ANZCTR - Registration

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]

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

Condition category

Condition code

Neurological

Other neurological disorders

Physical Medicine / Rehabilitation

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]

Rehabilitation

Intervention code [2]

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]

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]

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]

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]

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]

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]

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]

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]

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]

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]

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]

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]

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]

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]

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]

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]

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]

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]

Baseline and 6 months

Secondary outcome [9]

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]

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]

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]

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]

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]

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]

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]

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]

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]

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]

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]

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

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]

Queensland Children's Hospital - South Brisbane

Recruitment postcode(s) [1]

4101 - South Brisbane

Funding & Sponsors

Funding source category [1]

Charities/Societies/Foundations

Name [1]

Ramaciotti Foundation Health Investment Grant 2019HIG/043

Country [1]

Australia

Funding source category [2]

University

Name [2]

Faculty of Medicine, The University of Queensland

Country [2]

Australia

Primary sponsor type

University

Name

The University of Queensland

Country

Australia

Secondary sponsor category [1]

None

Name [1]

Country [1]

Ethics approval

Ethics application status

Approved

Ethics committee name [1]

Children's Health Queensland Human Research Ethics Committee

Ethics committee address [1]

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]

Australia

Date submitted for ethics approval [1]

25/11/2019

Approval date [1]

18/02/2020

Ethics approval number [1]

HREC/19/QCHQ/59444

Ethics committee name [2]

The University of Queensland's Human Research Ethics Committee

Ethics committee address [2]

Cumbrae-Stewart Building #72
The University of Queensland
St Lucia QLD 4072

Ethics committee country [2]

Australia

Date submitted for ethics approval [2]

25/11/2019

Approval date [2]

20/02/2020

Ethics approval number [2]

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

Public notes

Contacts

Principal investigator

Name

A/Prof Leanne Sakzewski

Address

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

Australia

Phone

+61 7 3069 7345

Email

[email protected]

Contact person for public queries

Name

Natalie Dos Santos

Address

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

Australia

Phone

+61 7 3069 7356

Email

[email protected]

Contact person for scientific queries

Name

Leanne Sakzewski

Address

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

Australia

Phone

+61 7 3069 7345

Email

[email protected]

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

Trial Review

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