Trial Review

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Trial registered on ANZCTR


Registration number
ACTRN12621000638831
Ethics application status
Approved
Date submitted
17/03/2021
Date registered
28/05/2021
Date last updated
3/04/2024
Date data sharing statement initially provided
28/05/2021
Type of registration
Retrospectively registered

Titles & IDs
Public title
BEAT CF platform: A digital infrastructure and data collection tool to evaluate treatments for pulmonary exacerbations in children and adults with Cystic Fibrosis.
Scientific title
BEAT CF platform: A digital infrastructure and data collection tool to evaluate treatments for pulmonary exacerbations in children and adults with Cystic Fibrosis.
Secondary ID [1] 303283 0
Nil known
Universal Trial Number (UTN)
Trial acronym
BEAT CF
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Cystic Fibrosis 320486 0
Condition category
Condition code
Human Genetics and Inherited Disorders 318362 318362 0 0
Cystic fibrosis
Respiratory 319287 319287 0 0
Other respiratory disorders / diseases

Intervention/exposure
Study type
Observational
Patient registry
False
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
The BEAT CF platform is a data collection instrument that will collect health and medical data from consented participants’ medical records. There are no specified interventions or exposures for participants who enrol in the BEAT CF platform. All information that is entered into the BEAT CF platform database - both treatment and diagnostic - is gathered as a part of standard clinical care for these patients. Participants will be requested to partake in completion of quality of life questionnaires in accordance with their regular clinic visit schedule, which is expected to be approximately every 3 months. The quality of life data is in addition to data gathered from routine standard clinical care.

Patients with Cystic fibrosis commonly experience Pulmonary exacerbations, or flare-ups of the lung disease. These exacerbations or flare ups often require hospitalisation for intensive treatment including with intravenous antibiotics.

Pulmonary exacerbations remain an important driver of progressive loss of lung function and premature death. Up to 25% of CF patients do not recover their baseline lung function, typically measured as the forced expiratory volume in one second (FEV1), after an exacerbation. Preventing loss of lung function with each exacerbation may be key to improving survival.

Antibiotics are a cornerstone of treatment for pulmonary exacerbations, but most antibiotic regimens are only informed by old, underpowered, or poor-quality trials. There is no consensus on the treatment of pulmonary exacerbations of CF. Across Australia, CF centres use a range of approaches and antibiotic regimens. Because preservation of lung function is important for extending life and quality of life, there is a need to determine the most effective empirical treatments of exacerbations.

As part of the BEAT CF platform, information about any intravenous antibiotics and other treatments like steroids or disease modulators will be captured, in order to describe the treatment of CF pulmonary exacerbations, their outcomes, as well as any clinical, demographic, and other lifestyle factors which might also influence these outcomes. All data relating to treatments and outcomes of exacerbations required to be entered into the BEAT CF platform will be sourced from participant's medical records.
Participants in the BEAT CF platform will be requested to complete a second type of quality of life questionnaire at the time of any hospitalisation for treatment of pulmonary exacerbations. This questionnaire, called the CRISS, is validated for participants aged 12 years and over.

There will be no experimental treatments administered as part of the BEAT CF platform. Across Australia, CF centres use a range of approaches and antibiotic regimens. All participants will be treated according to their centre’s standard of care. The BEAT CF platform provides the central infrastructure to simultaneously evaluate the range of treatment strategies used in CF across Australia.
The BEAT CF platform will establish effective and accurate collection of data to enable the future nesting of platform clinical trial(s). The BEAT CF platform is intended to continue perpetually. Participants who enrol in the BEAT CF platform will continue to contribute data to the platform unless they specifically withdraw their consent to participation.
Intervention code [1] 320017 0
Diagnosis / Prognosis
Comparator / control treatment
No control group
Control group
Uncontrolled

Outcomes
Primary outcome [1] 326835 0
The absolute change in the FEV1pp measured by spirometry
Timepoint [1] 326835 0
Post enrolment to the BEAT CF platform, during each hospitalisation for treatment of a pulmonary exacerbation - One week after commencement of intensive therapy for that exacerbation.
Secondary outcome [1] 392840 0
1. The relative change in the FEV1pp, measured by spirometry and defined as the proportional change (expressed as a percentage) in the FEV1pp at day 7-14 from day 0.
Timepoint [1] 392840 0
Post enrolment to the BEAT CF platform, during each hospitalisation for treatment of a pulmonary exacerbation - Approximately 1 week after commencement of intensive therapy for treatment of a that exacerbation.
Secondary outcome [2] 392841 0
2. Absolute change in the FEV1pp measured by spirometry
Timepoint [2] 392841 0
Post enrolment to the BEAT CF platform, during each hospitalisation for treatment of a pulmonary exacerbation - Approximately 7 (5-10) days, 14 days (14-30) days, 30 (30-60) days, 60 (60-90) days, and 180 (180-240) days after commencement of intensive therapy of that exacerbation
Secondary outcome [3] 392842 0
4. Time taken for FEV1pp measured by spirometry to return to greater than or equal to 90% of the baseline FEV1pp measured by spirometry.
Timepoint [3] 392842 0
Post enrolment to the BEAT CF platform, during each hospitalisation for treatment of a pulmonary exacerbation - Approximately 1 week, 2 weeks, 4 weeks, 8 weeks, and 24 weeks after commencement of intensive therapy for treatment of that exacerbation.
Secondary outcome [4] 392843 0
5. The absolute change in the severity of symptoms of respiratory infection as measured by the Chronic Respiratory Infection Symptom Score (CRISS)
Timepoint [4] 392843 0
Post enrolment to the BEAT CF platform, during each hospitalisation for treatment of a pulmonary exacerbation - approximately 7 days and 14 days after commencement of intensive therapy for treatment of that exacerbation.
Secondary outcome [5] 392844 0
6. The time to next exacerbation measured as the time (in days) from commencement of intensive therapy to the next commencement of intensive therapy after a period of no intensive therapy of > 7 days. This will be determined by data for hospitalisations for lung exacerbations reported in the BEAT CF platform database.
Timepoint [5] 392844 0
Following a post BEAT CF platform enrolment hospitalisation for treatment of a lung exacerbation, and a period of no intensive therapy of greater than 7 days after discharge from that exacerbation.
Secondary outcome [6] 392845 0
7. Any interruption of planned therapy before 7 days (168hr) and before 14 days (336hr) after commencement of intensive therapy, owing to intolerable effects presumed by the treating clinician to be attributable to therapy. Assessment will be made on data reported in the BEAT CF platform database
Timepoint [6] 392845 0
Post enrolment in the BEAT CF platform, during hospitalisation for a lung exacerbation: after commencement of intensive therapy in that hospitalisation, for up to 7 days (168hr) and 14 days (336hr) from commencement of the intensive therapy
Secondary outcome [7] 392848 0
8. Health related quality of life, as scored by The Cystic fibrosis Quality of Life questionnaire, revised, (CFQR).
Timepoint [7] 392848 0
For participants over 6 years of age, every 12 weeks post enrolment in the BEAT CF platform until the participant withdraws consent for continuing or the platform is closed.
Secondary outcome [8] 392849 0
9. A new detection of any strain of gram negative bacteria with in vitro resistance to any aminoglycoside, fluoroquinolone, antipseudomonal penicillin (including beta-lactam/beta-lactamase inhibitor combination), antipseudomonal cephalosporin, or carbapenem assessed by sputum culture or medical record review.
Timepoint [8] 392849 0
Any time post enrolment to the BEAT CF platform and for the 2 years prior to enrolment to BEAT CF.
Secondary outcome [9] 392850 0
10. Any new onset of Clostridium difficile associated diarrhoea assessed by faecal sample post enrolment to the BEAT CF platform, or by medical record review for the 1 year prior to BEAT CF enrolment.
Timepoint [9] 392850 0
Any time post enrolment to the BEAT CF platform and for the 1 years prior to enrolment to BEAT CF.
Secondary outcome [10] 394744 0
3. Relative change in the FEV1pp measured by spirometry
Timepoint [10] 394744 0
Post enrolment to the BEAT CF platform, during each hospitalisation for treatment of a pulmonary exacerbation - Approximately 2 weeks, 4 weeks, 8 weeks, and 24 weeks after commencement of intensive therapy for treatment of that exacerbation.

Eligibility
Key inclusion criteria
To be eligible to participate in the platform, a patient must meet the following criteria:
1. Diagnosis of CF by:
a. Sweat chloride level >60mmol/L, or
b. Sweat chloride > 40mmol/L and two mutations of the CF gene, or
c. Two known pathogenic mutations of the CF gene

2. Informed consent obtained from the patient or their legal representative
Minimum age
No limit
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
History of lung transplantation

Study design
Purpose
Natural history
Duration
Longitudinal
Selection
Defined population
Timing
Both
Statistical methods / analysis
Individual demographics and baseline clinical characteristics (at enrolment and at every commencement of intensive therapy for a lung exacerbation) will be reported for all participants.
The proportion of participants with the primary endpoint measured, and any lost to follow up, will be assessed and reported. Subgroup analyses will be performed by the treatments prescribed, and by the prespecified patient strata:
1. Patients with high baseline lung function (FEV1pp greater than or equal to 70%) in the preceding 12 months (or unable to perform spirometry due to young age) and no known Pseudomonas aeruginosa colonisation in the preceding 2 years (720 days)
2. Patients with high baseline lung function (FEV1pp greater than or equal to 70%) in the preceding 12 months (or unable to perform spirometery due to young age) and known Pseudomonas aeruginosa colonisation in the preceding 2 years (720 days)
3. Patients with low or moderate baseline lung function (FEV1pp less than 70%) in the preceding 12 months, regardless of Pseudomonas aeruginosa colonisation status
All other subgroup analyses will be reported as post hoc.
Categorical variables will be summarised at each level as a frequency and proportion with a 95% confidence or credible interval. Cell frequencies below five participants will be reported as “<5” to ensure individual confidentiality. Continuous variables will be summarised as mean and standard deviation for symmetric distributions and median and interquartile range (IQR) for asymmetric distributions.
Categorical data and time-to-event endpoints will be assessed using standard statistical analytical approaches including Chi squared, Fisher’s exact, Student t test and Mann-Whitney tests as appropriate. Predictors of treatment outcomes will be assessed using logistic, linear and Cox regression for binary, continuous and time-to-event endpoints respectively. All summary statistics will be calculated in R version 3.5.3 or later version, which is provided open-source by the R Foundation for Statistical Computing.

Recruitment
Recruitment status
Recruiting
Date of first participant enrolment
Anticipated
Actual
14/10/2020
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
10000
Accrual to date
935
Final
Recruitment in Australia
Recruitment state(s)
NSW,QLD,SA,WA,VIC
Recruitment hospital [1] 18905 0
Sydney Children's Hospital - Randwick
Recruitment hospital [2] 18906 0
The Children's Hospital at Westmead - Westmead
Recruitment hospital [3] 18907 0
Perth Children's Hospital - Nedlands
Recruitment hospital [4] 18908 0
Sir Charles Gairdner Hospital - Nedlands
Recruitment hospital [5] 18909 0
John Hunter Hospital - New Lambton
Recruitment hospital [6] 18910 0
John Hunter Children's Hospital - New Lambton
Recruitment hospital [7] 18911 0
The Royal Childrens Hospital - Parkville
Recruitment hospital [8] 18912 0
Queensland Children's Hospital - South Brisbane
Recruitment hospital [9] 18914 0
Womens and Childrens Hospital - North Adelaide
Recruitment hospital [10] 22359 0
Royal Prince Alfred Hospital - Camperdown
Recruitment hospital [11] 25657 0
Mater Hospital Brisbane - South Brisbane
Recruitment hospital [12] 26356 0
The Prince Charles Hospital - Chermside
Recruitment postcode(s) [1] 33412 0
2031 - Randwick
Recruitment postcode(s) [2] 33413 0
2145 - Westmead
Recruitment postcode(s) [3] 33414 0
6009 - Nedlands
Recruitment postcode(s) [4] 33415 0
2305 - New Lambton
Recruitment postcode(s) [5] 33416 0
3052 - Parkville
Recruitment postcode(s) [6] 33417 0
4101 - South Brisbane
Recruitment postcode(s) [7] 33419 0
5006 - North Adelaide
Recruitment postcode(s) [8] 37521 0
2050 - Camperdown
Recruitment postcode(s) [9] 41480 0
4101 - South Brisbane
Recruitment postcode(s) [10] 42328 0
4032 - Chermside

Funding & Sponsors
Funding source category [1] 307696 0
Government body
Name [1] 307696 0
Commonwealth of Australia as represented by the Department of Health
Country [1] 307696 0
Australia
Primary sponsor type
University
Name
University of Sydney
Address
Level 3, F23 Administration Building,
Corner of Eastern Avenue and City
Road, The University of Sydney,
NSW 2006 Australia
Country
Australia
Secondary sponsor category [1] 308396 0
None
Name [1] 308396 0
Address [1] 308396 0
Country [1] 308396 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 307729 0
Child and Adolescent Health Services Human Research Ethics Committee
Ethics committee address [1] 307729 0
Ethics committee country [1] 307729 0
Australia
Date submitted for ethics approval [1] 307729 0
22/05/2019
Approval date [1] 307729 0
06/08/2020
Ethics approval number [1] 307729 0

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 108302 0
Prof Thomas Snelling
Address 108302 0
University of Sydney
Faculty of Medicine and Health
School of Public Health
Edward Ford Building
Camperdown Campus
NSW 2006
Country 108302 0
Australia
Phone 108302 0
+61 2 9563 6886
Fax 108302 0
Email 108302 0
[email protected]
Contact person for public queries
Name 108303 0
Thomas Snelling
Address 108303 0
University of Sydney
Faculty of Medicine and Health
School of Public Health
Edward Ford Building
Camperdown Campus
NSW 2006
Country 108303 0
Australia
Phone 108303 0
+61 2 9563 6886
Fax 108303 0
Email 108303 0
[email protected]
Contact person for scientific queries
Name 108304 0
Thomas Snelling
Address 108304 0
University of Sydney
Faculty of Medicine and Health
School of Public Health
Edward Ford Building
Camperdown Campus
NSW 2006
Country 108304 0
Australia
Phone 108304 0
+61 2 9563 6886
Fax 108304 0
Email 108304 0
[email protected]

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
SourceTitleYear of PublicationDOI
EmbaseBEAT CF pulmonary exacerbations core protocol for evaluating the management of pulmonary exacerbations in people with cystic fibrosis.2023https://dx.doi.org/10.1186/s13063-023-07076-8
N.B. These documents automatically identified may not have been verified by the study sponsor.