Clinical trial NCT05883969: GO-PLAY - Early Family-Centered Intervention for Infants With High-Risk of Cerebral Palsy
Official title: Early Family-Centered Intervention for Infants With High-Risk of Cerebral Palsy: Protocol for the Randomized Controlled GO-PLAY Trial
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: NA
Conditions: Cerebral Palsy
Lead sponsor: Rigshospitalet, Denmark
Sponsor class: OTHER
Collaborators: name: Aalborg University Hospital
name: Aarhus University Hospital
First posted: 2023-06-01
Enrollment: 60
Sex: ALL
Minimum age (years): 3
Maximum age (years): 12
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: Background. Early diagnosis of cerebral palsy (CP) is important to enable appropriate intervention at a time when neuroplasticity is at its highest. Early intervention with focus on family-centered, home-based, parent-involved, and supervised by specialist therapists show positive cognitive and motor outcomes. This study adhere to international guidelines for early diagnosis and intervention, and include community therapists to ensure regular follow-up during and after the intervention period.

The aim of the current study is to compare the effectiveness of an early intervention program added to standard care, relative to standard care alone, on the early motor development in children from both a newborn and infant detectable risk pathway in a Danish multi-site setting.

Methods. In a randomized, controlled trial the response to the GO-PLAY (Goal Oriented ParentaL supported home ActivitY) intervention program added to standard care is superior to standard care alone is evaluated. The investigators will include infants from the Cerebral Palsy - Early Diagnosis and Intervention Trial (CP-EDIT registered separately at ClinicalTrials) and collect data at baseline, after intervention and at follow up when the children are 2 years corrected age.

The hypotheses are that the GO-PLAY intervention is more effective than standard care when the children are re-evaluated at the end of 6 months of intervention and that the parents involved in the GO-PLAY intervention will exhibit less signs of stress and anxiety and perceive the services that they are receiving as family-centered to a greater extent than parents of children receiving standard care.

Discussion. Approximately half of all infants with high risk of CP display high risk indicators identifiable by early screening before 5 months of age described as the newborn detectable risk pathway. The other half of all infants with CP are detected by parents, caregivers or health care professionals when displaying delayed motor milestones (e.g. hand asymmetry or not sitting at 9 months) and described as infant detectable risk pathway. There is a need to investigate if early intervention is effective in all infants with high suspicion of CP, also the ones with unremarkable neonatal history. Further, a systematic early intervention has not been tested in infants at high risk of CP in Denmark, where public health services include physiotherapy free of charge for infants with CP.

Detailed description: Signs of CP may be visible already at three months of age but the mean diagnostic age is 13 months in Denmark. An early diagnosis is important in order to start relevant intervention when neuroplasticity is highest.

The study group will be recruited from the CP-EDIT, where both the newborn- and infant-detectable risk pathway from 3-12 months corrected age are included. Neonates may have obvious risk factors in the neonatal period, but in the present study infants with clinical findings suggesting CP emerging in the first year of life are also included. An interim diagnosis based on recommended predictive tools for early diagnosis; cerebral MRI, the General Movement Assessment (if \< 5 months) and the Hammersmith Infant Neurological Examination will be an inclusion criteria for this study. In addition the Hand Assessment for Infants will be included to assess early hand asymmetry.

An estimated sample size of 60 participants in total; approximately 30 per group is expected. The infants will be allocated to groups based on stratified permuted block randomization: Stratification will be used to achieve an explicit balance for age in months (\<6 months compared with ≥ 6 months) at study enrollment and gross motor development (HINE global score \> 40 ambulant and ≤ 40 non ambulant). Eligible participants will be randomly assigned in permuted blocks of 2 and 6, according to computer-generated random numbers, to either GO-PLAY or standard care. The investigators anticipate that allocation concealment will be successful in preventing selection bias since the statistician conceal the allocation sequence from those assigning participants to the intervention groups, until the moment of assignment is disclosed; from which point the individual is part of the intention-to-treat (ITT) population.

The infants will be assessed on the occasions; at baseline, post intervention and follow up at 24 months corrected age

Eligibility criteria: Inclusion Criteria:

Infants will be included in the intervention study, if they fulfil at least one of the requirements A or B:

A. any two of the following:

* Neuroimaging predictive of a motor disability including the involvement of one or more of the following structures: sensori-motor cortex, basal ganglia, posterior limb of the internal capsule, pyramidal tracts.
* General Movement Assessment with absent fidgety GMs at fidgety age
* HINE scores \<57 at 3months or \<60 at 6months or \<63 at 9 months or \<66 at 12 months

B. both of the following:

* Unilateral brain injury on neuroimaging (MRI or ultrasound) predictive of CP
* Clinical signs of asymmetry

Exclusion Criteria:

* Infants with progressive or neurodegenerative disorders or genetic disorders not associated with CP
* Infants with other disability diagnoses e.g. Down Syndrome.
* children for whom neither parent is fluent in Danish.

Primary outcomes:
measure: Peabody Developmental Motor Scales - Second edition (PDMS-2)
timeFrame: Change from baseline at 6 months
Secondary outcomes:
measure: Alberta Infant Motor Scale (AIMS)
timeFrame: Change from baseline at 6 months
measure: Gross Motor Function Measure (GMFM-66)
timeFrame: Change from baseline at 6 months (if > 5 months at inclusion)
measure: Hand Assessment in Infants (HAI)
timeFrame: Change from baseline at 6 months (if<12 months)
measure: The Parental Stress scale (PSS)
timeFrame: Change from baseline at 6 months
measure: The Depression Anxiety Stress Scales (DASS-21)
timeFrame: Change from baseline at 6 months
measure: The Measure of Processes of Care 20 (MPOC-20)
timeFrame: 6 months post baseline
Other outcomes:
measure: Peabody Developmental Motor Scales - Second edition (PDMS-2)
timeFrame: at 24 months corrected age
measure: Gross Motor Function Measure (GMFM-66)
timeFrame: at 24 months corrected age
measure: Bayley Scales of Infant and Toddler Development 4 (BSID-4) -cognitive
timeFrame: at 24 months corrected age
measure: The Ages & Stages Questionnaire (ASQ-3)
timeFrame: at 24 months corrected age
measure: The Parental stress scale (PSS)
timeFrame: at 24 months corrected age
measure: The Depression Anxiety Stress Scales (DASS-21)
timeFrame: at 24 months corrected age
measure: The Measure of Processes of Care 20 (MPOC-20)
timeFrame: at 24 months corrected age
measure: Measure of Processes of Care-Service Provider (MPOC-SP)
timeFrame: 6 months post baseline
measure: Measure of Processes of Care-Service Provider (MPOC-SP)
timeFrame: at 24 months corrected age
Locations:
city: Aalborg
country: Denmark
facility: Aalborg University Hospital
city: Aarhus
country: Denmark
facility: Aarhus University Hospital
Study officials:
affiliation: Department of Paediatrics, University Hospital Rigshospitalet, Denmark
name: Christina Hoi-Hansen, Professor
role: STUDY_CHAIR
References: