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Trial registered on ANZCTR


Registration number
ACTRN12607000484448
Ethics application status
Approved
Date submitted
6/09/2007
Date registered
21/09/2007
Date last updated
21/09/2007
Type of registration
Retrospectively registered

Titles & IDs
Public title
The effect of botulinum toxin-A on the functional ability of the very young child with spastic hemiplegia due to cerebral palsy.
Scientific title
A randomised controlled trial to determine the effects of botulinum toxin-A on the functional ability of the very young child with spastic hemiplegia due to cerebral palsy.
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
spasticity in children with cerebral palsy 2341 0
Condition category
Condition code
Neurological 2445 2445 0 0
Other neurological disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
botulinum toxin-A (BOTOX) to treat spastic equinus by intramuscular injection; 6-12 U/kgtotal body weight; repeat injections at no less than six monthly intervals beginning at the motor stage of "pull to stand"
Intervention code [1] 2061 0
Treatment: Drugs
Comparator / control treatment
Standard best practice treatment: Botulinum toxin -A (BOTOX) to treat spastic equinus by intramuscular injection; 6-12 U/kgtotal body weight; repeat injections at no less than six monthly intervals beginning after two years of age AND when ambulant
Control group
Active

Outcomes
Primary outcome [1] 3345 0
quality of gait measured by Physicians Rating Scale (PRS), Toddler and Infant Motor Evaluation (TIME) and Gross Motor Performance Measure (GMPM)
Timepoint [1] 3345 0
3 monthly from time of independent walking until three years of age
Primary outcome [2] 3346 0
amount of dynamic calf muscle spasticity measured by Modified Tardieu Scale(MTS) and Modifie4d Ashworth Score (MAS)
Timepoint [2] 3346 0
at 0, 1, 3, 6, 7, 9, 12, 13, 15, 18, 19, 21, 24, 25, 17, 30, 31 months
Primary outcome [3] 3347 0
gross motor function as measured by the Gross Motor Function Measure (GMFM), and the Toddler and Infant Motor Evaluation (TIME)
Timepoint [3] 3347 0
0, 6, 12, 18, 24 months after motor stage of "pull to stand"
Secondary outcome [1] 5562 0
upper limb function measured by Assisting Hand Assessment (AHA)
Timepoint [1] 5562 0
at 3 years of age

Eligibility
Key inclusion criteria
spastic hemiplegia due to CP
Minimum age
9 Months
Maximum age
22 Months
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
already independently ambulating;
concurrent unrelated disability or serious long term illness

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
concealed allocation by numbered containers
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
a form of dynamic, block randomisation (minimisation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Efficacy
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Date of first participant enrolment
Anticipated
1/10/2002
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
30
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)

Funding & Sponsors
Funding source category [1] 2599 0
Commercial sector/Industry
Name [1] 2599 0
Allergan Australia Pty Ltd
Country [1] 2599 0
Australia
Primary sponsor type
Individual
Name
Sarah Love
Address
Physiotherapy Department
Princess Margaret Hospital
GPO Box D184 PERTH WA 6840
Country
Australia
Secondary sponsor category [1] 2351 0
Hospital
Name [1] 2351 0
Princess Margaret Hospital
Address [1] 2351 0
GPO Box D184 PERTH WA 6840
Country [1] 2351 0
Australia
Other collaborator category [1] 31 0
University
Name [1] 31 0
Curti University of Technology
Address [1] 31 0
GPO Box U1987
PERTH WA 6840
Country [1] 31 0
Australia
Other collaborator category [2] 32 0
Individual
Name [2] 32 0
AProf Eve Blair
Address [2] 32 0
ICHR
Roberts Road Subiaco WA6008
Country [2] 32 0
Australia
Other collaborator category [3] 33 0
Individual
Name [3] 33 0
Prof Joan Cole
Address [3] 33 0
c/Physio department
Princess Margaret Hospital
Country [3] 33 0
Australia

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 4518 0
Curtin University HREC
Ethics committee address [1] 4518 0
as above
Ethics committee country [1] 4518 0
Australia
Date submitted for ethics approval [1] 4518 0
Approval date [1] 4518 0
10/03/2002
Ethics approval number [1] 4518 0
CUT HR 9/2002
Ethics committee name [2] 4519 0
Womens and Children's Health Service HREC
Ethics committee address [2] 4519 0
Princess Margaret Hospital
Ethics committee country [2] 4519 0
Australia
Date submitted for ethics approval [2] 4519 0
Approval date [2] 4519 0
10/03/2002
Ethics approval number [2] 4519 0
PMH 633/ep

Summary
Brief summary
Cerebral palsy (CP) is a disorder affecting around 2.5 in 1000 live births. Commonly, children with CP have spasticity in their muscles causing stiffness. Children with spasticity move differently because of this stiffness. When botulinum toxin-A (BOTOX) is injected into a spastic muscle, it helps relax the muscle for around three months, after which the effect begins to wear off. During this time, the muscle is able to move more freely through a greater range, allowing the child to use more normal patterns of muscle recruitment.

BOTOX is recommended and PBS rebatable for children with CP who are over two years of age and who are ambulant. Usually, by two years of age these children have developed an abnormal pattern of movement, especially around the ankle.

Currently, the most important clinical question is whether BOTOX should be used to prevent this early imperfect standing and walking practice rather than be used after two years of age to correct the problem that has developed. Injections of BOTOX at a younger age may provide the child with an early sensation of more normal movement and lay down patterns of movement that will be useful in the long term.

The aim of this study is to compare, over a three year period, the effects of early (beginning at the motor stage of "pull-to-stand") repeated BOTOX injections on gross motor function and the development of walking with the effects of later ( beginning after two years of age) standard repeated BOTOX treatment. The main measures will be the gross motor function measures and quality of walking. Measurements (such as muscle spasticity, muscle length, parent questionnaires and functional ability of the upper limb) will also be made to determine which program most positively affects walking, other gross motor functions and /or quality of life.
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 28036 0
Address 28036 0
Country 28036 0
Phone 28036 0
Fax 28036 0
Email 28036 0
Contact person for public queries
Name 11193 0
Sarah Love
Address 11193 0
Physiotherapy Department
Princess Margaret Hospital
Roberts Road Subiaco 6008 WA
Country 11193 0
Australia
Phone 11193 0
+61 8 9340 8503
Fax 11193 0
+61 8 9340 8597
Email 11193 0
[email protected]
Contact person for scientific queries
Name 2121 0
Sarah Love
Address 2121 0
Physiotherapy Department
Princess Margaret Hospital
Roberts Road Subiaco 6008 WA
Country 2121 0
Australia
Phone 2121 0
+61 8 9340 8503
Fax 2121 0
+61 8 9340 8597
Email 2121 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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