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

U

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)

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

Trial contacts and locations

0

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Central trial contact

Stefanie Vincken, M.D.; Eef Vanderhelst, M.D. Ph.D.

Data sourced from clinicaltrials.gov

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