Trial Review

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Trial registered on ANZCTR


Registration number
ACTRN12610000323022
Ethics application status
Approved
Date submitted
31/03/2010
Date registered
21/04/2010
Date last updated
12/07/2012
Type of registration
Prospectively registered

Titles & IDs
Public title
Intrathecal baclofen therapy for management of spasticity: An Australian paediatric multicentre prospective audit.
Scientific title
Intrathecal baclofen therapy for management of spasticity: An Australian paediatric multicentre prospective audit.
Secondary ID [1] 1575 0
None
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Cerebral Palsy 257079 0
Spinal Cord Injury 257080 0
Condition category
Condition code
Neurological 257234 257234 0 0
Other neurological disorders

Intervention/exposure
Study type
Observational
Patient registry
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
Enrolment in this paediatric long term outcome audit will be offered to new patients offered ITB therapy.

We are collecting a minimum data set for children and teenagers receiving intrathecal baclofen (ITB) therapy in Australia.

We have applied The International Classification of Functioning, Disability and Health (ICF) model to assess the impact of ITB therapy upon participants.
The main domains of the ICF are:
1. Body Functions - physiological functions of body systems.
2. Body Structures - anatomical parts of the body such as organs, limbs and their components.
3. Activity is the execution of a task or action by an individual.
4. Participation is involvement in a life situation.

During the assessment phase individual therapy goals are set. The Canadian Occupational Measure (COPM) can be used to set the therapy goals and the Goal Attainment Scale (GAS).

Several outcome measures are being used to assess change in the ICF domains described above. These are fully discussed in the Outcomes section of this document.

On average 12 ITB pumps are implanted each year in Australian children. Therefore data, will be collected for 10 years.
Intervention code [1] 256247 0
Not applicable
Comparator / control treatment
nil
Control group
Uncontrolled

Outcomes
Primary outcome [1] 258128 0
Body Structure and Function 1

Communication
Changes in communication will be evaluated using three tools:
The Communication Function Classification System (CFCS).
Caregiver Priorities and Child Health Index of Life with Disabilities (CPCHILD)
Care and Comfort Hypertonicity Questionnaire.
Timepoint [1] 258128 0
Annually until transitioned to adult medical services.
Primary outcome [2] 258129 0
Body Structure and Function 2
Spasticity
Changes in spasticity will be evaluated using the Modified Ashworth Scale (MAS).
There are issues with using the MAS including:
- The procedure is poorly described.
- There is more than one criterion within each classification category for the scale.
- The scales use subjective terms such as more and slightly increased.
- There is a wide range of Inter-rater intraclass correlation coefficients (ICC).
With these numerous issues it has not been possible to develop a standardised way to use the MAS across the various centres. It is acknowledged that the MAS will be performed differently by the clinicians at each of the centres.
Timepoint [2] 258129 0
Annually until transitioned to adult medical services.
Primary outcome [3] 258130 0
Body Structure and Function 3
Dystonia
The Barry-Albright Dystonia (BAD) Scale is a reliable and responsive rating scale for secondary dystonia.
The BAD Scale is a five-point ordinal scale for secondary dystonia. It rates the severity of dystonia in eight body regions, including eyes, neck, mouth, trunk, upper limbs and lower limbs.
Timepoint [3] 258130 0
Annually until transitioned to adult medical services.
Secondary outcome [1] 263774 0
Body Structure and Function 4
Need for orthopaedic surgery
Theoretically, decreased spasticity from treatment with ITB therapy would reduce the occurrence of soft tissue contractures, prevent or delay bony deformities, and potentially impact the need for orthopaedic surgical interventions.
Specific musculoskeletal problems associated with spasticity include hip migration and scoliosis. The long term effect of ITB therapy on hip migration is poorly understood. The development of scoliosis following the introduction of ITB is an area of debate.
Participants will have annual spinal x-rays to monitor their cobb angles. Orthopaedic intervention, if required, will be documented.
Timepoint [1] 263774 0
Annually until transitioned to adult medical services.
Secondary outcome [2] 263866 0
Body Structure and Function 5
Medication Usage
The evaluation of changes of medication usage is important. Children with intractable spasticity are prescribed a variety of muscle relaxants and analgesic medications. Decreased oral medication usage is predicted.
The relationship between ITB therapy and seizures has been debated. There are a few anecdotal reports of increased seizure frequency after pump implantation, but no good data confirming such an increase, and in one pediatric multicenter study, no change in seizure frequency was observed. The introduction of new anticonvulsant medications will be collected.
Timepoint [2] 263866 0
Annually until transitioned to adult medical services.
Secondary outcome [3] 263867 0
Body Structure and Function 6
Pain assessment
The etiology of pain in children with chronic neurological disorders is multifactorial. Confounding factors such as reflux, constipation, feeding difficulties, positioning, spasticity, activities of daily living, hip pain and dental pain may all contribute to pain experienced by children and adolescents with chronic neurological disorders.
Accurate assessment of pain in children is challenging. Cognitive immaturity and inability to separate pain from fear and anxiety, and the subjectivity of pain all complicate assessment. Franck et al. suggest pain intensity can be described by a child with a cognitive age of 4 years. Several pain assessment scales have been developed that include self report, physiological measures and behavioural measures.
There is no easily administered technique for assessing pain directly in children. Houlihan et al. used the parent-report Child health Questionnaire (CHQ) to assess pain frequency in 198 children with cerebral palsy. 11% of patients reported pain almost every day. Pain was related to severity of motor impairment and the presence of a gastrostomy; parents of the most severely affected children reported the highest pain frequency. Significant consequences of pain included missed days of school and days in bed.
The Comfort/Emotions & Behaviour dimension of CPCHILD evaluates pain.
Timepoint [3] 263867 0
Annually until transitioned to adult medical services.
Secondary outcome [4] 263868 0
Activity 1
Mobility
Changes in participants mobility will be evaluated using three assessments, the Functional Mobility Scale (FMS)
The FMS was constructed to classify functional mobility in children, taking into account a range of assistive devices a child might use over three distances; 5 metres, 50 metres and 500 metres.
Gross Motor Function Classification System (GMFCS)
The GMFCS describes the gross motor function of children and youth with cerebral palsy on the basis of their self-initiated movement with particular emphasis on sitting, walking, and wheeled mobility. Distinctions between levels are based on functional abilities, the need for assistive technology, including hand-held mobility devices (walkers, crutches, or canes) or wheeled mobility, and to a much lesser extent, quality of movement.
1 minute walk test.
The 1 minute walk test is a measure of functional ability and walking endurance. Children are tested at their maximal walking speed.
Timepoint [4] 263868 0
Annually until transitioned to adult medical services.
Secondary outcome [5] 263869 0
Activity 2
Upper limb function
The impact of ITB therapy upon upper limb is important. This will be evaluated using the Manual Ability Classification System (MACS).
Timepoint [5] 263869 0
Annually until transitioned to adult medical services.
Secondary outcome [6] 263870 0
Activity 3
Activities of Daily Living
The Canadian Occupational Performance Measure (COPM) is used to:
- Identify problem areas in occupational performance
- Provide a rating of the client?s priorities in occupational performance
- Evaluate performance and satisfaction relative to those problem areas and
- Measure changes in a client's perception of his/her occupational performance
The COPM will be used to set Goal attainment scale (GAS) goals. The change in GAS goals will be used to document a change in the participants limitations in activities of daily living.
Timepoint [6] 263870 0
Annually until transitioned to adult medical services.
Secondary outcome [7] 263871 0
Participation 1
The Care & Comfort Hypertonicity Questionnaire (CCHQ) was developed to evaluate functional care needs, and to a lesser extent quality of life in children with increased tone of cerebral origin, particularly those with severe cerebral palsy. It is a self-report questionnaire and requires parents or caregivers to rate how easy or difficult it is for them or their child in the last two weeks to perform a range of tasks relative to a cooperative person without a disability
Timepoint [7] 263871 0
Annually until transitioned to adult medical services.

Eligibility
Key inclusion criteria
Enrolment in this paediatric long term outcome audit will be offered to new patients offered ITB therapy.

The decision whom is offered ITB therapy in Western Australia rests with Movement Disorder’s team at Princess Margaret Hospital. This decision is made following a multidisciplinary medical assessment process including a test dose of intrathecal baclofen.

Hospitals providing paediatric ITB programmes in Australia include Princess Margaret Hospital for Children (Perth), Royal Childrens Hospital (Melbourne), Monash Medical Centre (Melbourne), Westmead Childrens Hospital (Sydney), Women & Childrens Hospital (Adelaide) and the Royal Childrens Hospital (Brisbane).
Minimum age
No limit
Maximum age
18 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
There are no specific exclusion criteria.

Study design
Purpose
Screening
Duration
Longitudinal
Selection
Defined population
Timing
Prospective
Statistical methods / analysis

Recruitment
Recruitment status
Not yet recruiting
Date of first participant enrolment
Anticipated
26/04/2010
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
100
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)

Funding & Sponsors
Funding source category [1] 256745 0
Hospital
Name [1] 256745 0
Princess Margaret Hospital for Children
Country [1] 256745 0
Australia
Funding source category [2] 256746 0
Hospital
Name [2] 256746 0
The Children's Hospital at Westmead
Country [2] 256746 0
Australia
Funding source category [3] 256747 0
Hospital
Name [3] 256747 0
Royal Children's Hospital Brisbane
Country [3] 256747 0
Australia
Funding source category [4] 256748 0
Hospital
Name [4] 256748 0
Royal Children's Hospital Melbourne
Country [4] 256748 0
Australia
Funding source category [5] 256749 0
Hospital
Name [5] 256749 0
Victorian Paediatric Rehabilitation Service
Country [5] 256749 0
Australia
Funding source category [6] 256750 0
Hospital
Name [6] 256750 0
Women and Children's Hospital
Country [6] 256750 0
Australia
Primary sponsor type
Hospital
Name
Princess Margaret Hospital for Children
Address
Paediatric Rehabilitation
Roberts Rd
Subiaco
WA6008
Country
Australia
Secondary sponsor category [1] 256027 0
Hospital
Name [1] 256027 0
The Children's Hospital at Westmead
Address [1] 256027 0
Physical Disability Service
The Children's Westmead
Corner of Hawkesbury Road and Hainsworth Street
Westmead NSW 2145
Country [1] 256027 0
Australia
Secondary sponsor category [2] 256028 0
Hospital
Name [2] 256028 0
Royal Children's Hospital Brisbane
Address [2] 256028 0
Queensland Cerbral Palsy Health Service
Surgical Building, Floor 2
Royal Children's Hosptial
Heston Road
Brisbane QLD 4029
Country [2] 256028 0
Australia
Secondary sponsor category [3] 256029 0
Hospital
Name [3] 256029 0
Royal Children's Hospital Melbourne
Address [3] 256029 0
Department of Developmental Medicine
Royal Children's Hospital
Flemington Road
Parkville
Melbourne VIC 3052
Country [3] 256029 0
Australia
Secondary sponsor category [4] 256030 0
Hospital
Name [4] 256030 0
Victorian Paediatric Rehabilitation Service
Address [4] 256030 0
Victorian Paediatric Rehabilitation Service
Monash Medical Centre
246 Clayton Road
Clayton VIC 3168
Country [4] 256030 0
Australia
Secondary sponsor category [5] 256031 0
Hospital
Name [5] 256031 0
Women and Children's Hospital
Address [5] 256031 0
Paediatric Rehabilitation Department
Women and Children's Hospital Adelaide
72 King William Rd
Adelaide SA 5006
Country [5] 256031 0
Australia
Other collaborator category [1] 1180 0
Individual
Name [1] 1180 0
Dr Mary Clare Waugh
Address [1] 1180 0
Physical Disability Service
The Children's Westmead
Corner of Hawkesbury Road and Hainsworth Street
Westmead NSW 2145
Country [1] 1180 0
Australia
Other collaborator category [2] 1181 0
Individual
Name [2] 1181 0
Dr Lisa Copeland
Address [2] 1181 0
Queensland Cerbral Palsy Health Service
Surgical Building, Floor 2
Royal Children's Hosptial
Heston Road
Brisbane QLD 4029
Country [2] 1181 0
Australia
Other collaborator category [3] 1182 0
Individual
Name [3] 1182 0
Dr Giuliana Antolovich
Address [3] 1182 0
Department of Developmental Medicine
Royal Children's Hospital
Flemington Road
Parkville
Melbourne VIC 3052
Country [3] 1182 0
Australia
Other collaborator category [4] 1183 0
Individual
Name [4] 1183 0
Dr Barry Rawicki
Address [4] 1183 0
Victorian Paediatric Rehabilitation Service
Monash Medical Centre
246 Clayton Road
Clayton VIC 3168
Country [4] 1183 0
Australia
Other collaborator category [5] 1184 0
Individual
Name [5] 1184 0
Dr James Rice
Address [5] 1184 0
Paediatric Rehabilitation Department
Women and Children's Hospital Adelaide
72 King William Rd
Adelaide SA 5006
Country [5] 1184 0
Australia

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 258759 0
Princess Margaret Hospital for Children Ethics Committee Ethics Committee
Ethics committee address [1] 258759 0
Roberts Road
Subiaco
WA6008
Ethics committee country [1] 258759 0
Australia
Date submitted for ethics approval [1] 258759 0
02/03/2010
Approval date [1] 258759 0
20/04/2010
Ethics approval number [1] 258759 0
1797/EP
Ethics committee name [2] 258861 0
Princess Margaret Hospital for Children Ethics Committee (EC00268)
Ethics committee address [2] 258861 0
Ethics committee country [2] 258861 0
Date submitted for ethics approval [2] 258861 0
Approval date [2] 258861 0
Ethics approval number [2] 258861 0

Summary
Brief summary
Intrathecal baclofen (ITB) therapy is used to manage severe spasticity. ITB therapy is available to children and adolescents at 6 tertiary Paediatric hospitals in Australia. ITB is a new therapy, with 12-15 ITB pumps implanted in Australian children and adolescents each year. There is no standard pre assessment tool or follow up protocol for children and adolescents receiving ITB therapy in Australia.
The objective of this study is to identify and collect a minimum data set for assessment and follow up of children and adolescents receiving ITB therapy in Australia.
Two of the aims of this study are (1) Identifying which assessment tools are useful at assessing the effect of ITB upon your child’s daily functioning; and, (2) document the complications associated with ITB therapy.
For the purposes of data collection a prospective multicentre audit is proposed. This would include children attending Princess Margaret Hospital for Children (Perth), Royal Childrens Hospital (Melbourne), Monash Medical Centre (Melbourne), Westmead Childrens Hospital (Sydney), Women & Childrens Hospital (Adelaide) and the Royal Childrens Hospital (Brisbane). All Australian children and adolescents who are offered ITB therapy will be asked to enrol in this audit.
A standardised assessment protocol will be utilised by each centre to capture data across each domain of the World Health Organsiation international classification of functioning, disability and health.
Participation in this prospective audit reflects standard care and is voluntary.
Trial website
none
Trial related presentations / publications
none
Public notes

Contacts
Principal investigator
Name 31005 0
Address 31005 0
Country 31005 0
Phone 31005 0
Fax 31005 0
Email 31005 0
Contact person for public queries
Name 14252 0
Gavin Hutana
Address 14252 0
Department of Paediatric Rehabilitation
Princess Margaret Hospital for Children
Roberts Road
Subiaco
WA6008
Country 14252 0
Australia
Phone 14252 0
+61 8 9340 8886
Fax 14252 0
+61 8 9340 8001
Email 14252 0
[email protected]
Contact person for scientific queries
Name 5180 0
Gavin Hutana
Address 5180 0
Department of Paediatric Rehabilitation
Princess Margaret Hospital for Children
Roberts Road
Subiaco
WA6008
Country 5180 0
Australia
Phone 5180 0
+61 8 9340 8886
Fax 5180 0
Email 5180 0
[email protected]

No information has been provided regarding IPD availability


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

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