None listed
Conditions
Brief summary
This trial is evaluating a new imaging method, XV LVAS, which generates a 4D report of lung structure and function. This will be used to evaluate the increase in lung function reported in children with cystic fibrosis when starting the modulator therapy ETI (elexacaftor/tezacaftor/ivacaftor). Standard lung function tests will be compared to XV LVAS scan before and after commencement of ETI treatment. This will demonstrate the safety and clinical utility of XV LVAS scans in children and further characterise the improvement of lung function on commencement of ETI therapy.
Interventions
Patients will be asked to have an x-ray velocimetry (XV) scan, physical exam, questionnaire (cystic fibrosis questionnaire - revised (CFQ-R)) and lung function measures (multiple breath washout, spirometry, lung volumes, diffusing capacity) in addition to their standard care. This will be conducted at Baseline (approximately 1 month pre-ETI commencement) and at 2 months post-ETI).
The intervention for this study is the x-ray velocimetry (XV) scan which is a fluoroscopic chest x-ray of tidal br
Patients will be asked to have an x-ray velocimetry (XV) scan, physical exam, questionnaire (cystic fibrosis questionnaire - revised (CFQ-R)) and lung function measures (multiple breath washout, spirometry, lung volumes, diffusing capacity) in addition to their standard care. This will be conducted at Baseline (approximately 1 month pre-ETI commencement) and at 2 months post-ETI). The intervention for this study is the x-ray velocimetry (XV) scan which is a fluoroscopic chest x-ray of tidal breathing at 5 different angles to create a 3D moving image. The patient needs to lie still for approximately 5 minutes while breathing normally. Participants need a clinically-indicated CT scan to be eligible as this is used to analyse the XV scan to produce the XV LVAS (x-ray velocimetry lung ventilation analysis software) report. The medication ETI (elexacaftor/tezacaftor/ivacaftor) is newly listed on the PBS in Australia for cystic fibrosis patients 6 years and over. ETI dosing is age and weight dependant, with 2 morning and one night tablet taken with a high fat food. Children 6 to 11 years old and less than 30kg: morning tablet (elexacaftor 50 mg/tezacaftor 25 mg/ivacaftor 37.5 mg); night tablet (ivacaftor 75 mg). Children over 30kg or over 12 years to adult: morning tablet (elexacaftor 100 mg/tezacaftor 50 mg/ivacaftor 75 mg); night tablet (ivacaftor 150 mg). Children that take at least one tablet will be recorded as taking ETI, with compliance estimated at follow-up visit by parents (tablets left in weekly blister packs) and medication supplied by the patients as per usual clinical care.
Sponsors
Women's and Children's Hospital
Study design
Allocation
Non-randomised trial
Intervention model
Single group
Primary purpose
Diagnosis
Masking
Open (masking not used)
Eligibility
Sex/Gender
All
Age
3 Years to 18 Years
Healthy volunteers
No
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
Outcome results
None listed