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Trial registered on ANZCTR


Registration number
ACTRN12618000985280
Ethics application status
Approved
Date submitted
1/06/2018
Date registered
12/06/2018
Date last updated
7/08/2020
Date data sharing statement initially provided
14/05/2019
Type of registration
Retrospectively registered

Titles & IDs
Public title
Understanding outcomes for Australian children undergoing Selective Dorsal Rhizotomy
Scientific title
Australian Children undergoing Selective Dorsal Rhizotomy - a national registry of multidimensional outcomes pertaining to the International Classification of Functioning, Disability and Health.
Secondary ID [1] 294972 0
National Registry for Selective Dorsal Rhizotomy in Australia: PG1616
Universal Trial Number (UTN)
U1111-1214-5384
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Cerebral Palsy 307971 0
Condition category
Condition code
Neurological 307011 307011 0 0
Other neurological disorders
Physical Medicine / Rehabilitation 307151 307151 0 0
Other physical medicine / rehabilitation

Intervention/exposure
Study type
Observational
Patient registry
True
Target follow-up duration
10
Target follow-up type
Years
Description of intervention(s) / exposure
The Australian SDR Research Registry is a 10 year longitudinal cohort study of children with cerebral palsy who have had or will have the Selective Dorsal Rhizotomy procedure in an Australian centre or overseas centre. Data collected will include surgery and admission details, surgical and long term adverse events, and outcome measures across the body structure and function, activity and participation domains of the International Classification of Functioning, Disability and Health (ICF). Data will be collected at baseline, during inpatient admission and at one, two, five and ten years post intervention. The objective of collecting this data is to improve understanding of the short, medium and long term outcomes and any adverse effects of the intervention thereby providing clinicians with information to guide families considering this neurosurgical intervention..
Intervention code [1] 301295 0
Not applicable
Comparator / control treatment
No control group
Control group
Uncontrolled

Outcomes
Primary outcome [1] 305993 0
Change in Gross Motor Function Measure - 66 (GMFM-66) scores
Timepoint [1] 305993 0
Baseline, and at 1, 2, 5 and 10 years after selective dorsal rhizotomy surgery
Secondary outcome [1] 347232 0
Functional mobility assessed using the Functional Mobility Scale
Timepoint [1] 347232 0
Baseline and at 1, 2, 5 and 10 years post Selective Dorsal Rhizotomy surgery
Secondary outcome [2] 347235 0
Gait assessed using the Gait Profile Score and the Gait Variable Scores from 3-Dimensional Gait Analysis
Timepoint [2] 347235 0
Baseline and at 1, 2, 5 and 10 years post Selective Dorsal Rhizotomy surgery
Secondary outcome [3] 347241 0
Goal attainment assessed using the Canadian Occupational Performance Measure
Timepoint [3] 347241 0
Baseline and at 1 and 2 years post Selective Dorsal Rhizotomy
Secondary outcome [4] 347243 0
Pain assessed using the Faces Pain Scale-Revised, checklist of pain location and PROMIS Pediatric Pain Interference Scale
Timepoint [4] 347243 0
Baseline, and at 1, 2, 5 and 10 years post Selective Dorsal Rhizotomy
Secondary outcome [5] 347244 0
Quality of life assessed using the Cerebral Palsy Quality of Life Questionnaire (proxy report)
Timepoint [5] 347244 0
Baseline and at 1, 2, 5 and 10 years post Selective Dorsal Rhizotomy surgery
Secondary outcome [6] 347247 0
Surgical adverse events using the Clavien-Dindo classification
Timepoint [6] 347247 0
At time of surgery and during inpatient stay
Secondary outcome [7] 347748 0
Gross motor function assessed using the the Gillette Functional Assessment Questionnaire
Timepoint [7] 347748 0
Baseline and at 1, 2, 5 and 10 years post Selective Dorsal Rhizotomy surgery
Secondary outcome [8] 347749 0
Functional mobility assessed using the 6 minute walk test
Timepoint [8] 347749 0
Baseline and at 1, 2, 5 and 10 years post Selective Dorsal Rhizotomy surgery
Secondary outcome [9] 347750 0
Mobility assessed using the Timed up-and-go test
Timepoint [9] 347750 0
Baseline and at 1, 2, 5 and 10 years post Selective Dorsal Rhizotomy surgery
Secondary outcome [10] 347792 0
Long term adverse event of spinal deformity assessed using spine radiograph.
Timepoint [10] 347792 0
Baseline and at 1, 2, 5 and 10 years post Selective Dorsal Rhizotomy surgery
Secondary outcome [11] 347793 0
Long term adverse event of hip subluxation assessed by measuring hip migration percentages with pelvis radiograph.
Timepoint [11] 347793 0
Baseline and at 1, 2, 5 and 10 years post Selective Dorsal Rhizotomy surgery
Secondary outcome [12] 347794 0
Long term adverse event of foot deformity assessed from clinical examination in standing, and/or with assistance of a frontal (PA) and lateral radiograph in standing. Severity of complication is rated as mild, moderate or severe.
Timepoint [12] 347794 0
Baseline and at 1, 2, 5 and 10 years post Selective Dorsal Rhizotomy surgery
Secondary outcome [13] 347795 0
Presence of long term adverse event of lower limb sensory impairment as reported by the clinician and/or parent
Timepoint [13] 347795 0
Baseline and at 1, 2, 5 and 10 years post Selective Dorsal Rhizotomy surgery
Secondary outcome [14] 347796 0
Presence of long term adverse event of bladder/bowel dysfunction as reported by the clinician and/or parent
Timepoint [14] 347796 0
Baseline and at 1, 2, 5 and 10 years post Selective Dorsal Rhizotomy surgery

Eligibility
Key inclusion criteria
Children who have undergone or are undergoing selective dorsal rhizotomy procedure in Australia or overseas who consent to have their data collected in the registry.
Minimum age
No limit
Maximum age
18 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Nil

Study design
Purpose
Screening
Duration
Longitudinal
Selection
Defined population
Timing
Both
Statistical methods / analysis
Statistical analysis will be undertaken when a sample size of 14 is achieved. This is based on a previous study (Van Schie et al, 2011) where GMFM-66 scores increased from 69 (SD 8.9) to 76.2 (SD 8.5) in children classified as GMFCS I or II one year following SDR. This was determined using a paired t test with a power of 0.8 and an alpha of 0.05.
Descriptive data will be described using means and standard deviations, medians and ranges, or frequencies and proportions depending on the type and distribution of the data.

The primary research question (gross motor function) will focus on the results from the GMFM-66. Changes in gross motor function over time will firstly be assessed using the raw scores. When two time points are available a paired t test will be used. When three or more time points exist then a mixed model approach will be used to account for the correlation between the time points. Given the likely small sample test or model assumptions may be violated. Transformation of the data or non-parametric methods will then be utilized.

The GMFM-66 allows for comparison with reference curves over time allowing for the longitudinal assessment of the data (Hanna et al, 2008). Each child’s GMFM-66 score will be referenced against the average development pattern for children in his or her GMFCS level. After assigning centiles a change in function between 2 percentile measurements will be determined using an 80% coverage probability. For children functioning at GMFCS I a second percentile measurement has an 80% chance of being ±20 of the first measurement. For GMFCS level II the cut off is ±19.9. At each time point children will be classified as declined, increased or stable in their gross motor function based on these cut offs. This technique has been described previously in the literature (Hanna et al 2008, Bolster et al 2013). The frequency and proportion of children in each group will be examined over time and compared to expected outcomes from the CP population.

Differences in outcomes between procedure locations (overseas vs Australia) will be compared using independent t tests, Mann Whitney U test, or Chi square test, depending on the type and distribution of the data.

The remaining outcome measures (gait, quality of life, goal attainment and pain) will be analysed using paired t tests when 2 time points exist. When three or more time points exist then a mixed model approach will be used. Given the likely small sample model assumptions may be violated. Transformation of the data or non-parametric methods will then be employed.

All data will be analysed using Stata 14.1 (StataCorp, College Station, Texas). Alpha will be set at 0.05.


Recruitment
Recruitment status
Recruiting
Date of first participant enrolment
Anticipated
Actual
12/10/2017
Date of last participant enrolment
Anticipated
17/05/2022
Actual
Date of last data collection
Anticipated
1/12/2032
Actual
Sample size
Target
80
Accrual to date
27
Final
Recruitment in Australia
Recruitment state(s)
NSW,QLD,SA,WA,VIC
Recruitment hospital [1] 10968 0
The Children's Hospital at Westmead - Westmead
Recruitment hospital [2] 10969 0
Lady Cilento Children's Hospital - South Brisbane
Recruitment hospital [3] 10970 0
Princess Margaret Hospital - Subiaco
Recruitment hospital [4] 10971 0
The Royal Childrens Hospital - Parkville
Recruitment hospital [5] 10972 0
Womens and Childrens Hospital - North Adelaide
Recruitment postcode(s) [1] 22757 0
2145 - Westmead
Recruitment postcode(s) [2] 22758 0
4101 - South Brisbane
Recruitment postcode(s) [3] 22759 0
6008 - Subiaco
Recruitment postcode(s) [4] 22760 0
3052 - Parkville
Recruitment postcode(s) [5] 22761 0
5006 - North Adelaide

Funding & Sponsors
Funding source category [1] 299561 0
Charities/Societies/Foundations
Name [1] 299561 0
The Research Foundation, Cerebral Palsy Alliance
Country [1] 299561 0
Australia
Funding source category [2] 299649 0
Other Collaborative groups
Name [2] 299649 0
The Centre of Research Excellence in Cerebral Palsy
Country [2] 299649 0
Australia
Primary sponsor type
Hospital
Name
The Children's Hospital at Westmead
Address
Kids Rehab
The Children’s Hospital at Westmead
Locked Bag 4001
Westmead
NSW 2145
Country
Australia
Secondary sponsor category [1] 298877 0
Hospital
Name [1] 298877 0
Queensland Children's Hospital
Address [1] 298877 0
Paediatric Rehabilitation Service Queensland Children's Hospital, 501 Stanley Street South Brisbane 4101
Country [1] 298877 0
Australia
Secondary sponsor category [2] 298878 0
Hospital
Name [2] 298878 0
Women's and Children's Hospital
Address [2] 298878 0
Paediatric Rehabilitation Department
Women's and Children's Hospital
72 King William Road
North Adelaide
South Australia 5006
Country [2] 298878 0
Australia
Secondary sponsor category [3] 298880 0
Hospital
Name [3] 298880 0
The Royal Children's Hospital, Melbourne
Address [3] 298880 0
Victorian Paediatric Rehabilitation Service
The Royal Children's Hospital
50 Flemington Road
Parkville,
Victoria 3052

Country [3] 298880 0
Australia
Secondary sponsor category [4] 299026 0
Hospital
Name [4] 299026 0
Perth Children's Hospital
Address [4] 299026 0
Department of Paediatric Rehabilitation Perth Children's Hospital Locked Bag 2010 Nedlands, Western Australia 6909
Country [4] 299026 0
Australia
Other collaborator category [1] 280148 0
Individual
Name [1] 280148 0
Dr Simon Paget
Address [1] 280148 0
Kids Rehab
The Children’s Hospital at Westmead
Locked Bag 4001
Westmead
NSW 2145
Country [1] 280148 0
Australia
Other collaborator category [2] 280149 0
Individual
Name [2] 280149 0
Jenny Lewis
Address [2] 280149 0
Kids Rehab
The Children’s Hospital at Westmead
Locked Bag 4001
Westmead
NSW 2145
Country [2] 280149 0
Australia
Other collaborator category [3] 280150 0
Individual
Name [3] 280150 0
Nadine Smith
Address [3] 280150 0
Department of Paediatric Rehabilitation Perth Children's Hospital Locked Bag 2010 Nedlands, Western Australia 6909
Country [3] 280150 0
Australia
Other collaborator category [4] 280151 0
Individual
Name [4] 280151 0
Dr Emma Richardson
Address [4] 280151 0
Department of Paediatric Rehabilitation Perth Children's Hospital Locked Bag 2010 Nedlands, Western Australia 6909
Country [4] 280151 0
Australia
Other collaborator category [5] 280152 0
Individual
Name [5] 280152 0
Felicity Baker
Address [5] 280152 0
Paediatric Rehabilitation Department
Women's and Children's Hospital
72 King William Road
North Adelaide
South Australia 5006
Country [5] 280152 0
Australia
Other collaborator category [6] 280153 0
Individual
Name [6] 280153 0
Dr Olivia Lee
Address [6] 280153 0
Victorian Paediatric Rehabilitation Service
The Royal Children's Hospital
50 Flemington Road
Parkville,
Victoria 3052
Country [6] 280153 0
Australia
Other collaborator category [7] 280154 0
Individual
Name [7] 280154 0
Pam Thomason
Address [7] 280154 0
Victorian Paediatric Rehabilitation Service
The Royal Children's Hospital
50 Flemington Road
Parkville,
Victoria 3052
Country [7] 280154 0
Australia
Other collaborator category [8] 280155 0
Individual
Name [8] 280155 0
Dr Andrew Tidemann
Address [8] 280155 0
Paediatric Rehabilitation Department
Women's and Children's Hospital
72 King William Road
North Adelaide
South Australia 5006
Country [8] 280155 0
Australia
Other collaborator category [9] 280156 0
Individual
Name [9] 280156 0
Natasha Bear
Address [9] 280156 0
Department of Clinical Research and Education Child Adolescent
Princess Margaret Hospital for Children
GPO Box D184, Perth
Western Australia 6840
Country [9] 280156 0
Australia
Other collaborator category [10] 280157 0
Individual
Name [10] 280157 0
Dr Adam Fowler
Address [10] 280157 0
Department of Neurosurgery
The Children’s Hospital at Westmead
Locked Bag 4001
Westmead
NSW 2145
Country [10] 280157 0
Australia
Other collaborator category [11] 280158 0
Individual
Name [11] 280158 0
Dr Adam Scheinberg
Address [11] 280158 0
Victorian Paediatric Rehabilitation Service
The Royal Children's Hospital
50 Flemington Road
Parkville,
Victoria 3052
Country [11] 280158 0
Australia
Other collaborator category [12] 280159 0
Individual
Name [12] 280159 0
Meredith Wynter
Address [12] 280159 0
Paediatric Rehabilitation Service Queensland Children's Hospital, 501 Stanley Street South Brisbane 4101
Country [12] 280159 0
Australia
Other collaborator category [13] 280160 0
Individual
Name [13] 280160 0
Dr Kim McLennan
Address [13] 280160 0
Paediatric Rehabilitation Service Queensland Children's Hospital, 501 Stanley Street South Brisbane 4101
Country [13] 280160 0
Australia

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 300458 0
Sydney Children's Hospitals Network Human Research Ethics Committee
Ethics committee address [1] 300458 0
Research Ethics Office
Locked Bag 4001
Westmead
NSW 2145
Ethics committee country [1] 300458 0
Australia
Date submitted for ethics approval [1] 300458 0
21/10/2016
Approval date [1] 300458 0
15/05/2017
Ethics approval number [1] 300458 0
HREC/16/SCHN/383
Ethics committee name [2] 300468 0
Children and Adolescent Health Service Human Research Ethics Committee
Ethics committee address [2] 300468 0
GPO Box D184,
Perth, 6840,
Western Australia
Ethics committee country [2] 300468 0
Australia
Date submitted for ethics approval [2] 300468 0
02/02/2017
Approval date [2] 300468 0
10/08/2017
Ethics approval number [2] 300468 0
RGS0000000323

Summary
Brief summary
Selective dorsal rhizotomy (SDR) is a well-established neurosurgical intervention intended to permanently reduce spasticity in the lower limbs and improve mobility in selected children with cerebral palsy.
The aim of this study is to establish a retrospective and prospective national registry of relevant data pertaining to children undergoing SDR in Australia, and overseas (selection, intervention and outcome). Centres across Australia have agreed on a group of standardised assessments for children undergoing SDR both prior to and after the surgical procedure. The objective of collecting this data is to improve understanding of the short, medium and long term outcomes and any adverse effects of the intervention, and to provide clinicians with information to guide families considering this intervention.
Families of children who have undergone or are undergoing SDR (in Australia, and overseas) will be asked to consent to information relevant to SDR being stored in a research-specific online database. Information will be collected from the children's routine clinical assessments. Information will be stored without identifying details to improve privacy. Participation in this trial is voluntary.
Data collected in the registry will identify the characteristics of Australian children who have undergone SDR. In addition to details of the surgery including surgical complications; long term adverse events; and outcomes related to the International Classification of Functioning, Disability and Health domains of body structure and function, activity and participation.
The primary and secondary outcome measures have been developed for the CP and/or paediatric population and will be collected at baseline, and at one, two, five and ten years post intervention. These include gross motor function and mobility, achievement of goals, pain and quality of life. The need for further interventions after SDR surgery will be collected and include spasticity management and orthopaedic interventions in the ten years following SDR. The prevalence of any long term adverse effects will be collected in the ten years following SDR. The data collected pertaining to adverse events include spine and foot deformity, hip subluxation, sensory impairment and bladder/bowel dysfunction.
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 83726 0
Dr Simon Paget
Address 83726 0
Kids Rehab
The Children's Hospital at Westmead
Locked Bag 4001
Westmead NSW 2145
Country 83726 0
Australia
Phone 83726 0
+61 2 9845 0833
Fax 83726 0
+61 2 9845 0685
Email 83726 0
[email protected]
Contact person for public queries
Name 83727 0
Simon Paget
Address 83727 0
Kids Rehab
The Children's Hospital at Westmead
Locked Bag 4001
Westmead NSW 2145
Country 83727 0
Australia
Phone 83727 0
+61 2 9845 0833
Fax 83727 0
+61 2 9845 0685
Email 83727 0
[email protected]
Contact person for scientific queries
Name 83728 0
Simon Paget
Address 83728 0
Kids Rehab
The Children's Hospital at Westmead
Locked Bag 4001
Westmead NSW 2145
Country 83728 0
Australia
Phone 83728 0
+61 2 9845 0833
Fax 83728 0
+61 2 9845 0685
Email 83728 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
All data is de-identified and analysed as a multicentre cohort. No individual data will be made available. Results will be published in peer-reviewed journals and at local, National and International conferences.


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.