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Trial registered on ANZCTR


Registration number
ACTRN12624001257550
Ethics application status
Approved
Date submitted
20/09/2024
Date registered
14/10/2024
Date last updated
14/10/2024
Date data sharing statement initially provided
14/10/2024
Type of registration
Prospectively registered

Titles & IDs
Public title
A novel treatment approach targeting respiratory muscle strength in children who aspirate.
Scientific title
Prevention of aspiration lung disease in children – A novel treatment approach targeting respiratory muscle strength
Secondary ID [1] 313022 0
Nil Known
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Aspiration 335236 0
Lung Disease 335237 0
Bronchiectasis 335238 0
Condition category
Condition code
Respiratory 331778 331778 0 0
Other respiratory disorders / diseases

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Randomised control trial using respiratory muscle strength training (RMST).
An Expiratory muscle strength training (EMST) 150 or 75 device will be given to participants to use with their parent/carer supervision.

For the intervention arm, an EMST 150 or 75 device will be utilised whereby short duration, isometric contractions of the expiratory muscles generate the pressure to open the device’s release valve. During training, the device is adjusted to progressively increase the resistance through the measurement of a participant’s own Mean Expiratory Pressure (MEP) and setting the device to a percentage of that MEP for training (e.g. 75% of MEP).

If children are unable to perform MEP, then the device resistance will be adjusted in accordance with their clinical ability to perform the manoeuvre. In the sham intervention (control) arm children will receive a plastic mask (spacer mask), with no resistance and perform the same manoeuvre as the RMST.


Children will undergo at least one guided session for one hour provided by the researcher, either face to face or via telehealth whichever is preferred, and then complete 6 weeks of RMST comprising of 5 sets of 5 repetitions per day (25/day) for 5 days each week.

Carers will be given a diary to record RMST sessions over this 6 week period. A review session will take place at Week-3 and Week-7 in the respiratory outpatient clinic or via teleheatlh, for 30 minutes, with the researcher who will record progress. Treatment adherence, adverse events, and any information from respiratory related hospital presentations or admissions will be recorded.
Repeat Videofluoroscopy (VFSS), Pulmonary Function Tests (PFT) (where possible) and clinic review of both groups (RMST and control) will occur at Week-7 and another clinical respiratory review at 6 months post randomisation which will assess for adverse events and any information from respiratory related hospital presentations or admissions.
Intervention code [1] 329570 0
Treatment: Devices
Intervention code [2] 329631 0
Treatment: Other
Comparator / control treatment
Control treatment using "Sham" device (mask).
This will be the same as the protocol (above) for the EMST device, however using a face mask with no resistance. The same procedure will apply to this group.
Control group
Placebo

Outcomes
Primary outcome [1] 339430 0
Difference in change (Week-7 minus Baseline) in Penetration Aspiration Score (reflecting aspiration severity) between the two arms (Respiratory Muscle Strength Training vs control), assessed at Week-7.
Timepoint [1] 339430 0
Baseline and Week 7 post intervention commemcement.
Secondary outcome [1] 439962 0
Respiratory muscle strength where measures were able to be taken.
Respiratory muscle strength measures will be taken where possible as a composite secondary outcome.
Timepoint [1] 439962 0
Baseline, week 7 and 6 months post intervention commencement.
Secondary outcome [2] 439963 0
Respiratory exacerbations per child at 6 months
Timepoint [2] 439963 0
Baseline, 6 months post intervention commencement.
Secondary outcome [3] 439964 0
Management assessed at 6 months including change in fluid thickener level and/or food modification.
Change in fluid thickener level or food modification will be a composite secondary outcome.
Timepoint [3] 439964 0
6 months post intervention commencement.
Secondary outcome [4] 439965 0
Score of PedsQL
Score of Feeding Swallowing Impact Survey (FS-IS)
Timepoint [4] 439965 0
Baseline, 6 months post intervention commencement.
Secondary outcome [5] 439966 0
Score of Feeding Swallowing Impact Scale
Timepoint [5] 439966 0
Baseline, 6 months post intervention commencement.

Eligibility
Key inclusion criteria
(a) children aged 2-18 years with known aspiration (PAS score 6-8), or at risk of aspiration as determined by VFSS (PAS score 4-5), or placed on modified fluids due to high risk (PAS score 2-3), within the last 3 years and
(b) able to try and perform at least one pulmonary function test (CPF, MIP, MEP, SNIP or spirometry). If children are unable to perform the pulmonary function test, but able to perform the RMST manoeuvre, they will also be included
(c) able to perform RMST
Minimum age
2 Years
Maximum age
18 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
(a) no informed consent
(b) unable to cooperate with pulmonary function tests or RMST
• Inadequate cognitive function to understand instruction
• Medically unstable- eg, those requiring hospitalisation or where the treating medical team do not feel they are able to perform testing.
(c) aspiration caused by unrepaired larynx abnormality e.g. laryngeal cleft
(d) tracheostomy present.

Study design
Purpose of the study
Prevention
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Central randomisation by computer
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Permuted block randomisation
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s


Intervention assignment
Other design features
Phase
Not Applicable
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Not yet recruiting
Date of first participant enrolment
Anticipated
13/01/2025
Actual
Date of last participant enrolment
Anticipated
13/07/2026
Actual
Date of last data collection
Anticipated
29/03/2027
Actual
Sample size
Target
74
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
QLD
Recruitment hospital [1] 27136 0
Queensland Children's Hospital - South Brisbane
Recruitment postcode(s) [1] 43213 0
4101 - South Brisbane

Funding & Sponsors
Funding source category [1] 317463 0
Government body
Name [1] 317463 0
National Health and Medical Research Council
Country [1] 317463 0
Australia
Funding source category [2] 317464 0
Government body
Name [2] 317464 0
Children's Health QLD
Country [2] 317464 0
Australia
Primary sponsor type
University
Name
University of Queensland
Address
Country
Australia
Secondary sponsor category [1] 319753 0
Government body
Name [1] 319753 0
Chldren's Health QLD
Address [1] 319753 0
Country [1] 319753 0
Australia

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 316178 0
Children’s Health Queensland Hospital and Health Service Human Research Ethics Committee
Ethics committee address [1] 316178 0
http://www.childrens.health.qld.gov.au/research/human-research-ethics-committee
Ethics committee country [1] 316178 0
Australia
Date submitted for ethics approval [1] 316178 0
12/02/2024
Approval date [1] 316178 0
19/06/2024
Ethics approval number [1] 316178 0
HREC/23/QCHQ/105292

Summary
Brief summary
Impaired swallowing can result in food/fluids entering the lungs (aspiration), causing significant damage to developing lungs in children. Current management for aspiration in children includes change to food/fluid texture and/or enteral feeding with no specific treatments available. Respiratory muscle strength training (RMST) is effective in improving swallow function in adults with neurological diseases; however no data exists in children. The aim is to improve the management and respiratory outcomes of children who aspirate. We will conduct a study in which children will either complete respiratory muscle strength training with the RMST device or plain mask (control arm). We will see if this improves aspiration and quality of life.
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 137042 0
Dr Miriam Cameron
Address 137042 0
Queensland Children's Hospital, 501 Stanley St, South Brisbane, QLD, 4101
Country 137042 0
Australia
Phone 137042 0
+61 07 3069 7333
Fax 137042 0
Email 137042 0
[email protected]
Contact person for public queries
Name 137043 0
Miriam Cameron
Address 137043 0
Queensland Children's Hospital, 501 Stanley St, South Brisbane, QLD, 4101
Country 137043 0
Australia
Phone 137043 0
+61 07 3069 7333
Fax 137043 0
Email 137043 0
[email protected]
Contact person for scientific queries
Name 137044 0
Miriam Cameron
Address 137044 0
Queensland Children's Hospital, 501 Stanley St, South Brisbane, QLD, 4101
Country 137044 0
Australia
Phone 137044 0
+61 07 3069 7333
Fax 137044 0
Email 137044 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
Specific population and do not want to make this public


What supporting documents are/will be available?

Doc. No.TypeCitationLinkEmailOther DetailsAttachment
24221Ethical approval    388496-(Uploaded-30-09-2024-11-48-47)-HREC approval 070324.pdf



Results publications and other study-related documents

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No documents have been uploaded by study researchers.

Documents added automatically
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