Trial Review

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Trial registered on ANZCTR


Registration number
ACTRN12621001325897
Ethics application status
Approved
Date submitted
2/08/2021
Date registered
29/09/2021
Date last updated
29/09/2021
Date data sharing statement initially provided
29/09/2021
Date results information initially provided
29/09/2021
Type of registration
Retrospectively registered

Titles & IDs
Public title
Feasibility and impact of a physical activity and lifestyle program for Aboriginal families with Machado-Joseph disease living in the Top End of Australia
Scientific title
Feasibility and impact of a physical activity and lifestyle program for Aboriginal families with Machado-Joseph disease living in the Top End of Australia
Secondary ID [1] 304928 0
Nil known
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Machado-Joseph disease 323074 0
spinocerebellar ataxia type 3 323078 0
Difficulties walking and moving around 323079 0
Neurodegenerative diseases 323453 0
Condition category
Condition code
Physical Medicine / Rehabilitation 320655 320655 0 0
Physiotherapy
Neurological 321003 321003 0 0
Neurodegenerative diseases

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Brief Name:

Feasibility and impact of a physical activity and lifestyle program for Aboriginal families with Machado-Joseph disease living in the Top End of Australia


Why:

Physical activity and lifestyle programs are scarce for people with hereditary ataxias and neurodegenerative diseases. Aboriginal families in the Top End of Australia, who have lived with MJD for generations, co-designed a physical activity and lifestyle program, called the ‘Staying Strong Toolbox’. The aim of this study was to explore the feasibility and impact of the program on walking and moving around.

What:

Participants were informed of study requirements in their preferred language by local community researchers and provided either written or oral consent. Each participant communicated in their preferred language throughout the study.

Staying Strong Toolbox Program

The program was tailored to each individual through use of the Toolbox workbook (Carr 2021). Based on the Staying Strong framework, the workbook has six domains for keeping yourself strong on the inside and outside: ‘Exercising your body’, ‘going country’, ‘searching for good medicine’, ‘keeping yourself happy’, ‘families helping each other’, and ‘something important to do’ (Carr 2019) Within each of the six domains, the workbook includes a range of activities (tools in the Toolbox). The participant selects activities of their choice from each domain to be included in their individualised program, according to their functional level: Strong (independently mobile), Wobbly (mobilising using a walking aid), Wheelchair (‘wheelchair’ dependent) (Carr 2021). Groups of activities within each domain of the workbook and the workbook prototype has been published elsewhere (Carr 2021).

Using the workbook, participants (either alone, with family or physiotherapist (JC) and community researchers) selected from the range of activities within each domain, by ticking activities they wished to do to keep walking and moving around. Individuals discussed activities they had selected with the physiotherapist (JC), who provided guidance where necessary. This guidance included advice on how to tailor tasks to the participant’s functional level to ensure the most physical benefit could be gained, and to determine whether tasks could be performed independently or would require assistance. Lastly, participants set a specific goal to work on. Their goal and chosen activities were collated into a weekly planner by the physiotherapist (JC) in collaboration with each participant, to form a four-week program. Duration for set up process of each program ranged between 1-2 hours. Participants carried out their program over a four-week period.

Delivery
• Each participant had a copy of their weekly plan (on paper or as image on phone).
• Each participant undertook their Toolbox activities as per their weekly planner with flexibility to change.
• For each activity on each occasion, participants identified the assistance they required and who they required assistance from (e.g. alone, family members and/or the physiotherapist (JC), and was provided accordingly.
• Participant performance of activities was informally assessed by the physiotherapist (JC) throughout the program and guidance provided as required.
• The weekly planner was revised with participants weekly by the physiotherapist (JC) to establish whether participants felt activities needed to be included or removed. Discussions ranged between 30 minutes and 2 hours.
• On program completion, participants were provided with a printout of their own home program based on activities they had done throughout the program pilot.

Activities
• Minimum activity target for each participant was to be physically active for at least one hour, three times a week, over the four-week program period at a ‘somewhat hard’ level of exertion (RPE 12-14).

Facilitators
• The physiotherapist (JC) in collaboration with local community researchers provided:
o Set up and assistance with each participants’ program in each community.
o In-person assistance throughout each four-week program period as required.
o Coaching and encouragement throughout the program.
o Daily check-ins on weekdays via phone with participants in regard to their progress, unless participants made first contact.

Transport
• Transport requirements were determined by participants, provided either by family, MJD Foundation support workers, or researchers.
• Vehicles were provided by the MJD Foundation with shared access. Vehicles included a four-wheel drive vehicle (4WD) and wheelchair accessible bus on Groote Eylandt and a 4WD (three weeks) and car (one week) in Ngukurr.

Equipment available
• Equipment was available from the MJD Foundation: exercise bike, fishing gear, camp cooking gear, balls, water carriers, stationery, portable printer, EPIRB, 4WD recovery gear, fuel, basketball, football, stopwatch.
• Light snacks and refreshments were provided when researchers accompanied participants (e.g. cold drinks, fruit and/or sandwiches/bread/damper).

Who Provided:

Research Team
Aboriginal community researchers including community Elders (GL, GlL, JC, BD, OD) and physiotherapists with extensive experience in rural and remote setting (JC).

How:

Programs were conducted face to face with JC and community researcher partners or with community research partners alone. Some participants also trained independently as well as independently accompanied by family members.

Where:

See above in ‘What’ section - ‘Location’

When and how much:

See above in ‘What’ section: ‘Delivery’ and ‘Activities’

Tailoring:

See above in ‘What’ section - Staying Strong Toolbox program’

Modifications:

Participant performance of activities was informally assessed by the physiotherapist (JC) throughout the program and guidance provided as required. The weekly planner was revised with participants weekly by the physiotherapist (JC) to establish whether participants felt activities needed to be included or removed. Discussions ranged from 30 minutes to 2 hours.

How well:

Adherence with the program was assessed (reach > 80% of the minimum activity target) for each participant. A research diary was used to collect adherence data. Activities were self-reported by participants and/or observed by the research team according to participant preference. Activity data collected was as follows: activities completed, locations, assistance required, supports, duration, repetitions, attendance and reasons for non-attendance where relevant, serious adverse events or coinciding community events.

References:

Carr JJ, Lalara J, Lalara G, O'Hare G, Massey L, Kenny N, et al. 'Staying strong on the inside and outside' to keep walking and moving around: Perspectives from Aboriginal people with Machado Joseph Disease and their families from the Groote Eylandt Archipelago, Australia. PLoS One. 2019;14(3).

Carr JJ, Lalara J, Lalara G, Lalara G, Daniels B, Clough AR, et al. Staying Strong Toolbox: Co-design of a physical activity and lifestyle program for Aboriginal families with Machado-Joseph disease in the Top End of Australia. PLoS One. 2021;16(2):e0244311.
Intervention code [1] 321327 0
Treatment: Other
Intervention code [2] 321579 0
Lifestyle
Comparator / control treatment
No control group
Control group
Uncontrolled

Outcomes
Primary outcome [1] 328483 0
Six minute walk test - Measured walking ability and endurance.
This will be assessed as a composite outcome
Timepoint [1] 328483 0
Baseline (Week 0), pre-program (4 weeks), post-program (8 weeks)
Primary outcome [2] 328484 0
Standardised Ataxia Rating Scale - measured ataxia severity at an impairment level that provides a score from 0 (no ataxia) to 40 (most severe ataxia)
Timepoint [2] 328484 0
Baseline (Week 0), Pre-program (Week 4), Post-program (week 8)
Primary outcome [3] 328784 0
Timed Up and Go - Measured balance and mobility
This will be assessed as a composite outcome
Timepoint [3] 328784 0
Baseline (Week 0), Pre-program (Week 4), Post-program (Week 8)
Secondary outcome [1] 399145 0
EQ5D-5L - Quality of life
Timepoint [1] 399145 0
Pre-program (Week 4), Post-program (Week 8)
Secondary outcome [2] 399146 0
ICACAP-A - Wellbeing
Timepoint [2] 399146 0
Pre-program (Week 4), Post-program (Week 8)
Secondary outcome [3] 399147 0
Patient Specific Functional Scale (PSFS) - Goal attainment
Timepoint [3] 399147 0
Pre-program (Week 4), Post-program (Week 8)
Secondary outcome [4] 399148 0
Patient Global Impression of Change (PGIC) - self rated measure of perceived benefit
Timepoint [4] 399148 0
Post-program (Week 8)
Secondary outcome [5] 399150 0
In-depth interviews with program participants - Participants' acceptability of the program
Timepoint [5] 399150 0
Immediately after completion of the program
Secondary outcome [6] 399151 0
Steps taken - using Garmin Vivofit 4 (c) data (wrist-watch/activity tracker)
Timepoint [6] 399151 0
Four-week average before commencement of program, four week average during program
Secondary outcome [7] 400330 0
Participation and adherence to activities measured using a research diary: activities completed
Timepoint [7] 400330 0
Data recorded throughout duration of program and collected for review at the end of the program
Secondary outcome [8] 400331 0
Participation and adherence to activities measured using a research diary: activity locations
Timepoint [8] 400331 0
Data recorded throughout duration of program and collected for review at the end of the program
Secondary outcome [9] 400332 0
Participation and adherence to activities measured using a research diary: assistance required
Timepoint [9] 400332 0
Data recorded throughout duration of program and collected for review at the end of the program
Secondary outcome [10] 400333 0
Participation and adherence to activities measured using a research diary: supports
Timepoint [10] 400333 0
Data recorded throughout duration of program and collected for review at the end of the program
Secondary outcome [11] 400334 0
Participation and adherence to activities measured using a research diary: activity durations
Timepoint [11] 400334 0
Data recorded throughout duration of program and collected for review at the end of the program
Secondary outcome [12] 400335 0
Participation and adherence to activities measured using a research diary: repetitions
Timepoint [12] 400335 0
Data recorded throughout duration of program and collected for review at the end of the program
Secondary outcome [13] 400336 0
Participation and adherence to activities measured using a research diary: attendance and reasons for non-attendance where relevant
Timepoint [13] 400336 0
Data recorded throughout duration of program and collected for review at the end of the program
Secondary outcome [14] 400337 0
Participation and adherence to activities measured using a research diary: serious adverse events
Timepoint [14] 400337 0
Data recorded throughout duration of program and collected for review at the end of the program
Secondary outcome [15] 400338 0
Participation and adherence to activities measured using a research diary: coinciding community events
Timepoint [15] 400338 0
Data recorded throughout duration of program and collected for review at the end of the program

Eligibility
Key inclusion criteria
Criteria for inclusion of participants included a clinical or genetic diagnosis of MJD, aged 18 and over, identified as belonging to either Groote Eylandt or Ngukurr communities, and able to provide informed consent.
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Participants were excluded if they had additional health conditions that impacted their mobility (e.g. a neurological condition such as spinal cord injury; a vascular condition such as amputation or a moderate to severe cognitive, intellectual or mental disability) or if deemed medically unable to participate by their doctor.

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised 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
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Not Applicable
Type of endpoint/s
Efficacy
Statistical methods / analysis
As this study was a mixed method multiple case study, no sample size calculations were conducted. The sample sizes where appropriate for mixed method case studies to allow program flexibility and the inclusion of participants across all functional levels. Furthermore, small sample sizes allowed for rich description of each participant's involvement in the program.

Impact on each participant:
• Raw scores for each outcome measure for each participant were reported at each measurement timepoint, except for steps taken.
• For steps taken, mean scores were calculated for the four-week period between baseline and pre-program, and from pre-program to post-program.
• To describe change between timepoints, percentage change for each measure was calculated.

Impact across all participants:
Mobility, ataxia, steps taken:
• To determine impact on mobility and ataxia (TUG, 6MWT, SARA, steps taken), nonparametric statistical analysis was used (SPSS version 26) due to the small sample size and heterogeneity.
• All pre-program measures (baseline and pre-program) for TUG, 6MWT and SARA were first compared with post-program measures with Friedman’s test (X2, p values). Where statistical significance on Friedman’s test was found (p<0.05), pairwise comparisons between each timepoint were performed to determine where greatest changes occurred
(Wilcoxon signed rank test (p<0.05)).
• Changes in steps taken before the program compared with during the program were assessed using the Wilcoxon signed rank test.
QOL, wellbeing and goal attainment.
• Change was described by examining raw scores since no appropriate normative data were available to facilitate comparisons for EQ5D-5L and ICECAP-A.

Recruitment
Recruitment status
Completed
Date of first participant enrolment
Anticipated
Actual
5/11/2018
Date of last participant enrolment
Anticipated
Actual
19/01/2019
Date of last data collection
Anticipated
Actual
3/05/2019
Sample size
Target
8
Accrual to date
Final
8
Recruitment in Australia
Recruitment state(s)
NT
Recruitment postcode(s) [1] 34845 0
0822 - Angurugu
Recruitment postcode(s) [2] 34846 0
0852 - Ngukurr

Funding & Sponsors
Funding source category [1] 309308 0
Other Collaborative groups
Name [1] 309308 0
Lowitja Institute Aboriginal and Torres Strait Islander Health CRC (Lowitja Institute CRC) (grant ID: 017-SF-005)
Country [1] 309308 0
Australia
Funding source category [2] 309321 0
Charities/Societies/Foundations
Name [2] 309321 0
Machado-Joseph disease Foundation
Country [2] 309321 0
Australia
Primary sponsor type
University
Name
James Cook University
Address
1/14-88 McGregor Rd, Smithfield QLD 4878
Country
Australia
Secondary sponsor category [1] 310290 0
Charities/Societies/Foundations
Name [1] 310290 0
Machado-Joseph disease Foundation
Address [1] 310290 0
MJD Foundation Ltd.
Darwin, Northern Territory
Australia
PO Box 1745
Country [1] 310290 0
Australia

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 309137 0
Human Research Ethics Committee (HREC) of the Northern Territory Department of Health and Menzies School of Health Research
Ethics committee address [1] 309137 0
John Mathews Building Royal Darwin Hospital Campus, 58 Rocklands Dr, Tiwi NT 0810
Ethics committee country [1] 309137 0
Australia
Date submitted for ethics approval [1] 309137 0
31/01/2018
Approval date [1] 309137 0
05/03/2018
Ethics approval number [1] 309137 0
HREC 2018-3044
Ethics committee name [2] 309145 0
James Cook University HREC
Ethics committee address [2] 309145 0
JCU Connect Ethics Team, Room 310, Building 142 (The Science Place), 1 James Cook Dr, Douglas QLD 4814
Ethics committee country [2] 309145 0
Australia
Date submitted for ethics approval [2] 309145 0
29/03/2018
Approval date [2] 309145 0
14/11/2018
Ethics approval number [2] 309145 0
H7367

Summary
Brief summary
The most common spinocerebellar ataxia worldwide, Machado-Joseph disease (MJD) has the highest estimated prevalence in affected Aboriginal communities of the Top End of Australia. MJD, or spinocerebellar ataxia 3 (SCA3), is a rare autosomal dominant neurodegenerative disease that leads to progressive ataxia and functional decline. The ‘Staying Strong Toolbox’ is a physical activity and lifestyle program designed by Aboriginal families living in Groote Eylandt and Ngukurr, to keep their families walking and moving around. Families with MJD in these communities who were concerned about their declining mobility partnered with the MJD Foundation and university researchers to co-design the Staying Strong Toolbox, based on their experiences and what is known from MJD research. The aim of this study was in order to determine the feasibility and impact of the Staying Strong Toolbox program on walking and moving around for Aboriginal families with MJD in the Top End of Australia.

A mixed method multiple case study design was used to pilot the Staying Strong Toolbox. Eight individuals with MJD participated in the program for four weeks. Participants tailored their own program using the Toolbox workbook. Families, support workers and researchers facilitated each
individual’s program. Feasibility was determined through program participation, adherence, coinciding or serious adverse events, participant acceptability and cost. Impact was determined through measures of mobility, ataxia, steps, quality of life, wellbeing and goal attainment, assessed before and after the program.
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 113114 0
Dr Jennifer Carr
Address 113114 0
Gold Coast University Hospital, 1 Hospital Blvd, Southport QLD 4215
Country 113114 0
Australia
Phone 113114 0
+610756873027
Fax 113114 0
Email 113114 0
[email protected]
Contact person for public queries
Name 113115 0
Dr Jennifer Carr
Address 113115 0
Gold Coast University Hospital, 1 Hospital Blvd, Southport QLD 4215
Country 113115 0
Australia
Phone 113115 0
+610756873027
Fax 113115 0
Email 113115 0
[email protected]
Contact person for scientific queries
Name 113116 0
Dr Jennifer Carr
Address 113116 0
Gold Coast University Hospital, 1 Hospital Blvd, Southport QLD 4215
Country 113116 0
Australia
Phone 113116 0
+610756873027
Fax 113116 0
Email 113116 0
[email protected]

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
All data will be de-identified:
Age, level of function, community, all data pertaining to activity data, outcome measure results at each relevant time point
When will data be available (start and end dates)?
Upon publication of the study with no end date
Available to whom?
All data above will be de-identified and included in the study publication
Available for what types of analyses?
Any purpose
How or where can data be obtained?
Upon request or from the study publication once published by emailing the principal investigator: [email protected]


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

Documents added manually
No documents have been uploaded by study researchers.

Documents added automatically
No additional documents have been identified.