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Trial registered on ANZCTR


Registration number
ACTRN12609000360213
Ethics application status
Approved
Date submitted
16/03/2009
Date registered
26/05/2009
Date last updated
24/02/2017
Type of registration
Retrospectively registered

Titles & IDs
Public title
Evaluation of the effects of botulinum toxin A injections in improving ease of care and increasing comfort in children with marked cerebral palsy: a randomised controlled trial.
Scientific title
The effects of botulinum toxin A injections on ease of care and comfort in children with marked cerebral palsy
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Marked cerebral palsy:Gross Motor Function Classification System (GMFCS) 4-5 4479 0
Condition category
Condition code
Musculoskeletal 4754 4754 0 0
Normal musculoskeletal and cartilage development and function

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Botulinum toxin A injections to clinically selected muscles, occupational therapy, physiotherapy.
Dose: Total dose not to exceed 12 units per kg or 400 units total. Dosage per muscle group:
Upper limb
Intrinsic muscles hand: 1/2 unit per kg or maximum 20 units per muscle.
Finger flexors: 1/2 - 1 unit per kg or maximum 30 units.
Wrist flexors:1/2 - 1 unit per kg or maximum 30 units.
Pronators: 1/2 - 1 unit per kg or maximum 30 units.
Elbow flexors: 1-2 units per kg or maximum 60 units.
Pectoral muscles, lattisimus dorsi, teres major: 1/2 - 2 units per kg or maximum 60 units per muscle group.
Deltoid: 1 unit per kg or maximum 50 units.
Lower limb
Adductors: 2-4 units per kg maximum dose 80 units.
Hamstrings: 2-4 units per kg with maximum dose 80 units.
Quadriceps:2-4 units per kg maximum dose 80 units.
Gastrocnemius: 2-4 units per kg maximum dose 100 units.
Maximum dose 50 units per site. Any injection > 50 units requires more than one injection site. All injections are intramuscular with ultrasound and/or nerve stimulator guidance.
Injections (or placebo) occur at baseline. Injections are repeated at 6 months.
children will all receive physical therapy and occupational therapy following injections/ sham procedure for up to 6 weeks on a weekly basis. Therapy will include serial casting for maximum 2 weeks as required. Occupational therapy and or physiotherapy requirements are determined on patient goals by blinded therapist.
Injections will be repeated at a 6 month interval.
Analgesia for injections will be provided with intranasal fentanyl and topical anaesthetic.
Intervention code [1] 4225 0
Treatment: Drugs
Comparator / control treatment
Children in the control group will have sham treatment with simulated analgesia (intranasal administration of saline rather than fentanyl) and injections. Sham injections will occur once only. Sham injections will be performed by nurse, child and parent being screened from view of injection sites. Ultrasound localisation of muscles will be performed. Blunt needle tip will then be placed against skin to simulate needle injections. No substance will be injected. All children will receive occupational therapy and physiotherapy following the intervention for a period of 6 weeks. Therapy will occur weekly for 6 weeks.
Control group
Placebo

Outcomes
Primary outcome [1] 5620 0
Parents will report burden of care as measured by the Care and comfort hypertonicity questionnaire
Timepoint [1] 5620 0
baseline, one month, four months following completion of the first treatment. Injections are repeated at 6 months (second baseline) with repeat measures at one month and four months following second treatment.
Primary outcome [2] 5621 0
Parents will report overall satisfaction and performance for children in individually identified problem areas as determined by the Canadian occupational performance measure
Timepoint [2] 5621 0
baseline, at one month and four months. injections are repeated at 6 months with repeat measures at one month and four months following second treatment.
Secondary outcome [1] 9460 0
Parents will report quality of life as determined by the cerebral palsy (CP) quality of life tool: Cerebral Palsy Quality of Life (CPQoL) and child's health status as determined by health utility measure CP child.
Timepoint [1] 9460 0
baseline, one monthand four months following the first treatment. Injections are repeated at 6 months with repeat measures at one month and four months following this treatment.
Secondary outcome [2] 9461 0
Patients and parents will report a pain as recorded by the paediatric pain profile
Timepoint [2] 9461 0
baseline, one month, four month following initial injections. injections are repeated 6 months following initial injections (second baseline) with review points at one month and four months following these second injections.

Eligibility
Key inclusion criteria
Age 2-16 years, diagnosis of cerebral palsy classified GMFCS 4-5. Child/family have goals primarily concerned with improving care and/or comfort associated with spasticity affecting upper and/or lower limbs.
Minimum age
2 Years
Maximum age
16 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Medical contraindications to botulinum toxin A. Any botulinum toxin A injections occurring within 6 months of the study, any upper limb or lower limb casting within 2 months of initiation of the study, changes to oral or other spasticity management agents occuring within 2 months.

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)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?



Intervention assignment
Parallel
Other design features
Phase
Phase 4
Type of endpoint/s
Efficacy
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Date of first participant enrolment
Anticipated
16/03/2009
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] 4670 0
Hospital
Name [1] 4670 0
Qld Cerebral Palsy Health Service
Country [1] 4670 0
Australia
Primary sponsor type
Hospital
Name
Qld Cerebral Palsy Health Service
Address
Royal Childrens Hospital Brisbane
Herston Rd Herston Queensland
4029
Country
Australia
Secondary sponsor category [1] 4216 0
Charities/Societies/Foundations
Name [1] 4216 0
Royal Children's hospital Foundation
Address [1] 4216 0
C/O Royal childrens Hospital, Herston Rd Herston Brisbane Queensland 4029
Country [1] 4216 0
Australia

Ethics approval
Ethics application status
Approved

Summary
Brief summary
Children with the more severe forms of cerebral palsy often have difficulties with pain or discomfort associated with spasticity. Spasticity can also impact upon the burden of care parents experience. Botulinum toxin A has been used for over a decade in the treatment of spasticity for children with cerebral palsy however most research to date has focussed on functional gains for children with the less severe forms of cerebral palsy. this study hypothesises that botulinum toxin A injections when combined with a therapy program improves outcomes of burden of care and comfort for children with severe cerebral palsy when compared to a therapy program alone.
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 29400 0
Address 29400 0
Country 29400 0
Phone 29400 0
Fax 29400 0
Email 29400 0
Contact person for public queries
Name 12647 0
Dr Lisa Copeland
Address 12647 0
Qld Cerebral Palsy Health Service. Level 2 Surgical Building. Royal childrens hospital Brisbane. Herston Rd Herston Qld 4029
Country 12647 0
Australia
Phone 12647 0
+61 7 36365500
Fax 12647 0
+61 7 36365480
Email 12647 0
[email protected]
Contact person for scientific queries
Name 3575 0
Dr Lisa Copeland
Address 3575 0
Qld Cerebral Palsy Health Service. Level 2 Surgical Building. Royal childrens hospital Brisbane. Herston Rd Herston Qld 4029
Country 3575 0
Australia
Phone 3575 0
+61 7 36365500
Fax 3575 0
+61 7 36365480
Email 3575 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

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

Documents added automatically
No additional documents have been identified.