Assessment of regional lung ventilation following the commencement of ETI (elexacaftor/tezacaftor/ivacaftor) treatment in children with cystic fibrosis

Not Applicable

Recruiting

• Conditions: cystic fibrosis; Respiratory - Other respiratory disorders / diseases

• Registration Number: ACTRN12624000146594
• Lead Sponsor: Women's and Children's Hospital

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2024-02-15
• Last Update Posted: 9 September 2024

Recruitment & Eligibility

• Status: Recruiting
• Sex: All
• Target Recruitment: 20

Inclusion Criteria

Children with cystic fibrosis aged 5-18 years planning to start ETI (elexacaftor/tezacaftor/ivacaftor) treatment in the next 3 months
- must have a clinically indicated CT chest completed prior to screening or scheduled within the next 4 weeks
- must be willing to attempt all lung function testing and complete one of: spirometry, lung volumes, diffusing capacity, multiple breath washout

Exclusion Criteria

- pulmonary exacerbation within 4 weeks of planned screening visit
- currently receiving mechanical ventilation, intensive or other critical care
- contraindication to ionising radiation
- inability to remain still for periods of 30 seconds for each of the 5 XV image acquisitions during an XV scan
- use of ETI (elexacaftor/tezacaftor/ivacaftor) before Screening procedures are complete

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Changes in lung ventilation heterogeneity (VH) as measured by x-ray velocimetry lung ventilation analysis software (XV-LVAS) following commencement of ETI (elexacaftor/tezacaftor/ivacaftor) in children with cystic fibrosis[x-ray velocimetry (XV) scan and resulting x-ray velocimetry lung ventilation analysis software (XV LVAS) report Baseline (1-month pre-ETI commencement) to Follow-up (2 months post-ETI commencement)];Changes in regional lung ventilation (RLV) as measured by x-ray velocimetry lung ventilation analysis software (XV-LVAS) following commencement of ETI (elexacaftor/tezacaftor/ivacaftor) in children with cystic fibrosis[x-ray velocimetry (XV) scan and resulting x-ray velocimetry lung ventilation analysis software (XV LVAS) report Baseline (1-month pre-ETI commencement) to Follow-up (2 months post-ETI commencement)]

Secondary Outcome Measures

Name	Time	Method
Determine whether XV provides an accurate clinical assessment of lung function compared to traditional lung function testing by assessing correlations between lung function and XV LVAS measures (Composite outcome).[Correlations between XV scan measures (ventilation heterogeneity, regional lung ventilation) and the gold standard lung function measure FEV1 (forced expiratory volume at 1 second)<br>Correlations between XV scan measures (ventilation heterogeneity, regional lung ventilation) and the gold standard lung function measure FVC (forced vital capacity) Baseline (1-month pre-ETI commencement)<br>Follow-up (2 months post-ETI commencement)];Change in Quality of Life measurements[As determined by the Cystic Fibrosis Questionnaire - Revised (CFQ-R) Baseline (1-month pre-ETI commencement)<br>Follow-up (2 months post-ETI commencement)]