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Trial registered on ANZCTR


Registration number
ACTRN12620000114943p
Ethics application status
Submitted, not yet approved
Date submitted
12/12/2019
Date registered
7/02/2020
Date last updated
20/03/2020
Date data sharing statement initially provided
7/02/2020
Type of registration
Prospectively registered

Titles & IDs
Public title
Can RESPiratory hospital Admissions in children with cerebral palsy be reduced?: A feasibility randomized Controlled Trial (RESP-ACT)
Scientific title
Individualized assessment-based interventions for reducing respiratory hospital admissions in children with cerebral palsy: A feasibility randomized controlled trial
Secondary ID [1] 299936 0
None
Universal Trial Number (UTN)
Trial acronym
RESP-ACT - Feasibility
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Respiratory disease 315374 0
Cerebral palsy 315375 0
Condition category
Condition code
Respiratory 313676 313676 0 0
Other respiratory disorders / diseases
Neurological 313677 313677 0 0
Other neurological disorders
Physical Medicine / Rehabilitation 314017 314017 0 0
Physiotherapy
Physical Medicine / Rehabilitation 314018 314018 0 0
Speech therapy

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
The intervention group will receive a comprehensive respiratory assessment and an individualized action plan. Participants in the intervention group will be offered the interventions outlined in the “Consensus statement for prevention and management of respiratory disease in young people with cerebral palsy: Standards of care.” At the time of preparing this protocol, the consensus statement has not yet been published, and so the interventions will be described in detail here.

Children in the intervention group will receive an initial assessment that will include physiotherapy, speech pathology, and medical components. This assessment is expected to take a total of 5-6 hours. A physiotherapist will assess: respiratory signs and symptoms; breathing pattern; chest expansion; spinal deformity; chest tone; strength and function of cough; respiratory rate; and blood oxygen. A speech pathologist will use the following assessments: Eating and Drinking Ability Classification System (EDACS) or Dysphagia Outcome Severity Scale (DOSS); oromotor function; videofluoroscopy; and swallowing assessment. A respiratory registrar will do the medical assessment, including: nutritional status; reflux; seizure control; sialorrhea; and constipation/delayed gastric emptying.

Within 2 weeks of the assessment, the multidisciplinary treatment team will create an individualized plan for each child in the intervention group based on the results of this assessment. The children in this study will have complex medical issues and risks and so the plan for each child will be unique and complex, and will depend on clinicians’ clinical judgements and families’ preferences for treatments. Therefore, the study protocol cannot prescribe the frequency or intensity of treatment for any particular child. However, the general principles (summarized from the consensus statement) are as follows:
• In children who are aspirating: referral to speech pathologist to assess swallowing safety; consideration of modified textures and postural support.
• In children with epilepsy: monitoring of seizures and/or referral to epilepsy specialists for management of uncontrolled seizures.
• In children with gastro-oesophageal reflux disease: management of reflux.
• In children who are drooling: review of medications that may cause drooling if the child is aspirating saliva; consideration of management with oral medications, salivary gland Botulinum Toxin, or salivary gland surgery.
• In children with a history of apnoea, snoring or on examination there are large tonsils/swollen turbinates: referral to Ear, Nose and Throat specialists for assessment and management of upper airway obstruction.
• In children with asthma: management of asthma and monitoring of the effectiveness of medications.
• In children with respiratory illness: antibiotics (using antibiotic guidelines); saliva management; optimization of general health (including assessment of nutritional status, reflux, and risk of aspiration when unwell); dysphagia assessment and management plan to facilitate safe and functional feeding when unwell.
• In children with respiratory illness and a productive cough: referral to physiotherapists for airway clearance regimes.
• In children with symptoms of airways disease or sputum retention: referral to a respiratory physiotherapist and consideration of suction.
• In all children: Physiotherapists educate carers ways to maintain clear airways, identify ineffective cough, consider positioning and regular chest physiotherapy. Optimization of chest wall mobility to prevent restrictive lung disease, including maximizing physical activity and minimizing immobility; managing movement disorders; assessment of kyphosis, kyphoscoliosis, and lordoscoliosis, and use of postural plan; and where surgery for scoliosis is under consideration, a multidisciplinary team will evaluate risks and benefits. Assessment and optimization of nutrition. Optimization of respiratory function, physical activity, and fitness. Regular dental care. Vaccination against influenza.

The intervention will commence within a week of the time when the individualized plan is agreed upon. The intervention group will receive up to one hour of weekly individualized face-to-face physiotherapy and/or speech therapy review and advice for the first 4 weeks. After that, there will be ongoing physiotherapy (up to 1.5 hours/fortnight), speech pathology (up to 1 hour/fortnight), and/or medical (up to ½ hour/fortnight) management according to need from existing clinical staff at the children’s hospital and community-based therapy service. These sessions will continue for up to 12 months (the study period). During the individual sessions, the physiotherapist will provide education regarding the symptoms and signs of a respiratory exacerbation. A nurse will coordinate care and provide individual education and training for suction and ventilation technology where required. Duration, intensity, and location (hospital or home) of the interventions will be individualized. The clinicians administering the interventions will record all assessments and interventions, time taken, and adherence on a standard data collection sheet.
Intervention code [1] 316205 0
Treatment: Other
Comparator / control treatment
The control group will receive their regular treatment from treating practitioners as usual, with no additional assessments unless the treating team arranges these.
Control group
Active

Outcomes
Primary outcome [1] 322103 0
Implementation of the intervention assessed by number of participants enrolled within 2 months of recruitment.
Timepoint [1] 322103 0
End of recruitment period
Primary outcome [2] 322104 0
Implementation of the intervention assessed by number of participants randomized to a group.
Timepoint [2] 322104 0
End of recruitment period
Primary outcome [3] 322108 0
Implementation of the intervention assessed by number of participants providing data on the primary outcomes at 3 months
Timepoint [3] 322108 0
3 months after commencement of intervention
Secondary outcome [1] 377353 0
Implementation of the intervention assessed by number of participants providing data on the primary outcomes at 6 months
Timepoint [1] 377353 0
6 months after commencement of intervention
Secondary outcome [2] 378468 0
Implementation of the intervention assessed by number of participants providing data on the primary outcomes at 12 months
Timepoint [2] 378468 0
End of intervention period (12 months after commencement of intervention period)
Secondary outcome [3] 378469 0
Implementation of the intervention assessed by number of participants to whom the prescribed intervention was delivered).
Timepoint [3] 378469 0
End of intervention period (12 months after commencement of intervention period)
Secondary outcome [4] 378470 0
Practicality of the intervention assessed by adherence to treatment regimens by participants in the intervention group (as recorded by treating clinicians).
Timepoint [4] 378470 0
Each time an intervention is prescribed or delivered; end of trial.
Secondary outcome [5] 378471 0
Practicality of the intervention assessed by costs of interventions and assessments (staff hours and materials, as recorded by treating clinicians).
Timepoint [5] 378471 0
Each time an intervention is prescribed or delivered; end of trial.
Secondary outcome [6] 378472 0
Practicality of the intervention assessed by end-of-trial 30- to 60-minute face-to-face or phone interviews with families regarding contextual factors associated with delivery of the intervention, perceived benefits and difficulties, and perception of impact on service delivery demands).
Timepoint [6] 378472 0
End of trial
Secondary outcome [7] 378473 0
Practicality of the intervention assessed by end-of-trial 30- to 60-minute face-to-face interviews with clinicians regarding contextual factors associated with delivery of the intervention, perceived benefits and difficulties, and perception of impact on service delivery demands).
Timepoint [7] 378473 0
End of trial
Secondary outcome [8] 378474 0
Efficacy of the intervention assessed by number of days in hospital with respiratory illness (as reported online by families).
Timepoint [8] 378474 0
Fortnightly during the 12-month intervention period.
Secondary outcome [9] 378475 0
Efficacy of the intervention assessed by number of days in Intensive Care Unit with respiratory illness during the past fortnight (as reported online by families).
Timepoint [9] 378475 0
Fortnightly during the 12-month intervention period.
Secondary outcome [10] 378476 0
Efficacy of the intervention assessed by number of visits to General Practitioner for respiratory illness during the past fortnight (as reported online by families).
Timepoint [10] 378476 0
Fortnightly during the 12-month intervention period.
Secondary outcome [11] 378477 0
Efficacy of the intervention assessed by number of courses of antibiotics for respiratory illness during the past fortnight (as reported online by families).
Timepoint [11] 378477 0
Fortnightly during the 12-month intervention period.
Secondary outcome [12] 378478 0
Efficacy of the intervention assessed by number of exacerbations during the past fortnight (as reported online by families).
Timepoint [12] 378478 0
Fortnightly during the 12-month intervention period.
Secondary outcome [13] 378479 0
Efficacy of the intervention assessed by number of days missed from school for respiratory illness during the past fortnight (as reported online by families).
Timepoint [13] 378479 0
Fortnightly during the 12-month intervention period.
Secondary outcome [14] 378480 0
Efficacy of the intervention assessed by number of work days that a parent/carer missed work to care of the child with respiratory illness during the past fortnight (as reported online by families).
Timepoint [14] 378480 0
Fortnightly during the 12-month intervention period.
Secondary outcome [15] 378481 0
Efficacy of the intervention assessed by individual quality of life (CPQOL).
Timepoint [15] 378481 0
Beginning and end of the intervention period.
Secondary outcome [16] 378483 0
Efficacy of the intervention assessed by family quality of life (Beach Center on Disability Family Quality of Life survey).
Timepoint [16] 378483 0
Beginning and end of the intervention period.
Secondary outcome [17] 378484 0
Acceptability of the intervention assessed by approximately 30- to 60-minute face-to-face or phone interviews with parents of the children in the intervention group.
Timepoint [17] 378484 0
Immediately after completion of intervention
Secondary outcome [18] 378485 0
Acceptability of the intervention assessed by approximately 30- to 60-minute face-to-face interviews with staff involved in delivery of the intervention.
Timepoint [18] 378485 0
End of trial period.
Secondary outcome [19] 379300 0
Implementation of the intervention assessed by number of participants who adhered to the prescribed intervention.
Timepoint [19] 379300 0
End of trial period

Eligibility
Key inclusion criteria
• Cerebral palsy,
• 0 to 12 years of age inclusive, and
• at risk of respiratory hospitalization, as determined by the respiratory risk checklist developed by our research team from our 5-year prospective cohort design.

The respiratory risk checklist is a 19-item tool. It contains questions about the child’s frequency of respiratory symptoms, medical attention for respiratory illnesses in the previous year, method of nutritional intake, and presence of co-morbidities. All questions require only a tick in a box. The checklist is easily completed within 5 minutes.

The intervention is for children with CP at risk of at risk of respiratory hospitalizations. During the trial, some data regarding the children will be provided by the parents and clinicians of these children.
Minimum age
0 Years
Maximum age
12 Years
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
• On continuous home oxygen.
• On ventilatory support (Continuous Positive Airway Pressure (CPAP)/non-invasive ventilation).
• Tracheostomy.
• Enrolled in paediatric palliative care programme with restrictions or guidelines about active management other than end-of-life care.
• Living outside the metropolitan area.
• Family requires a professional translator or interpreter to participate in the trial.
• There are concerns that the home environment may not safe for clinicians to conduct the intervention.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
The random numbers will be generated by a team member who is not involved in deciding whether the children were eligible to enter the trial. The group allocations will then be put into a series of sealed opaque envelopes with only a number (indicating order of recruitment) on the outside.
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Participants will be randomly assigned to the intervention and control groups using simple randomization with computer-generated random numbers.
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?


The people assessing the outcomes
Intervention assignment
Parallel
Other design features
Phase
Not Applicable
Type of endpoint(s)
Efficacy
Statistical methods / analysis
As this is a feasibility study, no power calculation has been used to determine the sample size. The present study will supply data that can be used in a sample size calculation for a multicentre RCT.

Descriptive statistics (mean and standard deviation) with 95% confidence intervals will be calculated for all the outcomes, and presented for each group separately. These will be used to determine power for a larger RCT. For outcome measures with highly skewed distributions, medians, interquartile ranges and ranges will be reported. No significance testing between groups is needed to analyse these feasibility objectives. No interim analyses or subgroup analyses are planned due to the small sample size of this feasibility trial.

Recruitment
Recruitment status
Not yet recruiting
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
WA
Recruitment hospital [1] 15350 0
Perth Children's Hospital - Nedlands
Recruitment postcode(s) [1] 28661 0
6009 - Nedlands

Funding & Sponsors
Funding source category [1] 304397 0
Government body
Name [1] 304397 0
Government of Western Australia, Department of Health
Address [1] 304397 0
Level 2, C Block, 189 Royal Street,
East Perth WA 6004
Country [1] 304397 0
Australia
Primary sponsor type
Other
Name
Ability Centre
Address
106 Bradford Street, Coolbinia WA 6050
Country
Australia
Secondary sponsor category [1] 304656 0
Hospital
Name [1] 304656 0
Perth Children's Hospital
Address [1] 304656 0
15 Hospital Ave, Nedlands WA 6009
Country [1] 304656 0
Australia

Ethics approval
Ethics application status
Submitted, not yet approved
Ethics committee name [1] 304834 0
The Child and Adolescent Health Service Human Research Ethics Committee
Ethics committee address [1] 304834 0
15 Hospital Ave, Nedlands WA 6009
Ethics committee country [1] 304834 0
Australia
Date submitted for ethics approval [1] 304834 0
21/01/2020
Approval date [1] 304834 0
Ethics approval number [1] 304834 0

Summary
Brief summary
Cerebral palsy (CP) is the commonest physical disability in childhood. Respiratory illness is the leading cause of death for children with CP, and is responsible for more multi-day hospital admissions than any other cause. Children with CP admitted to hospital for respiratory illness stay in hospital, on average, 2.5 times longer than other children, and two in five are re-admitted the following year.

At present, there is no coordinated approach to preventing respiratory illness in children with CP. Children with respiratory illness are usually not referred for specialized care until their respiratory illness is advanced and often life-threatening. The BREATHE-CP (Better REspiratory and Airway Treatment and HEalth in Cerebral Palsy) research team have determined the early risk factors for respiratory illness in children with CP so that they can be treated much earlier. We have also systematically reviewed the research literature to determine the most effective treatments for preventing respiratory disease. However, there is very little research evidence. Treatment studies are urgently needed. We have also asked researchers and clinicians worldwide with expertise in respiratory health in CP what treatment approaches they would recommend, and developed a consensus statement for management of respiratory illness in children with CP.

This study will trial this recommended approach to management of respiratory illness in children with CP. Treatments will be individualized for children with CP at risk of respiratory illness to find out whether they are feasible, acceptable, and likely to be effective by comparison with current standard treatment. If the trial is successful, we will apply for funding to do a large-scale trial across several hospitals to improve respiratory health and quality of life for children with CP at risk of respiratory illness and reduce healthcare costs.
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 98362 0
Dr Marie Blackmore
Address 98362 0
Ability Centre
106 Bradford Street
Coolbinia WA 6050
Country 98362 0
Australia
Phone 98362 0
+61 892673867
Fax 98362 0
Email 98362 0
marie.blackmore@abilitycentre.com.au
Contact person for public queries
Name 98363 0
Dr Marie Blackmore
Address 98363 0
Ability Centre
106 Bradford Street
Coolbinia WA 6050
Country 98363 0
Australia
Phone 98363 0
+61 892673867
Fax 98363 0
Email 98363 0
marie.blackmore@abilitycentre.com.au
Contact person for scientific queries
Name 98364 0
Dr Marie Blackmore
Address 98364 0
Ability Centre
106 Bradford Street
Coolbinia WA 6050
Country 98364 0
Australia
Phone 98364 0
+61 892673867
Fax 98364 0
Email 98364 0
marie.blackmore@abilitycentre.com.au

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
De-identified individual quantitative data on efficacy of the intervention, including measures of respiratory outcomes and quality of life.
When will data be available (start and end dates)?
Immediately following publication, no end date
Available to whom?
Only researchers who provide a methodologically sound proposal on a case-by-case basis at the discretion of Primary Sponsor
Available for what types of analyses?
Individual participant data meta-analysis
How or where can data be obtained?
access subject to approvals by Principal Investigator (Marie.Blackmore@abilitycentre.com.au) or delegate (Noula.Gibson@abilitycentre.com.au).
What supporting documents are/will be available?
Study protocol
Informed consent form
Ethical approval
How or where can supporting documents be obtained?
Type [1] 5954 0
Study protocol
Citation [1] 5954 0
Link [1] 5954 0
Email [1] 5954 0
marie.blackmore@abilitycentre.com.au
Other [1] 5954 0
Alternatively Email: noula.gibson@abilitycentre.com.au
Attachment [1] 5954 0
Type [2] 5955 0
Informed consent form
Citation [2] 5955 0
Link [2] 5955 0
Email [2] 5955 0
marie.blackmore@abilitycentre.com.au
Other [2] 5955 0
Alternatively Email: noula.gibson@abilitycentre.com.au
Attachment [2] 5955 0
Type [3] 5956 0
Ethical approval
Citation [3] 5956 0
Link [3] 5956 0
Email [3] 5956 0
marie.blackmore@abilitycentre.com.au
Other [3] 5956 0
Alternatively Email: noula.gibson@abilitycentre.com.au
Attachment [3] 5956 0
Summary results
No Results