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Trial registered on ANZCTR


Registration number
ACTRN12618001564246
Ethics application status
Approved
Date submitted
13/09/2018
Date registered
19/09/2018
Date last updated
19/09/2018
Type of registration
Prospectively registered

Titles & IDs
Public title
Does integrating a parenting support program into management of phenylketonuria (PKU) improve treatment outcomes? A multiple baseline study.
Scientific title
Does integrating a parenting support program into management of phenylketonuria (PKU) improve treatment outcomes? A multiple baseline study.
Secondary ID [1] 295969 0
None
Universal Trial Number (UTN)
U1111-1220-0175
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Phenylketonuria (PKU) in children 309495 0
Parenting children with phenylketonuria (PKU) 309496 0
Condition category
Condition code
Metabolic and Endocrine 308332 308332 0 0
Metabolic disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
"Positive Parenting for Healthy Living Triple P" is a brief parent education and skills-training program that combines parenting information about child behaviour and illness management with the provision of illness relevant information. The intervention consists of two, 2-hour parent discussion group sessions designed to target the direct and indirect pathways in which parenting can impact on child outcomes - i.e. parenting practices, and parenting confidence and stress. Sessions are held one week apart and will be delivered either face-to-face or via telehealth. Program content draws on the theoretical principles that form the basis of the well-established Triple P program, which is a preventively orientated parenting and family support strategy derived from social-learning, functional analysis, and cognitive-behavioural principles. The intervention will be delivered by clinical psychologists and nurses who are accredited Triple P providers.
Intervention code [1] 312310 0
Behaviour
Comparator / control treatment
No control group
Control group
Uncontrolled

Outcomes
Primary outcome [1] 307314 0
Progress towards child behaviour goals, as assessed using the Goal Attainment Scale
Timepoint [1] 307314 0
Baseline, 4 weeks post-intervention (primary timepoint), and 4 month follow-up
Primary outcome [2] 307315 0
Child quality of life, as assessed using the PKU-specific Quality-of-Life questionnaire
Timepoint [2] 307315 0
Baseline, 4 weeks post-intervention (primary timepoint), and 4 month follow-up
Secondary outcome [1] 351432 0
Parenting behaviour, as assessed using the Parenting Scale
Timepoint [1] 351432 0
Baseline, 4 weeks post-intervention, and 4 month follow-up
Secondary outcome [2] 351433 0
Parent self-efficacy in managing child behaviour, as assessed using the Child Adjustment and Parent Efficacy Scale
Timepoint [2] 351433 0
Baseline, 4 weeks post-intervention, and 4 month follow-up
Secondary outcome [3] 351434 0
Child behaviour, as assessed using the Child Adjustment and Parent Efficacy Scale
Timepoint [3] 351434 0
Baseline, 4 weeks post-intervention, and 4 month follow-up
Secondary outcome [4] 351435 0
Parenting stress, as assessed using the Parenting Stress Index - Short Form
Timepoint [4] 351435 0
Baseline, 4 weeks post-intervention, and 4 month follow-up
Secondary outcome [5] 351440 0
Disease control: Blood phenylalanine levels will be assessed using Dried Blood Spot samples
Timepoint [5] 351440 0
Maximum four months prior to enrolment (requiring sufficient data to achieve stable baseline) to 4 month follow-up

Eligibility
Key inclusion criteria
Parents of 2- to 12-year-old children with PKU who require a protein restricted diet and supplementation of a phenylalanine-free amino acid formula and attending outpatient clinics at the Lady Cilento Children's Hospital (Brisbane).
Minimum age
18 Years
Maximum age
No limit
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
The child has a disability.
The child is under the care of Department of Communities (the State).
The parents have difficulties in reading an English newspaper.
The parents have a disability affecting cognition.
The parents are currently seeing a professional for the child’s behaviour difficulties.
The parents are currently receiving psychological help or counselling.
The parents have previously completed Triple P.

Study design
Purpose of the study
Educational / counselling / training
Allocation to intervention
Non-randomised 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
Who is / are masked / blinded?



Intervention assignment
Other design features
Phase
Type of endpoint(s)
Efficacy
Statistical methods / analysis
Due to small sample of families eligible to participate, data analyses will likely be underpowered and findings will be considered exploratory.

Correlations will examine relationships between key variables.
Repeated measures ANOVA or mixed-method repeated-measures (MMRM) linear regression models (depending on sample size), will assess change from baseline to 4 month follow up across the range of outcome measures used.

Recruitment
Recruitment status
Not yet recruiting
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)
QLD
Recruitment hospital [1] 11779 0
Lady Cilento Children's Hospital - South Brisbane
Recruitment postcode(s) [1] 23874 0
4101 - South Brisbane

Funding & Sponsors
Funding source category [1] 300566 0
Government body
Name [1] 300566 0
Children’s Health Queensland - Centre for Clinical Trials in Rare Neurodevelopmental Disorders
Address [1] 300566 0
62 Graham Street
South Brisbane QLD 4101
Country [1] 300566 0
Australia
Primary sponsor type
Individual
Name
Ms Anita Inwood
Address
Queensland Lifespan Metabolic Medicine Service
Lady Cilento Children's Hospital
501 Stanley Street
South Brisbane QLD 4101
Country
Australia
Secondary sponsor category [1] 300058 0
Individual
Name [1] 300058 0
Dr Amy Mitchell
Address [1] 300058 0
Parenting and Family Support Centre
School of Psychology
The University of Queensland
13 Upland Road
St Lucia QLD 4072
Country [1] 300058 0
Australia
Secondary sponsor category [2] 300075 0
Individual
Name [2] 300075 0
A/Prof Alina Morawska
Address [2] 300075 0
Parenting and Family Support Centre
School of Psychology
The University of Queensland
13 Upland Road
St Lucia QLD 4072
Country [2] 300075 0
Australia
Secondary sponsor category [3] 300076 0
Individual
Name [3] 300076 0
Prof David Coman
Address [3] 300076 0
Queensland Lifespan Metabolic Medicine Service
Lady Cilento Children's Hospital
501 Stanley Street
South Brisbane QLD 4101
Country [3] 300076 0
Australia

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 301357 0
Children's Health Queensland Hospital and Health Service Human Research Ethics Committee
Ethics committee address [1] 301357 0
Level 7, Centre for Children’s Health Research
Lady Cilento Children’s Hospital Precinct
62 Graham Street
South Brisbane QLD 4101
Ethics committee country [1] 301357 0
Australia
Date submitted for ethics approval [1] 301357 0
14/06/2018
Approval date [1] 301357 0
26/07/2018
Ethics approval number [1] 301357 0
HREC/18/QRCH/162
Ethics committee name [2] 301370 0
The University of Queensland Human Research Ethics Committee
Ethics committee address [2] 301370 0
Cumbrae-Stewart Building
The University of Queensland
St Lucia QLD 4072
Ethics committee country [2] 301370 0
Australia
Date submitted for ethics approval [2] 301370 0
03/08/2018
Approval date [2] 301370 0
08/08/2018
Ethics approval number [2] 301370 0
2018001620

Summary
Brief summary
Phenylketonuria (PKU) is a rare Inborn Error of Metabolism (IEM) with an incidence of approximately 1 in 10 - 15,000. Prior to the advent of newborn screening and effective dietary treatments, PKU caused a severe neurodevelopmental phenotype marked by global developmental delay, severe intellectual impairment, and seizures. Early diagnosis from newborn screening and introductions of dietary interventions prevent these severe neurological manifestations. Despite this, neuropsychological complications of PKU are observed when control is suboptimal, which include reduced executive function, attention deficit, decreased processing speed, cognitive impairment and disturbances in emotion and behaviour. Neuro-disabilities are thus a key phenotype amongst the PKU cohort when their metabolic control is suboptimal. This project is aimed at exploring practical tools to improve our management outcomes for children and families living with this rare chronic disease. We hypothesise that participating in a brief evidence-based parenting intervention (Triple P), when compared to baseline, will improve progress towards child behaviour/parenting goals, improve children’s quality of life, decrease parent-reported use of ineffective parenting practices, increase parent self-efficacy, reduce parent-reported child behavioural problems, reduce parenting stress, and improve children’s blood Phe levels.
Trial website
https://exp.psy.uq.edu.au/pku/
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 86730 0
Ms Anita Inwood
Address 86730 0
Queensland Lifespan Metabolic Medicine Service
Lady Cilento Children’s Hospital
South Brisbane, QLD, 4101
Country 86730 0
Australia
Phone 86730 0
+61 7 3068 4426
Fax 86730 0
Email 86730 0
anita.inwood@health.qld.gov.au
Contact person for public queries
Name 86731 0
Ms Anita Inwood
Address 86731 0
Queensland Lifespan Metabolic Medicine Service
Lady Cilento Children’s Hospital
South Brisbane, QLD, 4101
Country 86731 0
Australia
Phone 86731 0
+61 7 3068 4426
Fax 86731 0
Email 86731 0
anita.inwood@health.qld.gov.au
Contact person for scientific queries
Name 86732 0
Ms Anita Inwood
Address 86732 0
Queensland Lifespan Metabolic Medicine Service
Lady Cilento Children’s Hospital
South Brisbane, QLD, 4101
Country 86732 0
Australia
Phone 86732 0
+61 7 3068 4426
Fax 86732 0
Email 86732 0
anita.inwood@health.qld.gov.au

No data has been provided for results reporting
Summary results
Not applicable