Clinical Trial Details
— Status: Recruiting
Administrative data
| NCT number |
NCT04096430 |
| Other study ID # |
Pro00090963 |
| Secondary ID |
|
| Status |
Recruiting |
| Phase |
N/A
|
| First received |
|
| Last updated |
|
| Start date |
March 1, 2022 |
| Est. completion date |
May 31, 2025 |
Study information
| Verified date |
June 2024 |
| Source |
University of Alberta |
| Contact |
Lesley Pritchard-Wiart, PhD |
| Phone |
780-492-2971 |
| Email |
lwiart[@]ualberta.ca |
| Is FDA regulated |
No |
| Health authority |
|
| Study type |
Interventional
|
Clinical Trial Summary
Children with disabilities often access rehabilitation services to improve their abilities to
participate in everyday activities. Goal-directed therapy is considered an important
therapeutic strategy to achieve outcomes that are meaningful to families. Not a lot is known
about the effects of goal setting on rehabilitation outcomes. Strategies to help children
participate in the goal-setting process are rarely used in clinical practice. The aim of this
project is to test the effects of a child-focussed goal setting approach, Enhancing Child
Engagement in Goal Setting (ENGAGE), on therapy outcomes. Service use and the cost vs.
benefits of the ENGAGE approach compared to usual practice will also be examined. Children
with neurodevelopmental disabilities aged 5-12 years old (n=96) who access paediatric
rehabilitation services at six rehabilitation sites will participate. Therapists (n=24) at
participating sites in Alberta, Canada will be randomized into 1) the ENGAGE intervention
group or 2) the usual therapy practice control group. Children will participate in the ENGAGE
approach to goal setting or usual practice based on the allocation of their therapist. This
study will determine if the ENGAGE approach to goal setting affects child goal performance,
satisfaction with goal performance, functional abilities, participation, and parent and child
quality of life. The investigators will also evaluate differences in parent and child quality
of life in relation to parent costs (e.g., absenteeism, presenteeism, travel costs) and
compare amount of therapy time between the two groups to see which approach is more
cost-effective and efficient. After the study, children, parents and therapists will be asked
to discuss aspects that influenced effective implementation of the ENGAGE approach. This
study could provide evidence to improve meaningful child and family outcomes in paediatric
rehabilitation and improve efficiency of paediatric rehabilitation services.
Description:
PURPOSE:
This pragmatic trial aims to improve health outcomes for children with neurodevelopmental
disabilities (NDD) using a novel therapist-targeted, theory-driven, evidence-based approach
to goal setting, the Enhancing Child Engagement in Goal Setting (ENGAGE) approach. This study
includes an economic analysis and service utilization component and an implementation process
analysis to evaluate cost-effectiveness and to support long-term sustainability.
RESEARCH QUESTIONS:
Primary question: Does ENGAGE improve children's engagement in therapy, goal-related
performance (primary outcome), functional abilities, participation in home, school and/or
community, and child and caregiver quality of life compared to the current standard of care?
Secondary questions: 1) What goal, child, family and therapist factors mediate and/or
moderate the effects of ENGAGE on the above intervention outcomes? and 2) Does ENGAGE improve
cost effectiveness from the perspectives of parents and service delivery efficiency compared
to the current standard of care?
METHODOLOGY/IMPLEMENTATION STRATEGY AND EVALUATION:
Study design: A pragmatic, cluster randomized controlled trial (RCT) design will be used with
therapists randomized to one of two groups; the ENGAGE intervention group or the usual care
control group. The RCT will be a six-site trial with two groups (ENGAGE training
absent/present) with two periods of post-intervention assessment (immediate post-treatment
and 3-month follow-up). The trial will take place within established public paediatric
rehabilitation sites in Alberta. An economic analysis consisting of a cost-benefit analysis
from the perspectives of parents and a comparison of service utilization costs will be
conducted in conjunction with the RCT. To facilitate more widespread implementation, a
qualitative process evaluation will be conducted to delineate core versus peripheral
components of the intervention, and therapist, child and parent perspectives on the
contextual features that influenced implementation.
Sample: The sample size will be 96 children (12 therapists as clusters per group and 4
children per therapist) at six sites. Based on the investigators' pilot work, it is
anticipated that child dropout from the pre-post intervention period will be minimal (i.e.,
less than 5%). A target change score of 2.0, a clinically significant change on the COPM
(primary outcome), with a standard deviation of 2.75 corresponds to an effect size of 0.723
for the comparison of means. A sample size of 96 will result in an effect size of at least
0.682 in the primary outcome (COPM performance rating) with alpha=0.05 and 80% power assuming
an intra-cluster correlation (ICC) of 0.1 using a two-sided, cluster adjusted, t-test for the
comparison of means. An ICC of 0.1 was selected based on results of a previous cluster RCT
with children with cerebral palsy (ICCs between 0.08 to 0.13). Since therapist attrition is
possible over the duration of the study, the sampling strategy was designed so that a cluster
size of 11 would still provide 80% power to detect an effect size of 0.716, below the target
effect size. Smaller effect sizes will be detectable if the ICC is smaller than 0.1. The
sample size was adjusted from 88 to 96 to allow for an 8% loss to follow-up (1 therapist, 4
children per group). The investigators do not anticipate retention issues for the baseline
and post-intervention assessments (primary evaluation period). The probability of loss to
follow-up may increase at the 3-month follow-up assessment. The estimate of loss to follow-up
is based on the investigators' experience with trials with children with disabilities.
Inclusion criteria are children who 1) are 5-12 years old, 2) are referred to PT and/or OT
for a period of direct treatment, and 3) speak English. Further inclusion/Exclusion criteria
can be found in the protocol in documents section.
Recruitment: Over 18 months, children (n=96) will be sequentially recruited by 24 therapists
from six paediatric Alberta rehabilitation sites (4 children per therapist).
Randomization: A computer-generated, permuted-block randomization sequence using site as a
stratification variable will be used to allocate 24 OTs and PTs across 6 sites to the ENGAGE
or control group to ensure balanced groups.
Study groups: Intervention Group - Therapists will receive training on our principles-based
goal setting approach and strategies in the goal setting toolbox. Control group - The control
group will comprise usual care. With the exception of the strategies outlined in ENGAGE,
rehabilitation interventions used to achieve identified goals will not vary from usual
practice. Consistent with a pragmatic trial, this approach will enable us to evaluate
effectiveness of ENGAGE in typical clinical settings. .
Treatment duration and intensity for both groups will differ based on nature of goals,
treatment strategies, and family preference. It is anticipated that treatment block lengths
will vary from 3-8 sessions over 2-8 weeks, representing typical clinical variation.
Fidelity monitoring- Following the training, the strategies used by ENGAGE therapists will be
tracked to evaluate treatment fidelity prior to recruiting participants. Feedback will be
provided as needed to therapists as part of the implementation plan to facilitate ongoing and
consistent use of the intervention strategies. Treatment frequency, intensity, intervention
strategies, and feedback frequency and mechanisms will be documented by therapists at each
treatment session. Co-interventions will be monitored for each participant. Recruitment and
formalized data collection will begin once intervention therapists at the site achieve an
acceptable level of fidelity defined as adherence to ENGAGE principles at least 90% of the
time. For example, the extent children are involved in identifying their own goals will be
monitored and how often therapists use feedback on goal-related performance at each treatment
session. Practices will also be monitored in the control group using an open-ended form to
prevent contamination from exposure to ENGAGE principles. Ongoing documentation of practices
and monitoring will be used to evaluate the need for additional or different implementation
support in the intervention group and to enable comparison of group practices.
During ENGAGE training and throughout the study the investigators will reinforce that
intervention therapists need to avoid discussion of principles and practices with the control
group therapists. Therapists will complete a questionnaire about their typical practices with
respect to goal setting at the beginning of the training session (intervention) and
orientation (control). These responses will identify if the control group therapists deviate
from their approach to goal setting used at baseline over the course of the study and allow
for between group comparisons of strategy use.
Data collection and Outcomes: Identical assessments will be conducted at 1) baseline
(pre-treatment), 2) post-treatment (within 10 days), and 3) at 3-months post-treatment.
Primary outcome measure is goal performance, satisfaction with performance, goal attainment
(Canadian Occupational Performance Measure (COPM)). All data will be entered into REDCap.
An economic analysis consisting of a cost benefit analysis from the family perspective will
be conducted in conjunction with this trial. Costs related to ENGAGE relative to typical care
will be tracked using a therapy session questionnaire for both groups. In addition, total
costs incurred per child during the intervention will be determined by multiplying therapist
direct and indirect (e.g., documentation) time by a corresponding unit price. Mean cost per
child will then be calculated for each group. Costs from the family perspective will be
tracked using a parent questionnaire and outcomes will be parent and child quality of life
measures.
Analysis: Data and demographic characteristics will be described (e.g., means, standard
deviations) for both groups. Change scores (post minus pre, follow-up minus post) will be
summarized for each outcome, with COPM changes post intervention as the primary analysis
(Question 1) and the other outcomes as secondary analyses. For each change score and outcome,
a cluster adjusted t-test will be used to compare mean change score between groups (ENGAGE,
control). A confidence interval will be reported for the difference between group mean
scores. Mixed effects linear regression models on all outcomes will include group and time
(fixed effects), a therapist random effect (to adjust for the clustering), and a child random
effect (to adjust for repeated measures on each child). Time will be a categorical variable
so that post and follow-up times can be compared with pre-treatment assessments. A time by
group interaction will also be considered to assess the effects of group.
Question 2: Mixed effects multiple linear regression models similar to the above will also be
developed for each outcome with the additional variables of site, site by group as an
interaction (to assess site effect) and other theoretically important variables (e.g.,
cognitive abilities, age, parenting style) as covariates.
Question 3: Mean cost per child and the mean effectiveness result per child for each group
will be represented in an incremental cost-effectiveness ratio (ICER) - the ratio of the
difference between groups in mean cost per patient to the difference in mean effectiveness
using the two quality of life measures (CarerQol and KIDSCREEN) as outcomes. Extensive
sensitivity analysis including probabilistic sensitivity analysis will be undertaken to test
robustness of the results.
Process evaluation: Semi-structured interviews will be conducted during post-intervention
assessment with pairs of parent and child participants (n=24 pairs from the ENGAGE condition;
n=12 pairs from the control condition), stratified by centre, child age and child diagnosis
to ensure variability in the sample. Therapists (n=24) at each of the six sites will also
participate in qualitative interviews to explore their experiences with the ENGAGE approach
and the implementation process.The CFIR (Consolidated Framework for Implementation Research)
framework will be used in conjunction with the Theoretical Domains Framework to sensitize the
researchers to known barriers and facilitators to implementation. Interviews will be
audio-recorded, professionally transcribed, and last approximately 45-60 minutes. Data will
be analyzed using inductive thematic analysis. Journaling will be used throughout data
collection and analysis to record preliminary themes/ideas about the data, experiences in the
research process and reasoning around decisions in data collection and analysis, and to
facilitate research reflexivity.
