic Fibrosis

Status Not yet recruiting

Clinical Trial 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.

Clinical Trial 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. ;

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis

• NCT number: NCT05526027
• Study type: Interventional
• Source: Universitair Ziekenhuis Brussel
• Contact: Stefanie Vincken, M.D.
• Phone: +32 2 477 68 41
• Email: stefanie.vincken@uzbrussel.be
• Status: Not yet recruiting
• Phase: N/A
• Start date: September 1, 2022
• Completion date: December 31, 2024