Trial Review

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Trial registered on ANZCTR


Registration number
ACTRN12623000600640
Ethics application status
Approved
Date submitted
13/04/2023
Date registered
1/06/2023
Date last updated
16/11/2023
Date data sharing statement initially provided
1/06/2023
Type of registration
Retrospectively registered

Titles & IDs
Public title
The PEPI ARC study: Feasibility study for early detection of cerebral palsy in pre term infants
Scientific title
Partnering Early to Provide for Infants At Risk of Cerebral palsy (PEPI ARC)-
Evaluating the feasibility of implementing the New Zealand Best Practice Recommendations for Early diagnosis of Cerebral Palsy through a regional early diagnosis Hub.
Secondary ID [1] 309381 0
None
Universal Trial Number (UTN)
U1111-1290-7890
Trial acronym
PEPI ARC - Partnering Early to Provide for Infants At Risk of Cerebral palsy
Linked study record

Health condition
Health condition(s) or problem(s) studied:
cerebral palsy 329607 0
Condition category
Condition code
Neurological 326534 326534 0 0
Other neurological disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
The PEPI ARC seeks to be a central hub for information sharing and education around assessments for early detection of cerebral palsy (CP) and diagnosis. The PEPI ARC Hub will be delivered using two components: a community based, multidisciplinary face-to-face diagnostic clinic in Wellington, and a virtual hub to support regional CP-specific neurodevelopmental follow up, education and information sharing and foster relationships between the Wellington team, local community and regional Child Development Service (CDS) and paediatricians.

All infants enrolled in PEPI ARC will receive standard care while admitted to the Neonatal Intensive Care Unit (NICU) in Wellington. In addition, whanau will be provided with a neonatal ‘passport’ to help improve information sharing between families and health care providers during transition of care from NICU to regional centres and follow-up services, and to standardise the use of CP diagnostic tests.

Face to face clinic:
Will be offered to whanau living in the Wellington region for medical, growth and neurodevelopmental assessments between 12-16 weeks corrected gestational age (CGA). This will replace the appointment the infant would have with their neonatologist at this age. Clinic appointments will take place in a community setting and will last for approximately 90 minutes. The multidisciplinary team (MDT) will consist of a neonatologist or developmental paediatrician trained in General Movement Assessment (GMA), a therapist trained in GMA and Hammersmith Infant Neurological Examination (HINE), a social worker and a clinic nurse. Infants may also be able to access Speech and Language Therapist (SLT)/dietician assessments if clinically indicated.

If a whanau would like to attend the clinic but are unable to; the PEPI ARC team will offer an MDT assessment using telehealth in conjunction with a home visit from their Visiting Neurodevelopmental Therapist (VNDT). The family will be invited to film and forward a GMA video for assessment prior to the visit. The HINE can then be performed by the VNDT and the PEPI ARC team can collate and feedback the outcome of assessment.

All infants will have their development, growth and general health assessed, be referred to diagnostic and supportive services if concerns or delays are detected. For all infants seen in the PEPI ARC clinic, a formal clinic letter will be sent to the whanau and their local health care team. At the end of the clinic appointment, the assessment findings and relevant information and support will be shared with the whanau regarding the likelihood of CP.

Virtual hub
For whanau living outside the Wellington region, the virtual hub will provide flexible support for families, paediatricians and therapists to aid early assessment follow up prior to 5 months corrected age. The Hub will offer:

Families/whanau:
- A neonatal neurodevelopmental passport to improve information sharing between families and health care providers during transition of care from NICU to regional centres and follow-up services, and to standardise CP-specific assessments.
- Education and information about filming GMA videos and transferring them securely to the PEPI ARC team. Includes infographic and informatio sheets (electronic copies and paper form available from regional and PEPI ARC team) in Te Reo and English explaining the process.
- Reporting of GMA videos and direct communication of results to whanau.
- A dedicated email address to contact the PEPI ARC team for advice and support if required.
- CP information package including how to access peer-to–peer support and other support services if their child is diagnosed with CP or high-risk of CP. Includes electronic and paper copies of information sheets available from regional and PEPI ARC team.

For paediatricians/therapists:
- Education sessions on early CP diagnosis, assessment tools and interpretation of results.
- Reporting and peer-review of GMA videos.
- Assistance with collating and assessing the likely diagnosis based on the triangulation of the HINE/GMA/neuroimaging results.
- Assistance with communicating results of assessment to families.
- Advice/Information about:
Early intervention
Surveillance of comorbidities and complications of CP
Communicating a diagnosis of CP
Parental support
- Standardised information to provide to whanau about the diagnostic process.
- CP information package to provide to families that receive a diagnosis of CP or high-risk of CP (available in electronic and paper form)

PEPI ARC assessment schedule
General Movements Assessment (GMA): 3 months CGA (12-14 weeks CGA) at PEPI ARC if not completed prior, or a repeat as required. Repeated 2 weeks later if abnormal/absent movements, video taken by parents/caregivers, or VNDT
Hammersmith Infant Neurological Examination (HINE): 3 months CGA (12-14 weeks) at PEPI ARC. Repeated at 4 months CGA (16-18 weeks) if assessments are inconclusive at the initial appointment.
Magnetic Resonance Imaging (MRI): Prior Neuroimaging will be reviewed at 12-16 weeks CGA at PEPI ARC.
If the infants have an abnormal/absent GMA, and/or low HINE score and assessed as high-risk of CP, referral for MRI will be considered if indicated
Infant and Child Feeding Questionnaire (ICFQ): Completed by whanau at PEPI ARC clinic
Whanau questionnaires: Within 2 weeks of clinic or at 5-6 months CGA
Health Professionals Questionnaire: Within 2 weeks of clinic or after 6 months of Hub being operational
PEPI ARC staff Questionnaire:3 months from commencement of the PEPI ARC hub, and again at 12 months of the hub running.
Whanau interview: From 6 months of the PEPI ARC running with staged review to ensure cross section of participants recruited for interview. Offered using telehealth or in person
Health Professional interview: From 12 months of PEPI ARC hub running, randomly selected participants will be interviewed until saturation of experiences reached

All resources have been developed specifically for PEPI ARC Hub to ensure local, regional and culturally appropriateness
Intervention code [1] 325816 0
Early detection / Screening
Comparator / control treatment
No control group
Control group
Uncontrolled

Outcomes
Primary outcome [1] 334374 0
1. Implementation: To determine to what extent the Best Practice recommendations for early diagnosis of CP can be equitably provided for high-risk infants admitted to the Wellington level 3 NICU in New Zealand.
Assessed
The proportion of infants assessed for CP as per the best practice recommendation:
• by region of residence
• by ethnicity, including Maori and Pasifika.
Measured
• NICU discharge data.
• Infant medical records.
• Demographic information for infants eligible for the BPR pathway.
Timepoint [1] 334374 0
24 months of participants enrolment
Primary outcome [2] 334651 0
2. Implementation: If the fidelity of the BPR can be maintained, including diagnostic assessments completed, communication with parents/ whanau and referrals to therapy interventions and surveillance for common comorbidities and complications.
Assessed:
• Diagnostic tests completed.
• Diagnosis communicated with parents/ whanau (timing and acceptability of communication)
• Timing of therapist review post diagnosis of high risk/risk CP.
• Referral for surveillance of comorbidities and medical complications as indicated.
• Information provided about practical and emotional support for whanau.
Measured
• Infant medical records
• PEPI ARC clinic notes
• Records for assessments
• Referral data
• Questionnaire and Semi-structured interviews
Timepoint [2] 334651 0
24 months of participants enrolment
Secondary outcome [1] 420423 0
3. Acceptability: To evaluate the acceptability of a PEPI ARC hub model of care by whanau
Assessed
• Using theoretical base of the Theoretical Framework of Acceptability for the constructs of acceptability: affective attitude, burden, ethicality, intervention coherence, opportunity costs, perceived effectiveness and self-efficacy
Measured
Questionnaire and semi structured interviews of whanau have all been designed specifically for PEPI ARC Hub and have not been validated
Timepoint [1] 420423 0
24 months of participants enrolment
Secondary outcome [2] 420424 0
4. Acceptability: To evaluate the acceptability of a PEPI ARC hub model of care by health professionals
Assessed
• Using theoretical base of the Theoretical Framework of Acceptability for the constructs of acceptability: affective attitude, burden, ethicality, intervention coherence, opportunity costs, perceived effectiveness and self-efficacy
Measured
Questionnaire and semi structured interviews of heath professionals have all been designed specifically for PEPI ARC Hub and have not been validated
Timepoint [2] 420424 0
24 months of participants enrolment
Secondary outcome [3] 420425 0
5. Demand: To determine the demand for the PEPI ARC hub among whanau
Assessed
• The number of infants approached to consent for the study enrolled in PEPI ARC.
• Proportion of whanau who consent for their infants to take part in PEPI ARC.
• Proportion of infants that attend clinic.
• Proportion of infants that complete regional follow-up appointments with paediatrician and therapist e.g., appointments to complete the GMA, HINE assessments and receive feedback on the outcomes.
• Proportion of whanau that opt to use telehealth.
• Type of support requested by whanau
Measured
PEPI ARC clinic statistics.
• PEPI ARC clinic notes.
• Infant medical records (virtual hub).
• Communications with PEPI ARC team from whanau
Timepoint [3] 420425 0
24 months of participants enrolment
Secondary outcome [4] 421533 0
6. Demand: To determine the demand for the PEPI ARC hub among health professionals
Assessed
• Number and profiles of health professionals accessing the hub for support.
• Type of support requested by health professionals.
Measured
PEPI ARC clinic statistics.
• PEPI ARC clinic notes.
• Infant medical records (virtual hub).
• Communications with PEPI ARC team from health professionals


Timepoint [4] 421533 0
24 months of participants enrolment
Secondary outcome [5] 421534 0
7. Limited-Efficacy:
i) To evaluate if the PEPI ARC hub model reduces the age of diagnosis of CP.
Assessed
• Age when a preliminary high-risk of CP or CP diagnosis was made.
• Diagnostic outcomes (i. CP, ii. High-risk of CP, iii. No CP, other diagnosis, iv. No CP, no clinical concerns).
• Age of CP confirmed.
Measured
• PEPI ARC clinic notes.
• Infant medical records.
Timepoint [5] 421534 0
24 months of participants enrolment
Secondary outcome [6] 421540 0
8. Limited Efficacy: To explore the experiences around communication and information sharing between whanau and health professionals.
Assessed
Semi structured interviews for both whanau and health professionals have been designed specifically for PEPI ARC Hub and have not been validated
Timepoint [6] 421540 0
24 months of participants enrolment

Eligibility
Key inclusion criteria
All infants admitted to Wellington NICU who meet the inclusion criteria and their parent/caregivers will be invited to participate in PEPI ARC. There are no limits placed on sample size and a formal sample size calculation is not required. The alternative option is to continue with standard care.

Eligibility
i. Admitted to the NICU at Wellington Regional Hospital.
ii. Survived until discharge from NICU.
iii. Discharge address located within the Wellington region or within one of Wellington NICU’s referral regions.
iv. Meet the Best Practice Recommendations criteria for risk of CP.

Inclusion criteria
1. Any Infant born less than 28+0 weeks gestation
Or
2. Infant born at or after 28+0 weeks’ gestation with one or more detectable risk factors for CP:

i. Weight less than 1000 g.
ii. Intrauterine growth restriction.
Birth weight less than 3rd percentile on population-based or customized growth charts or at clinician’s discretion based on concern about pathological growth restriction.
iii. Abnormal findings on neuroimaging associated with CP.
(e.g., grade 3 and 4 intraventricular haemorrhage, post haemorrhagic ventricular dilatation, hydrocephalus, periventricular leukomalacia (PVL), stroke, brain maldevelopment).
iv. Grade 2 or 3 hypoxic ischaemic encephalopathy (HIE).
v. Neonatal encephalopathy of other aetiology.
vi. Neonatal meningitis/ encephalitis - bacterial or viral.
vii. Cardiac surgery.
viii. Clinical or parental concerns or other significant risk factor – clinician discretion

Surveys will be distributed to health professionals involved in the above infants care or seek information through contacting the Hub. Health professionals are not study participants.
Minimum age
2 Weeks
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Exclusion Criteria
i. Infant born with a life limiting condition and not expected to survive past the first year of life.
ii. Whanau chose standard care.
iii. Infant and whanau discharge address is outside Wellington NICU’s catchment area or they are expected to move out of the catchment area before 3 months corrected gestational age.

Study design
Purpose of the study
Diagnosis
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
Other design features
All infants recruited will have the standardised assessments and interventions, but these will be delivered by either one of the two study components: face to face or virtual, As this is a feasibility study, the aim is for all infants to receive equitable care, whether living locally or regionally.
Phase
Not Applicable
Type of endpoint/s
Efficacy
Statistical methods / analysis
Data will be collected from March 2023 to November 2024 and analysed using descriptive statistics to assess participant characteristics, recruitment rates or infants and their whanau, attendance rate in clinics as well as completion of diagnostic tools, referral for early intervention and surveillance. Analysis of likert scale data from whanau, health professional and PEPI ARC team questionnaires will also be analysed using descriptive statistics. Continuous data results will be reported using the median and interquartile range (lower and upper quartiles). Pearson’s Chi-squared or Fisher’s exact test will be used to compare the extent of implementation of the best practice guidelines between regions and ethnicities. A p value of <0.05 will be considered to be statistically significant. All analysis will be conducted in Excel® 2019 or RStudio®.
Questionnaire, interview, and email data will be processed and analysed using Qualitative content analysis. One PEPI ARC researcher will edit the qualitative data by transcribing the interviews and member checking the transcripts, checking and summarising the email audit trail and questionnaire data. Two researchers will immerse themselves in the data, develop codes, categories and themes meaningful to PEPI ARC. The four analysis phases: deconcextualisation, reconcextualistion, categorisation and compilation will be repeated throughout the analysis process to ensure methodological quality and trustworthiness. Trustworthiness will be developed by employing recommended methods of developing a codebook, maintaining an audit trial and reflective journal, member checking and peer discussions. The analysis process will occur separately for whanau and health professional data but will occur concurrently to help determine any cross over of themes. Themes will be interpreted, discussed and help inform the quantitative data. Data analysis will be completed using NVIVO software (QSR International 2021).

Recruitment
Recruitment status
Recruiting
Date of first participant enrolment
Anticipated
Actual
3/03/2023
Date of last participant enrolment
Anticipated
29/02/2024
Actual
Date of last data collection
Anticipated
1/07/2025
Actual
Sample size
Target
100
Accrual to date
45
Final
Recruitment outside Australia
Country [1] 25375 0
New Zealand
State/province [1] 25375 0
Wellington

Funding & Sponsors
Funding source category [1] 313576 0
Charities/Societies/Foundations
Name [1] 313576 0
Cerebral Palsy Alliance
Country [1] 313576 0
Australia
Funding source category [2] 313578 0
Charities/Societies/Foundations
Name [2] 313578 0
New Zealand Federation of Women's Institute Inc
Country [2] 313578 0
New Zealand
Funding source category [3] 313638 0
Government body
Name [3] 313638 0
Research Activation Grant: Health Research Council of New Zealand
Country [3] 313638 0
New Zealand
Primary sponsor type
Hospital
Name
Te Whatu Ora - Capital Coast District Health Board
Address
Wellington Regional Hospital
49 Riddiford Street
Newtown
Wellington 6021
Country
New Zealand
Secondary sponsor category [1] 315362 0
None
Name [1] 315362 0
none
Address [1] 315362 0
None
Country [1] 315362 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 312757 0
Central Health and Disability Ethics Committees
Ethics committee address [1] 312757 0
Ministry of Health
Health and Disability Ethics Committees
PO BOX 5013
Wellington 6140
Ethics committee country [1] 312757 0
New Zealand
Date submitted for ethics approval [1] 312757 0
19/12/2022
Approval date [1] 312757 0
31/01/2023
Ethics approval number [1] 312757 0
2022 FULL 13434

Summary
Brief summary
The key to improving life-long outcomes and reducing impairments for children with CP, is early diagnosis. Based on available best evidence, and input including from parents, paediatricians and therapists, the New Zealand Cerebral Palsy Clinical Network has developed a national Best Practice recommendations for Early Detection of CP, Intervention and Monitoring (BPR). However, there is a large gap between the BPR and clinical practice in New Zealand. Workshops for whanau revealed a ‘one-stop shop’ could be a solution allowing for more equitable, timely, standardised and culturally safe approaches. PEPI ARC has four key study purposes:
Primary
1. Implementation: To determine to what extent the BPR for early diagnosis of CP can be equitably delivered for high-risk infants admitted to the Wellington level 3 NICU in New Zealand.
Secondary
2. Acceptability: To evaluate the acceptability of a PEPI ARC hub model of care by whanau and health professionals.
3. Demand: To determine the demand for the PEPI ARC hub among whanau and health professionals.
4. Limited-Efficacy:
i) To evaluate if the PEPI ARC hub model reduces the age of diagnosis of CP.
ii) To explore the experiences around communication and information sharing between whanau and health professionals.

Hypothesis
H1: An equal number of infants will be assessed using the BPR independent of region of residence or ethnicity.
H2: Whanau and health professionals will find the PEPI ARC Hub model of care acceptable (over the acceptability constructs).
H3: The demand will be high with infants who meet criteria choosing to enrol in the PEPI ARC study over standard care.
H4: > 34% of infants with CP will be diagnosed before 12 months CGA, and > 17% before the age of 6 months CGA.
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 125798 0
Dr Angelica Allermo-Fletcher
Address 125798 0
NICU
Wellington Hospital
49 Riddiford Street
Newtown
Wellington 6021
Country 125798 0
New Zealand
Phone 125798 0
+64 272325336
Fax 125798 0
Email 125798 0
Angelica.AllermoFletcher@ccdhb.org.nz
Contact person for public queries
Name 125799 0
Dr Angelica Allermo-Fletcher
Address 125799 0
NICU
Wellington Hospital
49 Riddiford Street
Newtown
Wellington 6021
Country 125799 0
New Zealand
Phone 125799 0
+64 272325336
Fax 125799 0
Email 125799 0
Angelica.AllermoFletcher@ccdhb.org.nz
Contact person for scientific queries
Name 125800 0
Dr Angelica Allermo-Fletcher
Address 125800 0
NICU
Wellington Hospital
49 Riddiford Street
Newtown
Wellington 6021
Country 125800 0
New Zealand
Phone 125800 0
+64 272325336
Fax 125800 0
Email 125800 0
Angelica.AllermoFletcher@ccdhb.org.nz

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

Doc. No.TypeCitationLinkEmailOther DetailsAttachment
18854Study protocol  pepiarc@ccdhb.org.nz
18855Informed consent form  pepiarc@ccdhb.org.nz
18856Ethical approval  pepiarc@ccdhb.org.nz



Results publications and other study-related documents

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