Trial Review

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Trial registered on ANZCTR


Registration number
ACTRN12623000595617
Ethics application status
Approved
Date submitted
11/05/2023
Date registered
31/05/2023
Date last updated
31/05/2023
Date data sharing statement initially provided
31/05/2023
Type of registration
Retrospectively registered

Titles & IDs
Public title
Elexacaftor/tezacaftor/ivacaftor (ETI) Observations Substudy within BEAT CF (Bayesian Evidence Adaptive Treatment of Cystic Fibrosis)
Scientific title
BEAT CF PEx ETI Observations Substudy - collecting information to
assess the effect of commencement of ETI therapy, in children aged 6-11 years old, on outcomes including lung function, nutrition, and (CF) related quality of life.
Secondary ID [1] 309642 0
None
Universal Trial Number (UTN)
Trial acronym
Linked study record
This is a substudy of ACTRN12621000638831

Health condition
Health condition(s) or problem(s) studied:
Cystic Fibrosis 329971 0
Condition category
Condition code
Human Genetics and Inherited Disorders 326880 326880 0 0
Cystic fibrosis

Intervention/exposure
Study type
Observational
Patient registry
False
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
Elexacaftor/tezacaftor/ivacaftor (ETI) is a novel therapy for cystic fibrosis (CF) that has recently been introduced into the 6-11 year old age group in Australia. Clinical trials sponsored by the ETI product manufacturer have indicated that ETI is likely to result in improved lung function and health related quality of life. However, there is a knowledge gap regarding the impact of ETI when used in the real world. This study seeks to address this knowledge by assessing the impact of ETI, using the BEAT CF platform, when this therapy is commenced in children aged 6-11 years old.

Participants in this substudy must already be participating in the BEAT CF study (already registered in ACTRN12621000638831). Eligible participants from the BEAT CF study are those aged 6-11 years old, who are commencing ETI therapy.

Participating in this substudy involves doing spirometry, having your weight and height measured and your parents filling out a health related quality of life questionnaire (CFQ-R) at the same times as if you were not in this substudy (your regular clinic appointments), as well as having these measurements about an extra 2-3 times. The timing for these measurements are prior to starting ETI therapy, and approximately 14 days, 1 month, 3 months, and 6 months after.
Intervention code [1] 326070 0
Not applicable
Comparator / control treatment
No control group
Control group
Uncontrolled

Outcomes
Primary outcome [1] 334722 0
Forced Expiratory Volume (FEV1) measured using spiromtery will be used to assess lung function.
Timepoint [1] 334722 0
Baseline, then 14 days, 1 month, 3 months and 6 months (primary timepoint) after first dose of ETI therapy
Primary outcome [2] 334723 0
Nutrition which will be measured by change in weight and body mass index (weight divided by the square of height).
Participant weight will predominately be measured using scales in the CF clinic. If the participant is not due to attend the CF clinic when this substudy requires a weight measurement scales from the participant's home will be used to measure their weight.
Participant height will be measured using a stadiometer in the CF clinic. If the participant is not due to attend the CF clinic when this substudy requires a height measurement, height will be measured at home using a tape measure.
Any at home measurements will be reported to the BEAT CF site coordinator at the CF clinic.
Timepoint [2] 334723 0
Baseline, then 14 days, 1 month, 3 months and 6 months (primary timepoint) after first dose of ETI therapy.
Primary outcome [3] 334724 0
Quality of Life measured by the Cystic Fibrosis Questionnaire - Revised
Timepoint [3] 334724 0
Baseline, then 14 days, 1 month, 3 months and 6 months (primary timepoint) after first dose of ETI therapy.
Secondary outcome [1] 421870 0
ETI Associated side-effects, as documented in the medical record. Known/possible adverse reactions/events are headache, upper respiratory tract infection (common cold), stomach ache (abdominal pain) and diarrhoea.
Timepoint [1] 421870 0
As they occur during the 6 month time period following first dose of ETI therapy.

Eligibility
Key inclusion criteria
1. Be Enrolled in the BEAT CF PEx Cohort
2. Be Aged 6 to 11 years old
3. Have provided written informed consent (participant or legally responsible guardian) for participation in the ETI Observations substudy
4. Plan to commenceing ETI therapy within the next 6 months
Minimum age
6 Years
Maximum age
11 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Responsible Clinician deems enrolment is not in the patient’s best interest.

Study design
Purpose
Screening
Duration
Longitudinal
Selection
Defined population
Timing
Prospective
Statistical methods / analysis
The sub-study is descriptive.
We will report the mean (also the distribution, median and 25th to 75th percentile) of the absolute change in ppFEV1 from baseline to the nominated timepoints. Likewise, we will describe the change in weight, BMI and CFQ-R. The 6 month timepoint is considered of primary importance. Where numbers permit, we will report this separately by each year of age, sex, genotype, pancreatic function, and by the presence of pseudomonas at baseline. We will make qualitative comparisons with the same outcomes reported in the clinical trials.

Recruitment
Recruitment status
Recruiting
Date of first participant enrolment
Anticipated
Actual
8/05/2023
Date of last participant enrolment
Anticipated
24/05/2024
Actual
Date of last data collection
Anticipated
6/12/2024
Actual
Sample size
Target
210
Accrual to date
3
Final
Recruitment in Australia
Recruitment state(s)
NSW,SA,WA,VIC
Recruitment hospital [1] 24703 0
The Royal Childrens Hospital - Parkville
Recruitment hospital [2] 24704 0
The Children's Hospital at Westmead - Westmead
Recruitment hospital [3] 24705 0
Sydney Children's Hospital - Randwick
Recruitment hospital [4] 24706 0
John Hunter Children's Hospital - New Lambton
Recruitment hospital [5] 24707 0
Womens and Childrens Hospital - North Adelaide
Recruitment hospital [6] 24708 0
Perth Children's Hospital - Nedlands
Recruitment postcode(s) [1] 40324 0
2031 - Randwick
Recruitment postcode(s) [2] 40323 0
2145 - Westmead
Recruitment postcode(s) [3] 40325 0
2305 - New Lambton
Recruitment postcode(s) [4] 40322 0
3052 - Parkville
Recruitment postcode(s) [5] 40326 0
5006 - North Adelaide
Recruitment postcode(s) [6] 40327 0
6009 - Nedlands

Funding & Sponsors
Funding source category [1] 313827 0
University
Name [1] 313827 0
University of Sydney
Country [1] 313827 0
Australia
Funding source category [2] 313880 0
Charities/Societies/Foundations
Name [2] 313880 0
Murdoch Children's Research Institute
Country [2] 313880 0
Australia
Primary sponsor type
University
Name
University of Sydney
Address
Research Portfolio
F23 Administration Building
The University of Sydney NSW 2006
Country
Australia
Secondary sponsor category [1] 315662 0
None
Name [1] 315662 0
Address [1] 315662 0
Country [1] 315662 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 312984 0
Child and Adolescent Health Services Human Research Ethics Committee
Ethics committee address [1] 312984 0
Perth Children’s Hospital, 15 Hospital Avenue, Nedlands WA 6009
Ethics committee country [1] 312984 0
Australia
Date submitted for ethics approval [1] 312984 0
15/02/2023
Approval date [1] 312984 0
27/04/2023
Ethics approval number [1] 312984 0
RGS0000001265

Summary
Brief summary
In this study we want to look at the impact starting on the new treatment for cystic fibrosis (CF) - Elexacaftor/tezacaftor/ivacaftor (ETI – also known as Trikafta) has on children ages 6-11 years in Australia. In the clinical trials conducted before a drug is approved for sale, there are various controls and measures around which children receive the treatment and what is measured. The results of these clinical trials showed that ETI is likely to improve lung function and health related quality of life. This study is about finding out if the same improved lung function and quality of life is seen in the real world, where there are less controls around which children can take the medication.
We are adding this study to the BEAT CF project as the data we need to work out the impact of Trikafta in the real world is the same type of data that is being collected in BEAT CF.
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 126582 0
Prof Tom Snelling
Address 126582 0
University of Sydney
Edward Ford Building
Room 123
Camperdown NSW 2006
Country 126582 0
Australia
Phone 126582 0
+61 2 9563 6886
Fax 126582 0
Email 126582 0
tom.snelling@sydney.edu.au
Contact person for public queries
Name 126583 0
Dr Shivanthan Shanthikumar
Address 126583 0
Dr Shivanthan Shanthikumar
MCRI
Royal Children's Hospital
Flemington Road
Parkville Victoria 3052 Australia
Country 126583 0
Australia
Phone 126583 0
+61 3 9345 5522
Fax 126583 0
Email 126583 0
shivanthan.shanthikumar@rch.org.au
Contact person for scientific queries
Name 126584 0
Dr Shivanthan Shanthikumar
Address 126584 0
Dr Shivanthan Shanthikumar
MCRI
Royal Children's Hospital
Flemington Road
Parkville Victoria 3052 Australia
Country 126584 0
Australia
Phone 126584 0
+61 3 9345 5522
Fax 126584 0
Email 126584 0
shivanthan.shanthikumar@rch.org.au

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

Documents added manually
No documents have been uploaded by study researchers.

Documents added automatically
No additional documents have been identified.