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Trial registered on ANZCTR


Registration number
ACTRN12616000366459
Ethics application status
Approved
Date submitted
7/03/2016
Date registered
22/03/2016
Date last updated
3/05/2019
Date data sharing statement initially provided
3/05/2019
Date results information initially provided
3/05/2019
Type of registration
Prospectively registered

Titles & IDs
Public title
A pilot study of gabapentin for managing pain in children with dystonic cerebral palsy
Scientific title
A pilot study of gabapentin for managing pain in children with dystonic cerebral palsy
Secondary ID [1] 288697 0
Nil known
Universal Trial Number (UTN)
U1111-1180-4442
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Dystonic cerebral palsy 297917 0
Condition category
Condition code
Physical Medicine / Rehabilitation 298081 298081 0 0
Other physical medicine / rehabilitation
Anaesthesiology 298082 298082 0 0
Pain management
Neurological 298103 298103 0 0
Other neurological disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Intervention: Gabapentin
Dose administered: The initial dosing regime will be the same for all participants. For all children, the dosage will be increased over 5 weeks as stated below:
Week 1 – 100 mg daily
Week 2 – 100 mg twice daily
Week 3 - 200 mg twice daily
Week 4 – 300 mg twice daily
Week 5 – 300 mg three times a day
However there is the potential for the dose to be escalated beyond this to a maximum of 50 mg/kg/day for heavier children, or for those children who have had no change in symptoms and have tolerated the medication. Dosage regime and escalation will be managed by the study doctor allocated to each participant.
Duration of administration: 12 weeks
Mode of administration: Orally, and for children who have a feeding tube in situ, the capsule contents will be dissolved in water and given via a gastrostomy, nasojejunal, gastrojejunal or nasogastric tube.
Weekly phone calls to the study participants will be made by the study doctor to ensure that the dosage regime is being followed and to monitor tolerance to the medication.
The hospital pharmacy will dispense the medication and will only provide families with the amount of medication required to take them to the next study assessment visit, whereby they will be provided with more depending on the dosage at that time point. Pharmacy will maintain records regarding when and how much study medication is dispensed by each patient in the study. Parents will be asked to return all unused capsules at each clinic visit. Reasons for departure from the expected dispensing regimen will be recorded. Any discrepencies will be investigated.
Intervention code [1] 294123 0
Treatment: Drugs
Comparator / control treatment
No control group
Control group
Uncontrolled

Outcomes
Primary outcome [1] 297597 0
This is a feasibility pilot study, and the main objective is to gather preliminary data on gabapentin for managing pain in children with dystonic CP to inform a future RCT. This will include trialling the recruitment process. There will be a composite feasibility outcome of recruitment number, recruitment rate and retention rate, all assessed by review of study records.
Timepoint [1] 297597 0
At conclusion of study - 12 weeks
Primary outcome [2] 297677 0
Complications and side effects of the medication, will be assessed by review of daily medication logs completed by parents. For example, possible side-effects such as drowsiness and confusion, upset stomach, weakness, mood or behaviour changes will be recorded by parents on the daily medication diaries.
Timepoint [2] 297677 0
At conclusion of study - 12 weeks (but side-effects and complications and feedback from parents will all be closely monitored by the study doctors on a weekly basis).
Secondary outcome [1] 321538 0
Pain Measured by: Faces Pain Scale – Revised (FPS-R), Pediatric Pain Profile (PPP), Weekly pain diary
Timepoint [1] 321538 0
Baseline, 6 weeks, and 12 weeks
Secondary outcome [2] 321540 0
Comfort
Measured by:
Care and Comfort Hypertonicity Questionnaire (CCHQ)
The Caregiver Priorities and Child Health Index of Life with Disabilities (CPCHILD)
Timepoint [2] 321540 0
Baseline, 6 weeks, and 12 weeks
Secondary outcome [3] 321541 0
Dystonia
Measured by:
Barry Albright Dystonia (BAD) Scale
Health Utilities Index 3 (HUI 3) Multi-Attribute Health Status Classification System
Timepoint [3] 321541 0
BAD Scale - Baseline, 6 weeks, and 12 weeks.
HUI-3 - Screening/baseline, and 12 weeks

Eligibility
Key inclusion criteria
1) Confirmation of dystonia after completion of the Hypertonicity Assessment Tool (HAT). Those children who have dystonia or a mixed diagnosis (spasticity and dystonia) will be eligible.
2) Severity of dystonia will be determined with the Barry Albright Dystonia (BAD) scale. Children with a score of 15 or higher on the BAD scale will be eligible.
3) Pain will be quantified using the Health Utilities Index 3 (HUI 3). Those children scoring IV or V will be eligible.
Minimum age
6 Years
Maximum age
18 Years
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Children excluded from the study will be those who:
1. Are younger than 6 years because they will have more difficulty with reliably self-reporting and will not be able to perform some of the quantitative measurements.
2. Are currently on gabapentin or have been on gabapentin in the previous 3 months
3. Have demonstrated hypersensitivity to gabapentin or the inactive ingredients in the capsules in the past
4. Are currently taking other medications that may interact with gabapentin (i.e. antacid, cimetidine, morphine, and opioids).
5. Pregnant female participants

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Allocation is not concealed
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
Type of endpoint(s)
Efficacy
Statistical methods / analysis
This study is a pilot to primarily examine feasibility and does not require a power calculation. Descriptive statistics for baseline participant characteristics will include mean age and range and spread of severity of CP as described by the GMFCS, MACS and CFCS. Individual change scores on the COPM, CP CHILD, CCHQ, PPP and BAD will be calculated. The range of change scores for each outcome measure will be reported for the group. Variation in effect sizes for individuals and magnitude in responses will be established. Information about side effects/adverse events, compliance and parental and child acceptance of the treatment will be described. Data analysis will help to inform sample sizes and inclusion criteria for a future trial.


Recruitment
Recruitment status
Completed
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 5396 0
The Royal Childrens Hospital - Parkville
Recruitment hospital [2] 8233 0
Lady Cilento Children's Hospital - South Brisbane
Recruitment hospital [3] 8234 0
Womens and Childrens Hospital - North Adelaide
Recruitment hospital [4] 8235 0
The Children's Hospital at Westmead - Westmead
Recruitment postcode(s) [1] 12848 0
3052 - Parkville
Recruitment postcode(s) [2] 16291 0
4101 - South Brisbane
Recruitment postcode(s) [3] 16292 0
5006 - North Adelaide
Recruitment postcode(s) [4] 16293 0
2145 - Westmead

Funding & Sponsors
Funding source category [1] 293058 0
Other
Name [1] 293058 0
Murdoch Childrens Research Institute
Address [1] 293058 0
Royal Children's Hospital
Flemington Road
Parkville VIC 3052
Country [1] 293058 0
Australia
Funding source category [2] 296642 0
Charities/Societies/Foundations
Name [2] 296642 0
Australian Pain Society
Address [2] 296642 0
Suite 103, Level 1
3-5 West Street
North Sydney NSW 2060
Country [2] 296642 0
Australia
Primary sponsor type
Other
Name
Murdoch Childrens Research Institute
Address
Royal Children's Hospital
Flemington Road
Parkville VIC 3052
Country
Australia
Secondary sponsor category [1] 291837 0
None
Name [1] 291837 0
Address [1] 291837 0
Country [1] 291837 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 294566 0
Drug Trial Subcommittee at the Royal Children's Hospital Melbourne
Ethics committee address [1] 294566 0
Flemington Road
Parkville VIC 3052
Ethics committee country [1] 294566 0
Australia
Date submitted for ethics approval [1] 294566 0
02/02/2016
Approval date [1] 294566 0
30/05/2016
Ethics approval number [1] 294566 0

Summary
Brief summary
The primary objective of this feasibility pilot study is to gather preliminary data on the use of gabapentin to reduce pain in children with dystonic CP to inform a future RCT including trialling the recruitment process and retention rate, the outcome measures, and to identify complications and side effects from the medication which could impact on the design of an RCT. The study will specifically examine the variability of results for different outcome measures to determine effect sizes that will be used to inform the planning of a future trial (choice of primary outcome measure, sample size planning).
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 64174 0
Dr Adrienne Harvey
Address 64174 0
Developmental Disability and Rehabilitation Research Group
Murdoch Childrens Research Institute
Royal Children's Hospital
Flemington Road
Parkville VIC 3052
Country 64174 0
Australia
Phone 64174 0
+61 3 9345 7540
Fax 64174 0
Email 64174 0
adrienne.harvey@mcri.edu.au
Contact person for public queries
Name 64175 0
Dr Adrienne Harvey
Address 64175 0
Developmental Disability and Rehabilitation Research Group
Murdoch Childrens Research Institute
Royal Children's Hospital
Flemington Road
Parkville VIC 3052
Country 64175 0
Australia
Phone 64175 0
+61 3 9345 7540
Fax 64175 0
Email 64175 0
adrienne.harvey@mcri.edu.au
Contact person for scientific queries
Name 64176 0
Dr Adrienne Harvey
Address 64176 0
Developmental Disability and Rehabilitation Research Group
Murdoch Childrens Research Institute
Royal Children's Hospital
Flemington Road
Parkville VIC 3052
Country 64176 0
Australia
Phone 64176 0
+61 3 9345 7540
Fax 64176 0
Email 64176 0
adrienne.harvey@mcri.edu.au

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment
Results will be reported as a group
What supporting documents are/will be available?
No other documents available
Summary results
Have study results been published in a peer-reviewed journal?
No
Other publications
Have study results been made publicly available in another format?
Yes
Other publication details
Citation type [1] 1987 0
Conference poster
Citation/DOI/link/details [1] 1987 0
Results were presented at the Australian Pain Society meeting in April 2019 through a scientific poster.
A manuscript will be submitted for publication in a peer reviewed journal in the next 3 months
Attachments [1] 1987 0
Results – basic reporting
Results – plain English summary