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Trial registered on ANZCTR


Registration number
ACTRN12618000384257
Ethics application status
Approved
Date submitted
28/02/2018
Date registered
14/03/2018
Date last updated
23/12/2021
Date data sharing statement initially provided
20/02/2019
Date results provided
23/12/2021
Type of registration
Retrospectively registered

Titles & IDs
Public title
An observational study of functional screening tests in people with cervical dystonia.
Scientific title
An observational study of functional screening tests in people with cervical dystonia
Secondary ID [1] 292458 0
Nil known
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
cervical dystonia 304230 0
Condition category
Condition code
Neurological 303579 303579 0 0
Other neurological disorders

Intervention/exposure
Study type
Observational
Patient registry
False
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
Participants will be screened by a neurologist for eligibility to join the study at their routine clinic appointment for Botox injections. Participants will then complete the study at Westmead Hospital in the same week as their Botox injection. Participants will have baseline measures of dystonia severity, current levels of exercise, cognition and involvement of the cerebellum assessed using the Toronto Western Spasmodic Rating Scale (TWSTRS), the Incidental and Planned Exercise Questionnaire (IPEQ), the Montreal Cognitive Assessment (MoCA) and the Scale for the Assessment and Rating of Ataxia (SARA). Participants will be assessed by 2 physiotherapists on the following standardised physical assessments of balance, walking and upper arm function:

Functional Gait Assessment (formerly Dynamic Gait Index)
Balance Evaluation Systems Test - MiniBEST
5 x sit to stand test
4 squares step test
Figure of Eight test – time to walk a standardized figure of eight
Box and Block test
Coin rotation test

Participants will also complete 2 questionnaires assessing fear of falling and balance confidence as follows:

Activities Balance Confidence Scale (self-reported item)
Falls self-efficacy international scale (self-reported item)

Assessments will take approximately 2 hours to complete.

Participants who are able will repeat the assessment on a second occasion within one week of the botox injection. Participants will then be asked to keep a falls diary for the following 6 months.
Intervention code [1] 298768 0
Early Detection / Screening
Comparator / control treatment
Nil
Control group
Uncontrolled

Outcomes
Primary outcome [1] 304967 0
Standing balance assessed using the MiniBEST test.
Timepoint [1] 304967 0
Measures repeated twice in the week surrounding a botox injection
Primary outcome [2] 304968 0
Gait assessed using the Functional Gait Assessment.
Timepoint [2] 304968 0
Measures repeated twice in the week surrounding the botox injection
Primary outcome [3] 305046 0
Fear of falling assessed using the Falls self-efficacy Scale International.
Timepoint [3] 305046 0
Measures taken once in the week surrounding the botox injection.
Secondary outcome [1] 343763 0
Incidence of falls in people with cervical dystonia using falls diaries.
Timepoint [1] 343763 0
6 months following participation in the study
Secondary outcome [2] 344107 0
Upper limb Function assessed by the Box and Block test.
Timepoint [2] 344107 0
Measures repeated twice in the week surrounding the botox injection.
Secondary outcome [3] 344294 0
Standing balance assessed using the 4-square step test.
Timepoint [3] 344294 0
Primary outcome measure assessed twice in the week surrounding the botox injection.
Secondary outcome [4] 344295 0
Standing balance assessed by the 5x sit to stand test.
Timepoint [4] 344295 0
Primary outcome measure assessed twice in the week surrounding the botox injection.
Secondary outcome [5] 344296 0
Gait assessed by the Figure 8 turn test.
Timepoint [5] 344296 0
Primary outcome measure assessed twice in the week surrounding the botox injection.
Secondary outcome [6] 344297 0
Balance confidence assessed by the Activities-specific Balance Confidence Scale.
Timepoint [6] 344297 0
Primary outcome measure assessed twice in the week surrounding the botox injection.
Secondary outcome [7] 344298 0
Hand dexterity assessed by the Coin rotation test.
Timepoint [7] 344298 0
Measures repeated twice in the week surrounding the botox injection.

Eligibility
Key inclusion criteria
Participants will be invited to join the study if they are over 18 years, have been diagnosed with cervical dystonia of unknown cause, can speak English, and are able to walk unaided.
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
People with other musculoskeletal or neurological disorders besides dystonia affecting the lower limbs will be excluded.

Study design
Purpose
Screening
Duration
Cross-sectional
Selection
Defined population
Timing
Prospective
Statistical methods / analysis
Considering that cervical dystonia is a rare condition, it will be very difficult to recruit a large number of participants to this study. However given that psychometric analyses of these scales have never before been conducted in people with cervical dystonia, it is worthwhile attempting these analyses with as large a sample size as the study possibly recruits. Given the limited patients in the sampling frame, a pragmatic approach has been adopted. We therefore aim to recruit 30 participants to this study, and assess the reliability and utility of these scales in people with cervical dystonia.
Data will be descriptively analysed as means and standard errors with confidence intervals calculated. Each standardised assessment will be evaluated against normative values and those of other neurological populations using one sample t-tests with the norm as the comparator value.
For intra-rater and test-retest reliabilities, intraclass correlations will be calculated. Spearman correlation coefficients will be used to determine concurrent validity among the functional tests. If the falls data support analysis (ie there are enough fallers to split the data into two groups providing adequate power to conduct a statistical analysis), cut-off points and the minimal detectable difference for each scale will be determined. For cut off points, a descriptive statistic (Shapiro-Wilks) will be performed to determine the distribution and to consider the need for data transformation. Appropriate tests (t test, Mann-Whitney U test, or cross-tabulation chi-square test) will determine which variables differ significantly between fallers and non-fallers. A regression analysis will then be performed using reported falls as the dependent variable with variables that were statistically different between faller and non-faller groups used as independent variables. Sensitivity and specificity in predicting falls status will be calculated, sensitivity is the percentage of the fallers who were correctly identified and specificity is the percentage of non-fallers that were correctly identified. If this analysis cannot be done in the current study, but data support its potential importance, a follow up study will be conducted with identification of risk of falls as the primary objective.

Recruitment
Recruitment status
Stopped early
Data analysis
Data analysis is complete
Reason for early stopping/withdrawal
Participant recruitment difficulties
Date of first participant enrolment
Anticipated
Actual
25/05/2017
Date of last participant enrolment
Anticipated
Actual
10/12/2019
Date of last data collection
Anticipated
Actual
16/06/2020
Sample size
Target
30
Accrual to date
Final
22
Recruitment in Australia
Recruitment state(s)
ACT,NSW,NT,QLD,SA,TAS,WA,VIC
Recruitment hospital [1] 10235 0
Westmead Hospital - Westmead
Recruitment postcode(s) [1] 21900 0
2145 - Westmead

Funding & Sponsors
Funding source category [1] 297026 0
Charities/Societies/Foundations
Name [1] 297026 0
Westmead Medical Research Foundation
Country [1] 297026 0
Australia
Primary sponsor type
Individual
Name
Melani Boyce
Address
Physiotherapy Department
Westmead Hospital
Hawkesbury Rd
Westmead, NSW, 2145
Country
Australia
Secondary sponsor category [1] 298026 0
None
Name [1] 298026 0
Address [1] 298026 0
Country [1] 298026 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 298211 0
Western Sydney Local Health DIstrict Human Research and Ethics Committee
Ethics committee address [1] 298211 0
Ethics committee country [1] 298211 0
Australia
Date submitted for ethics approval [1] 298211 0
25/10/2016
Approval date [1] 298211 0
14/11/2016
Ethics approval number [1] 298211 0
AU RED HREC/16/WMEAD/393

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 76354 0
Ms Melani Boyce
Address 76354 0
Physiotherapy Department
Westmead Hospital
Hawkesbury Rd
Westmead, NSW, 2145
Country 76354 0
Australia
Phone 76354 0
+61 2 8890 6500
Fax 76354 0
Email 76354 0
[email protected]
Contact person for public queries
Name 76355 0
Melani Boyce
Address 76355 0
Physiotherapy Department
Westmead Hospital
Hawkesbury Rd
Westmead, NSW, 2145
Country 76355 0
Australia
Phone 76355 0
+61 2 8890 6500
Fax 76355 0
Email 76355 0
[email protected]
Contact person for scientific queries
Name 76356 0
Melani Boyce
Address 76356 0
Physiotherapy Department
Westmead Hospital
Hawkesbury Rd
Westmead, NSW, 2145
Country 76356 0
Australia
Phone 76356 0
+61 2 8890 6500
Fax 76356 0
Email 76356 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
Individual participant data will not be shared due to privacy reasons.


What supporting documents are/will be available?

Doc. No.TypeCitationLinkEmailOther DetailsAttachment
1410Informed consent form    373324-(Uploaded-19-02-2019-21-40-39)-Study-related document.docx



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.