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Trial registered on ANZCTR


Registration number
ACTRN12618000084280
Ethics application status
Approved
Date submitted
13/04/2017
Date registered
19/01/2018
Date last updated
10/01/2019
Date data sharing statement initially provided
10/01/2019
Type of registration
Prospectively registered

Titles & IDs
Public title
Comparison of Average volume assured pressure support (AVAPS) and standard bilevel modes of non invasive ventilation in children with neuromuscular disorders.
Scientific title
Average volume assured pressure support (AVAPS) vs pressure support ventilation comparing polysomnography results and ventilaton. A crossover study in paediatric neuromuscular patients.
Secondary ID [1] 291693 0
None
Universal Trial Number (UTN)
U1111-1195-4576
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Hypoventilation 302862 0
Neuromuscular disease 302863 0
Condition category
Condition code
Respiratory 302342 302342 0 0
Sleep apnoea
Neurological 302343 302343 0 0
Other neurological disorders
Musculoskeletal 302397 302397 0 0
Other muscular and skeletal disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Crossover trial design. Participants are randomised to receive either standard pressure support ventilation followed by AVAPS or AVAPS followed by standard pressure support ventilation. The comparator is standard bilevel pressure support ventilation. The intervention is the randomisation of the order AVAPS ventilation is given to the patients whilst being blinded to the mode. Automated volume assured pressure support is currently only used in patients who don't tolerate standard bilevel pressure support. AVAPS is delivered in the same way as standard pressure support ventilation - via a nasal mask, sometimes an Oro-nasal mask. AVAPS and standard pressure support ventilation both provide 2 pressures - an inspiratory pressure and an expiratory pressure. In standard therapy, these are set and don’t change throughout the night. In AVAPS a range of pressures are set and the machine alters the pressure provided according to the patient’s breathing through the night in order to achieve a targeted tidal volume.
There is no washout period between modes of ventilation.

The same ventilator will be used throughout, the mode will be altered. Initiation and titration of each mode of ventilation will take place at the Sydney Children's Hosptial sleep lab by and experienced sleep scientist with at least 5 years experience. Patients attend the hospital in the afternoon and undergo a titration sleep study overnight. They also perform a number of respiratory function tests on the afternoon before the titration study and the morning following the study. These take approximately 1 hour. The patient will continue on each mode of ventilation at home for 3 months. The ventilator will be used everynight throughout sleep. Adherence and actual time used is an outcome measure of the study. Adherence data will be downloaded from the machine. They will be reviewed at their standard follow up appointments. They will recieve a phone call at 1 week, 1 month and 3 months post commencement of each mode to answer questionnaires.

Intervention code [1] 297779 0
Treatment: Devices
Comparator / control treatment
Each patient will also undergo 3 months of standard bilevel pressure support ventilation as the comparator. They will be asked to use the ventilation nightly throughout sleep. The actual time the machine is used for is an outcome measure of the study. The machine is used at home for a total of 6 months.
Control group
Active

Outcomes
Primary outcome [1] 301764 0
Change in polysomnography parameters -Transcutaneous CO2 (TcCO2) change from baseline. Transcutaneous CO2 monitor attached throughout polysomnograph
Timepoint [1] 301764 0
Compare to baseline and 3 months post initiation of each mode of ventilation
Secondary outcome [1] 333795 0
Compliance - Adherence data downloaded from the machine to measure actual time machine is used.
Timepoint [1] 333795 0
Data from the machine is downloaded remotely at 1 week, 1 month and 3 months post initiation of the ventilation mode. 3 months of data is downloaded at the clinic visit. This is then repeated on the second mode of ventilation.
Secondary outcome [2] 333796 0
Quality of life outcomes. Measured by questionnaires.
PEDSQL 4.0 Core
PEDSQL Neuromuscular module
PEDSQL Duchenne muscular dystrophy module
Timepoint [2] 333796 0
Completed at 1 month and 3 months post initiation of each ventilation mode.
Secondary outcome [3] 333929 0
Tolerance - Visual analogue scale for comfort.
Timepoint [3] 333929 0
Completed at 1 months and 3 months post initiation of each ventilation mode.
Secondary outcome [4] 333930 0
clinical response-Measured by number of hospital admissions/respiratory exacerbations. Composite outcome. Answered by parent/patient interview and medical record review.
Timepoint [4] 333930 0
Completed at 1 month and 3 months post initiation of each ventilation mode.
Secondary outcome [5] 333988 0
Epworth sleepiness scale - Child and adolescent.
Timepoint [5] 333988 0
Completed at 1 month and 3 months post initiation of each ventilation mode.
Secondary outcome [6] 342059 0
Polysomnography results - TST, TST in REM, EMG readings
Timepoint [6] 342059 0
During the night of titration on each mode of ventilation

Eligibility
Key inclusion criteria
Chronic neuromuscular disorder
Clinical evidence of need for non invasive ventilation
Adherence to standard care
Stable clinical status
Minimum age
0 Years
Maximum age
18 Years
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Acute respiratory failure
Unstable haemodynamically or co-existing respiratory condition eg Cystic Fibrosis.
Respiratory illness within 4 weeks of initial titration
Unwilling or unable to adhere to non invasive ventilation
Tracheostomy
Rapidly progressive neurological disorder in which the respiratory impairment would deteriorate rapidly over 12months eg SMA type 1

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)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Who is / are masked / blinded?



Intervention assignment
Other design features
Phase
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
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)
NSW

Funding & Sponsors
Funding source category [1] 296191 0
Hospital
Name [1] 296191 0
Sydney Children's Hospital
Address [1] 296191 0
High st
Randwick
NSW
2031
Country [1] 296191 0
Australia
Primary sponsor type
Individual
Name
Laura Fawcett
Address
Sydney Children's Hospital
High St
Randwick
NSW
2031
Country
Australia
Secondary sponsor category [1] 295103 0
None
Name [1] 295103 0
Address [1] 295103 0
Country [1] 295103 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 297433 0
Sydney Children's Hospitals Network Human Research Ethics Committee (SCHN HREC)
Ethics committee address [1] 297433 0
Ethics committee country [1] 297433 0
Date submitted for ethics approval [1] 297433 0
01/05/2017
Approval date [1] 297433 0
08/12/2017
Ethics approval number [1] 297433 0

Summary
Brief summary
We believe that AVAPS mode of ventilation is tolerated better by patients than standard therapy. We aim to demonstrate that AVAPS is not inferior to standard pressure support ventilation when looking at clinical outcomes.
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 74022 0
Dr Laura Fawcett
Address 74022 0
Sydney Children's Hospital
High St
Randwick
NSW
2031
Country 74022 0
Australia
Phone 74022 0
+61293821111
Fax 74022 0
Email 74022 0
laura.fawcett@health.nsw.gov.au
Contact person for public queries
Name 74023 0
Dr Laura Fawcett
Address 74023 0
Sydney Children's Hospital
High St
Randwick
NSW
2031
Country 74023 0
Australia
Phone 74023 0
+61293821111
Fax 74023 0
Email 74023 0
laura.fawcett@health.nsw.gov.au
Contact person for scientific queries
Name 74024 0
Dr Laura Fawcett
Address 74024 0
Sydney Children's Hospital
High St
Randwick
NSW
2031
Country 74024 0
Australia
Phone 74024 0
+61293821111
Fax 74024 0
Email 74024 0
l.aura.fawcett@health.nsw.gov.au

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment
Grouped data will be made available.
What supporting documents are/will be available?
No other documents available
Summary results
Not applicable