Clinical Trial Details
— Status: Recruiting
Administrative data
| NCT number |
NCT05017363 |
| Other study ID # |
RES0044126 |
| Secondary ID |
|
| Status |
Recruiting |
| Phase |
N/A
|
| First received |
|
| Last updated |
|
| Start date |
September 1, 2021 |
| Est. completion date |
June 2024 |
Study information
| Verified date |
October 2023 |
| Source |
University of Alberta |
| Contact |
Lesley Pritchard, 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. The investigators are also
interested in service use and the cost vs. benefits of the ENGAGE approach compared to usual
practice. 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 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. The investigators
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. The investigators will also ask children, parents, therapists,
and managers 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 the 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: This study will use a pragmatic, cluster randomized controlled trial (RCT)
design 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 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 enable us to detect 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. The investigators have selected an ICC of 0.1 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 investigators have
designed the sampling strategy so that a cluster size of 11 would still provide 80% power to
detect an effect size of 0.716, below our target effect size. Smaller effect sizes will be
detectable if the ICC is smaller than 0.1. The sample size has been 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. Our estimate of loss to follow-up is based on our experience with
trials with children with disabilities.
Recruitment: Over 18 months, children (n=96) will be sequentially recruited by 24 therapists
from six paediatric Alberta rehabilitation sites (4 children per therapist). The
investigators have confirmed partner sites, who believe these numbers are feasible based on
the number of therapists and typical caseloads in their programs and our previous experience
collecting pilot data. Study therapists will identify potential participants who they are
about to see for regular therapy. Therapists will provide families with a tablet with a video
of the researchers introducing the study and inviting them to participate (see video script
in documentation section), or they will email the video link to families who are receiving
services virtually due to COVID 19 restrictions. Parents can either consent to participate
through an online form (REDCap) or request additional information by signing the contact
information form. Contact information will be provided to the researchers if parents sign
either of these two forms. If parents request additional information, they will be contacted
and if they are interested in participating after a discussion about the study, consent will
obtained (audio-recorded). Child assent will be obtained by the treatment therapist at the
next therapy visit or will be obtained over the phone as well. The investigators will use the
most feasible option as suggested by parents. Some children will not be able to verbalize
assent over the telephone. If parents do not wish to participate, treatment with their
therapists, will continue as usual.
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. Blocked randomization by site will facilitate
consistency of therapy interventions and child characteristics between groups with the
exception of the ENGAGE in the intervention arm. If therapists within more than one program
at a site participate, therapists will also be randomized by program. The randomization
sequence will be uploaded to REDCap to allow for centralized, online randomization.
Randomization of therapists (and not children) will decrease contamination in the usual
practice group (i.e., so therapists are not asked to go between intervention and usual care
practices) and blocking by site will facilitate equal group distribution related to therapy
interventions (e.g., types of therapy) and child characteristics (e.g., age, diagnosis). Dr.
Rosychuk (co-applicant) will generate the randomization sequence and an independent research
assistant not associated with the trial will conduct blinded randomization.
Study groups: Intervention Group - Therapists will receive training on our principles-based
goal setting approach and strategies in the goal setting toolbox. Training will include an
overview of tools and strategies including the Perceived Efficacy and Goal Setting Tool
(PEGS), which is an established goal setting tool for children aged 5-9. In addition,
training will be provided on administration of the Canadian Occupational Performance Measure
(COPM), the most widely used goal setting tool in paediatric rehabilitation that has been
used with children as young as seven years. The investigators will introduce simple
strategies to assist children in identifying goals and to ensure ongoing focus on goals using
principles of motivational interviewing, strategies to assess and nurture perceived
competence (self-efficacy), and child-friendly feedback strategies on goal-related
performance. Therapists will consist of pairs within sites providing similar interventions to
similar children so that treatment and child characteristics other than the goal setting
intervention will be similar within each site.
Control group - The control group will comprise usual care. Research on goal setting in
paediatric rehabilitation in Alberta suggests that usual practice is highly variable, often
reflecting therapist preference and a lack of formalized goal setting processes. Research
conducted elsewhere also suggests that rehabilitation goals are inconsistently established
and documented and not based on theoretically informed principles. Recent findings from our
pilot work revealed that adherence to our four principles was a significant shift from
traditional practice that did not emphasize child engagement, attention to self-efficacy, or
strategies based on principles of behaviour change.
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 investigators will track the strategies used
by ENGAGE therapists 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. This feedback will be
provided through email, phone or Zoom. 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 investigators will monitor the extent children are involved in
identifying their own goals 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.
Data collection: Identical assessments will be conducted at 1) baseline (pre-treatment), 2)
post-treatment (within 10 days), and 3) at 3-months post-treatment. Three months is a common
time period for follow-up for rehabilitation intervention studies reflecting retention within
a time frame reasonable to attribute to the intervention. Trained assessors, blinded to group
allocation, will complete the measures with families using Zoom to account for risks
associated with COVID-19. The investigators will email a copy of all tools to each family,
and screen share the same tools using Zoom to go through with the family and document
answers. Family members will be blinded to all initial responses on the measures. It is
hypothesized significant between group differences in favour of the intervention group on the
primary and secondary outcomes. The investigators hypothesize that these differences will be
maintained at follow-up across measures. All outcome measures will be used at baseline,
post-intervention and at 3-month follow-up, administered by trained assessors blinded to
group allocation. All outcome measures are established tools, and have strong psychometric
properties with children with disabilities and/or their parents.
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
(CarerQol-7D and KIDSCREEN-10).
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-P 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. Variables will be dropped from the
model one at a time if the p-value is <0.05 and not needed for model fit. This modelling will
allow us to assess the effects of the interventions in the presence of important variables
that may not be balanced across groups by randomization. This approach will be used since
there may be some systematic differences in therapist caseload. All main analyses will be
based on 'as per protocol' analysis, as recommended for pragmatic trials. Analyses will be
performed by an analyst blinded to group assignment using R, directed by Dr. Rosychuk.
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: Data collection will occur pre-implementation, during and
post-implementation, as outlined below.
Pre-implementation: Quantitative data: Therapists will complete the readiness for change
scale of the Organizational change questionnaire - Climate of Change, Processes, and
Readiness measure at the training session for ENGAGE. Therapists and managers will complete a
short demographic questionnaire.
Qualitative data: As part of their training, therapists in the ENGAGE group will discuss
anticipated barriers to this approach as well as potential strategies to optimize
implementation. This discussion will be very informal and facilitated by broad discussion
questions. Front-line managers will also participate in 45-minute interviews (either over the
phone or Zoom depending on what is most convenient). These interviews will be designed to
understand the managers' anticipated barriers to the ENGAGE approach. All interviews will be
audio-recorded and professionally transcribed.
During implementation: Bi-weekly meetings will be conducted over Zoom with the therapists at
each site. These interviews will be conducted by phone or Zoom, based on participant
preference. The purpose of these discussions will be to regularly assess and document
barriers and facilitators to implementation. The discussion will be very informal and
informed by the Consolidated Framework for Implementation Research (CFIR) and the Theoretical
Domains Framework (TDF). These discussion will be audio-recorded and transcribed.
Post-implementation: Qualitative data: Semi-structured interviews will be conducted
post-implementation with both the parent and child-participants (ENGAGE condition),
therapists (ENGAGE condition) and managers. Parent and child-participants will be interviewed
before or following their post-implementation assessment. Therapists allocated to the ENGAGE
group will be interviewed individually either over the phone/video following the completion
of therapy (within 14 days). Managers will be interviewed shortly after all of their
therapists have completed the study. CFIR framework was used in conjunction with the TDF to
develop the interview guides. Interviews (45-60 minutes) will be audio-recorded and
professionally transcribed.
Data will be analyzed using inductive thematic analysis following Braun & Clarke's 6-phase
framework. Demographic data and the questionnaire data (readiness for change) from the
therapist will be will be summarized using descriptive statistics. Journaling will be used to
record preliminary themes/ideas about the data, experiences in the research process and
rationale for data collection and analysis decisions, and to facilitate researcher
reflexivity.
