Trial Review

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Trial registered on ANZCTR


Registration number
ACTRN12620000071921
Ethics application status
Approved
Date submitted
3/12/2019
Date registered
29/01/2020
Date last updated
25/02/2024
Date data sharing statement initially provided
29/01/2020
Type of registration
Prospectively registered

Titles & IDs
Public title
Preschool HABIT-ILE: A randomised controlled trial to determine efficacy of intensive rehabilitation compared to usual care to improve motor skills of children, aged 2 to 5 years, with bilateral cerebral palsy.
Scientific title
Preschool HABIT-ILE: A randomised controlled trial to determine efficacy of intensive rehabilitation compared to usual care to improve motor skills of children, aged 2 to 5 years, with bilateral cerebral palsy.
Secondary ID [1] 299716 0
None
Universal Trial Number (UTN)
The Universal Trial Number (UTN) is U1111-1243-6633
Trial acronym
Preschool HABIT-ILE
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Cerebral Palsy 315203 0
Condition category
Condition code
Neurological 313520 313520 0 0
Other neurological disorders
Physical Medicine / Rehabilitation 313521 313521 0 0
Occupational therapy

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
HABIT-ILE is a motor learning approach simultaneously addressing coordination of the upper and lower limbs. Key elements of HABIT-ILE are:

Dose: 30 hours of therapy achieved through a 2-week intensive group-delivered model for 3 hrs/day over 10 days in addition to a home program for generalization of learning. Home program dose will aim to achieve a further 10 hours over the two week intervention period for a total dose of preschool HABIT-ILE of 40 hours.

Mode: Groups of 4-6 children (1:1 or 2:1 therapist:child ratio according to ability).

Content and tailoring: The intervention will be based on the child’s motor abilities (determined at baseline), age, interests and caregiver-identified functional goals. Tasks/activities are made incrementally more challenging. Practice will be embedded in play, using part and whole task practice with high repetition including (i) table top fine motor play-based activities; (ii) activities of daily living when sitting/standing/walking; and (iii) gross motor play. Lower extremity motor abilities and postural control will be progressed from lying to sitting on the floor and then to a small bench, and transitions between floor to sitting, sitting to standing.

Intervention providers: Physiotherapists and occupational therapists, who have completed standardized training and are experienced in delivering HABIT-ILE with older children. A speech pathologist will consult with the team if children have specific communication (receptive and/or expressive) or feeding difficulties.

The level of intensity (e.g. low, moderate, vigorous) and how this is assessed: Low to vigorous. Will vary across the intervention session. Children will be wearing polar heart rate monitors during the intervention sessions.

Strategies used to assess or monitor participant adherence to the intervention, (e.g. session attendance checklists, diary, etc) : Daily sign on/off sheets for caregivers, daily activity log detailing activities throughout course of session completed by one of treating therapists.

For 30 minutes, at the conclusion of each session, the child’s treating therapists will meet individually with the child’s caregiver/s (one-on one) and discuss the daily program and make suggestions about activities that could be practised at home. These will be detailed on a written home program form and will include a home practice log completed by the caregiver. The home program activities will be reviewed daily by the child’s treating therapist in collaboration with the child’s caregiver and updated as appropriate. Parents will be able to take short videos/photos of home practice activities on their smart phone if they wish so to have a visual reminder to take home. Location: Centre for Children’s Health Research and QCH, South Brisbane.

Fidelity: Standardised therapist training will be provided to therapists employed to deliver the intervention. The training package will include an intervention manual and onsite training during the first Preschool HABIT-ILE camp lead by a master trainer
Location: Centre for Children’s Health Research and QCH, South Brisbane.
Intervention code [1] 316082 0
Rehabilitation
Intervention code [2] 316083 0
Treatment: Other
Comparator / control treatment
Usual care over the six month wait-list period will vary for children with CP and can range from weekly clinic-based therapy sessions to school-based consultative services provided on a monthly, quarterly or yearly basis. In order to understand the variability in usual care received, all families in both groups will complete a usual care diary for the duration of study involvement. The diary (via an APP) will record the frequency and duration of physiotherapy, and occupational therapy and any other concurrent medical interventions such as intramuscular Botulinum Toxin A injections and/or serial casting. All children in the control group will be offered Preschool HABIT-ILE commencing at the the 6 month retention time point (T3).
Control group
Active

Outcomes
Primary outcome [1] 321975 0
Objective assessment of fine and gross motor skills
The Peabody Developmental Motor Skills (PDMS-2) will evaluate gross and fine motor skills. This standardised, norm reference measure for children from birth to 5 years of age, has been validated as a discriminative measure, and demonstrated responsiveness to change for toddlers with CP.
Timepoint [1] 321975 0
Baseline measures will be taken between 1-3 weeks prior to the intervention (T0). Primary outcome at Primary End-Point (T2) within 1-2 weeks following the 2 week intervention program and; retention (T3) at 6 months post intervention
Secondary outcome [1] 376889 0
Bimanual Hand Performance
Both Hands Assessment measures how children with bilateral CP use their hands together in bimanual activities. Rasch measurement modelling showed strong evidence of internal construct validity, with two separate item difficulty hierarchies for children with: (a) symmetric upper limb use; (b) asymmetric upper limb use. The test uses a selection of toys to elicit bimanual hand behaviour in a structured play session. The BoHA takes minutes to complete.
Timepoint [1] 376889 0
Baseline measures will be taken between 1-3 weeks prior to the intervention (T0). Secondary outcome at Secondary End-Point (T2) within 1-2 weeks following the 2 week intervention program and; retention (T3) at 6 months post intervention
Secondary outcome [2] 376890 0
Gross motor function Gross Motor Function Measure-66 is a criterion referenced observation measure developed using Rasch modelling to measure gross motor function of children with CP.
Timepoint [2] 376890 0
Baseline measures will be taken between 1-3 weeks prior to the intervention (T0). Secondary outcome at secondary End-Point (T2) within 1-2 weeks following the 2 week intervention program and; retention (T3) at 6 months post intervention
Secondary outcome [3] 376892 0
Self-Care Paediatric Evaluation of Disability Inventory Computer Adapted Test (PEDI-CAT) is a Rasch analysed parent completed questionnaire which measures ability in functional domains of self-care, mobility, social/cognitive and responsibility using normative standard scores and scaled scores with good validity, reliability, and standardisation with typically developing children. The 'content-balanced' ” will be used because the test developers advised that this version is more suitable for research purposes. This change was made prior to recruitment commencing.
Timepoint [3] 376892 0
Baseline measures will be taken between 1-3 weeks prior to the intervention (T0). Secondary outcome at secondary End-Point (T2) within 1-2 weeks following the 2 week intervention program and; retention (T3) at 6 months post intervention
Secondary outcome [4] 376893 0
COPM Performance of occupational performance goals.
The Canadian Occupational Performance Measure (COPM) will be used to measure performance of and satisfaction with individually defined self-care, leisure or productivity goals. Test retest reliability is high (ICC 0.76-0.89) and the COPM is responsive to change. Children and their caregivers will set up to three goals. Perceived performance of an individualized goal and satisfaction with performance is rated on a 1-10 scale with higher scores reflecting higher perceived performance and satisfaction.
Timepoint [4] 376893 0
Baseline measures will be taken between 1-3 weeks prior to the intervention (T0). Secondary outcome at secondary End-Point (T2) within 1-2 weeks following the 2 week intervention program and; retention (T3) at 6 months post intervention
Secondary outcome [5] 376894 0
Global Performance: ACTIVLIM-CP
The ACTIVLIM-CP is a parent completed questionnaire covering a range of daily activities either involving the arms or legs, or both. The questionnaire comprises 43 items on a unidimensional scale, with high reliability (R=0.98) and reproducibility (R=0.97). The questionnaire is suitable for use with children aged 2 to 18 years.
Timepoint [5] 376894 0
Baseline measures will be taken between 1-3 weeks prior to the intervention (T0). Secondary outcome at secondary End-Point (T2) within 1-2 weeks following the 2 week intervention program and; retention (T3) at 6 months post intervention
Secondary outcome [6] 376895 0
Quality of Life
The Infant Toddler Quality of Life Questionnaire (ITQOL) is designed for infants aged 2 months to 5 years of age. The ITQOL comprises 97 items, with good evidence for discriminative validty and reliability. The questionnaire will be completed by parent/carer of the child.
Timepoint [6] 376895 0
Baseline measures will be taken between 1-3 weeks prior to the intervention (T0). Secondary outcome at secondary End-Point (T2) within 1-2 weeks following the 2 week intervention program and; retention (T3) at 6 months post intervention
Secondary outcome [7] 376896 0
Quality of Life
The Child Health Utility Index (CHU9) is a paediatric health related quality of life measure for use in economic evaluation. The measure consists of nine questions. In this study, the CHU9 will be completed by the child’s primary caregiver.
Timepoint [7] 376896 0
Baseline measures will be taken between 1-3 weeks prior to the intervention (T0). Secondary outcome at secondary End-Point (T2) within 1-2 weeks following the 2 week intervention program and; retention (T3) at 6 months post intervention
Secondary outcome [8] 376897 0
Health Economics Health care utilization will be assessed using a resource use questionnaire (HRU). Utility will be derived from the CHU-9D, a generic child quality of life measure designed specifically for economic evaluation and which has been validated in an Australian population. Incremental Cost Effectiveness Ratios (ICERs) will be estimated and where appropriate sensitivity analyses undertaken as in previous RCTs by our group.
Timepoint [8] 376897 0
Baseline and 6 months
Secondary outcome [9] 376898 0
Objective physical activity
Assessed via The ActiGraph GT3X+ (a triaxial accelerometer housed inside a watch-sized device). ActiGraph GT3X+ accelerometry is valid and feasible to detect and quantify objective bimanual performance in children with CP. One ActiGraph GT3X+ will be worn on each wrist, one additional ActiGraph GT3X+ will be worn on the less-affected ankle, and one Polar OH1 Optical HR Monitor will be worn on one upper arm during PDMS-2, BoHA, and GMFM-88 assessments. Two ActiGraphs only (1 less-affected ankle, 1 less-affected wrist) will also be worn for 7 days during all waking hours at each timepoint to assess daily habitual physical activity.
Timepoint [9] 376898 0
Baseline measures will be taken between 1-3 weeks prior to the intervention (T0). Secondary outcome at secondary End-Point (T2) within 1-2 weeks following the 2 week intervention program and; retention (T3) at 6 months post intervention
Secondary outcome [10] 378566 0
Mobility
Paediatric Evaluation of Disability Inventory Computer Adapted Test (PEDI-CAT) is a Rasch analysed parent completed questionnaire which measures ability in functional domains of self-care, mobility, social/cognitive and responsibility and using normative standard scores and scaled scores with good validity, reliability, and standardisation with typically developing children. The “speedy” version will be used in order to minimise assessor burden.
Timepoint [10] 378566 0
Baseline measures will be taken between 1-3 weeks prior to the intervention (T0). Secondary outcome at secondary End-Point (T2) within 1-2 weeks following the 2 week intervention program and; retention (T3) at 6 months post intervention
Secondary outcome [11] 378567 0
COPM Satisfaction with occupational performance goals.
The Canadian Occupational Performance Measure (COPM) will be used to measure performance of and satisfaction with individually defined self-care, leisure or productivity goals. Test retest reliability is high (ICC 0.76-0.89) and the COPM is responsive to change. Children and their caregivers will set up to three goals. Perceived performance of an individualized goal and satisfaction with performance is rated on a 1-10 scale with higher scores reflecting higher perceived performance and satisfaction.
Timepoint [11] 378567 0
Baseline measures will be taken between 1-3 weeks prior to the intervention (T0). Secondary outcome at secondary End-Point (T2) within 1-2 weeks following the 2 week intervention program and; retention (T3) at 6 months post intervention
Secondary outcome [12] 378570 0
Upper limb movement.
Assessed via The ActiGraph GT3X+ (a triaxial accelerometer housed inside a watch-sized device). ActiGraph GT3X+ accelerometry is valid and feasible to detect and quantify objective bimanual performance in children with CP. One ActiGraph GT3X+ will be worn on each wrist, one additional ActiGraph GT3X+ will be worn on the less-affected ankle, and one Polar OH1 Optical HR Monitor will be worn on one upper arm during PDMS-2, BoHA, and GMFM-88 assessments. Two ActiGraphs only (1 less-affected ankle, 1 less-affected wrist) will also be worn for 7 days during all waking hours at each timepoint to assess daily habitual physical activity.
Timepoint [12] 378570 0
Baseline measures will be taken between 1-3 weeks prior to the intervention (T0). Secondary outcome at secondary End-Point (T2) within 1-2 weeks following the 2 week intervention program and; retention (T3) at 6 months post intervention
Secondary outcome [13] 405810 0
Social/Cognitive
Pediatric Evaluation of Disability Inventory Computer Adapted Test (PEDI-CAT) is a Rasch analysed parent completed questionnaire which measures ability in functional domains of of self care, mobility, social/cognitive and responsibility using normative standard scores and scaled scores with good validity, reliability, and standardisation with typically developing children. The “speedy” version will be used in order to minimise assessor burden.
Timepoint [13] 405810 0
Baseline measures will be taken between 1-3 weeks prior to the intervention (T0). Secondary outcome at secondary End-Point (T2) within 1-2 weeks following the 2 week intervention program and; retention (T3) at 6 months post intervention
Secondary outcome [14] 405811 0
Responsibility
Pediatric Evaluation of Disability Inventory Computer Adapted Test (PEDI-CAT) is a Rasch analysed parent completed questionnaire which measures ability in functional domains of self care, mobility, social/cognitive and responsibility using normative standard scores and scaled scores with good validity, reliability, and standardisation with typically developing children. The “speedy” version will be used in order to minimise assessor burden.
Timepoint [14] 405811 0
Baseline measures will be taken between 1-3 weeks prior to the intervention (T0). Secondary outcome at secondary End-Point (T2) within 1-2 weeks following the 2 week intervention program and; retention (T3) at 6 months post intervention

Eligibility
Key inclusion criteria
Key inclusion criteria:
(a) diagnosed with bilateral CP (diplegia/quadriplegia), GMFCS levels II (walks with limitations) to IV (limited self-mobility but able to do a standing transfer with the assistance of 1 person);
(b) aged 2 to 5 years, 5 months at study entry
(c) ability to grasp light objects and lift more impaired arm 15cm above a table surface;
(d) able to understand instructions and complete testing.
Minimum age
2 Years
Maximum age
5 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Key exclusion criteria:
(a) uncontrolled seizures
(b) orthopaedic or neurological surgery in the six months prior to or scheduled during study period (eligible for inclusion if at least 12 months orthopaedic or neurological surgery)
(c) visual impairment interfering with treatment/testing; and
(d) inability to undertake standing transfers and/or walk a few steps (with a walker).

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)
Simple randomisation using a randomisation table created by computer software (i.e. computerised sequence generation)
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
Dr Mark Chatfield, Biostatistician, will provide expert advice for guiding and assisting with the analysis. Analyses will follow standard principles for RCTs using two-group comparisons on all participants on an intention-to-treat basis. Imputation techniques will avoid bias as a consequence of non-ignorable missing data during follow up. Primary comparison immediately post intervention (T2) based on PDMS-2 Total Motor Quotient scores will be between treatment groups using linear regression with treatment group (HABIT-ILE/waitlist) included as the main effect and baseline PDMS-2 Motor Quotient as the covariable. Effect estimates will be presented as mean difference and 95% confidence interval. Secondary analyses will use similar methods to compare outcomes between groups immediately post intervention (T2) for gross motor function, bimanual hand function, self-care and performance of and satisfaction with individualised goals. In cases where interval data are not able to be transformed appropriately for regression analyses, non-parametric methods (Mann-Whitney U) will be used for between-treatment comparisons. Possible differential attrition will be assessed by comparing baseline characteristics of drop-outs and continuing participants using t-tests (or Mann-Whitney U) for continuous variables and chi-squared tests for categorical variables. Sensitivity analyses of all outcomes will be conducted using multiple imputation techniques, to investigate the effect of non-ignorable missing data during follow up.

Recruitment
Recruitment status
Recruiting
Date of first participant enrolment
Anticipated
1/10/2020
Actual
1/01/2021
Date of last participant enrolment
Anticipated
30/06/2024
Actual
Date of last data collection
Anticipated
31/12/2024
Actual
Sample size
Target
60
Accrual to date
21
Final
Recruitment in Australia
Recruitment state(s)
QLD
Recruitment hospital [1] 15164 0
Queensland Children's Hospital - South Brisbane
Recruitment postcode(s) [1] 28466 0
4101 - South Brisbane

Funding & Sponsors
Funding source category [1] 304188 0
Charities/Societies/Foundations
Name [1] 304188 0
Ramaciotti Foundation Health Investment Grant 2019HIG/043
Country [1] 304188 0
Australia
Funding source category [2] 304285 0
University
Name [2] 304285 0
Faculty of Medicine, The University of Queensland
Country [2] 304285 0
Australia
Primary sponsor type
University
Name
The University of Queensland
Address
The University of Queensland
St Lucia QLD 4072
Country
Australia
Secondary sponsor category [1] 304419 0
None
Name [1] 304419 0
Address [1] 304419 0
Country [1] 304419 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 304659 0
Children's Health Queensland Human Research Ethics Committee
Ethics committee address [1] 304659 0
Level 7, Centre for Children's Health Research
Lady Cilento Children's Hospital Precinct
62 Graham Street South Brisbane QLD 4101
Ethics committee country [1] 304659 0
Australia
Date submitted for ethics approval [1] 304659 0
25/11/2019
Approval date [1] 304659 0
18/02/2020
Ethics approval number [1] 304659 0
HREC/19/QCHQ/59444
Ethics committee name [2] 304742 0
The University of Queensland's Human Research Ethics Committee
Ethics committee address [2] 304742 0
Cumbrae-Stewart Building #72
The University of Queensland
St Lucia QLD 4072
Ethics committee country [2] 304742 0
Australia
Date submitted for ethics approval [2] 304742 0
25/11/2019
Approval date [2] 304742 0
20/02/2020
Ethics approval number [2] 304742 0
2020000336/HREC/19/QCHQ/59444

Summary
Brief summary
In Australia, 35,000 people have cerebral palsy (CP), and between 60-70% of these have difficulties with movement on both sides of their body. There are currently no effective interventions for children with CP that affects both sides of their body to improve their ability to use their hands, walk and perform daily life tasks. We will test the efficacy of Hand Arm Bimanual Intensive Training Including Lower Extremity (HABIT-ILE), in pre-school aged children with cerebral palsy and compare results to usual care.
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 97754 0
A/Prof Leanne Sakzewski
Address 97754 0
The University of Queensland
Queensland Cerebral Palsy and Rehabilitation Research Centre (QCPRRC)
Centre for Children’s Health Research
Level 6, 62 Graham Street
South Brisbane Qld 4101 Australia
Country 97754 0
Australia
Phone 97754 0
+61 7 3069 7345
Fax 97754 0
Email 97754 0
l.sakzewski1@uq.edu.au
Contact person for public queries
Name 97755 0
Dr Natalie Dos Santos
Address 97755 0
The University of Queensland
Queensland Cerebral Palsy and Rehabilitation Research Centre (QCPRRC)
Centre for Children’s Health Research
Level 6, 62 Graham Street
South Brisbane Qld 4101 Australia
Country 97755 0
Australia
Phone 97755 0
+61 7 3069 7356
Fax 97755 0
Email 97755 0
n.dossantos@uq.edu.au
Contact person for scientific queries
Name 97756 0
A/Prof Leanne Sakzewski
Address 97756 0
The University of Queensland
Queensland Cerebral Palsy and Rehabilitation Research Centre (QCPRRC)
Centre for Children’s Health Research
Level 6, 62 Graham Street
South Brisbane Qld 4101 Australia
Country 97756 0
Australia
Phone 97756 0
+61 7 3069 7345
Fax 97756 0
Email 97756 0
l.sakzewski1@uq.edu.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?
The study team are available to collaborate with other research teams upon receipt of a reasonable request to access study data. Expressions of interest to access study data (all of the individual participant data collected during the trial), made out the Principal Investigator, Assoc. Prof. Leanne Sakzewski will be considered and the group level or individual level de-identified data could be shared as appropriate.
When will data be available (start and end dates)?
Data will be available on completion of Data collection - 31/12/2022. End date: 2027.
Available to whom?
The study team are available to collaborate with other research teams upon receipt of a reasonable request to access study data.
Available for what types of analyses?
Expressions of interest to access study data, made out the Principal Investigator, Assoc. Prof. Leanne Sakzewski will be considered and the group level or individual level de-identified data could be shared as appropriate for IPD met-analyses.
How or where can data be obtained?
Expressions of interest to access study data, made out the Principal Investigator, Assoc. Prof. Leanne Sakzewski will be considered. Postal Address: Assoc. Prof. Leanne Sakzewski
UQ - Queensland Cerebral Palsy and Rehabilitation Research Centre (QCPRRC) Centre for Children’s Health Research Level 6, 62 Graham Street
South Brisbane Qld 4101 Australia E l.sakzewski1@uq.edu.au


What supporting documents are/will be available?

Doc. No.TypeCitationLinkEmailOther DetailsAttachment
5998Informed consent form    378674-(Uploaded-14-07-2020-11-48-50)-Study-related document.docx
5999Other    Study Flyer 378674-(Uploaded-14-07-2020-11-49-51)-Study-related document.pptx
6000Informed consent form    378674-(Uploaded-14-07-2020-11-51-55)-Study-related document.docx



Results publications and other study-related documents

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