CHILD-BRIGHT READYorNot[TM] Brain-Based Disabilities Trial
Phase: NA
Conditions: Autism Spectrum Disorder, Cerebral Palsy, Epilepsy, Fetal Alcohol Spectrum Disorders, Spina Bifida
Sponsor: McMaster University

NOTE: CTO#1666 Amendment approved by HiREB on January 9, 2023. Target sample was 264 
participants; however, we randomized 52 participants, and did not complete the full-scale RCT. Linking 
health card numbers with provincial administrative data is labour intensive and costly, and the small 
number would not provide meaningful results. As approved by ethics, we dropped the health economic 
outcome analysis (Health card number linking, Health Utilities Index (HUI), Resource Use Questionnaire 
(RUQ)) and destroy stored health card number data. 
Page 1 of 74                                         Protocol - READYorNotTM Brain-Based Disabilities Trial (version date: April 13, 2022) 
 
 
STUDY PROTOCOL 
 
Title of Study: 
 
CHILD-BRIGHT READYorNotTM Brain-Based Disabilities Trial  
 
Nominated Principal Investigator:  
Dr. Jan Willem Gorter – McMaster University, McMaster Children’s Hospital, Hamilton 
 
Co-Principal Investigator:  
 
Dr. Ariane Marelli – McGill University & McGill University Health Centre, Montreal 
 
CORE Investigators: 
Dr. Khush Amaria – CBT Associates of Toronto: Cognitive Behavioural Therapy Services  
Dr. Adrienne Kovacs – Oregon Health & Science University, Portland (Co-PI CHILD-BRIGHT) 
Dr. Ronen Rozenblum – Brigham and Women’s Hospital and Harvard Medical School, Boston 
 
Co-Investigators (alphabetic order):  
Dr. John Andersen – University of Alberta, Glenrose Rehabilitation Hospital, Edmonton 
Dr. Rima Azar - Mount Allison University, New Brunswick  
Dr. Shelley Doucet – University of New Brunswick, Saint John 
Dr. Anne Fournier – CHU Mère-Enfant, Sainte Justine Hospital, Montreal 
Dr. Andrew Mackie – University of Alberta, Stollery Children's Hospital, Edmonton 
Dr. Maryam Oskoui – McGill University & Montreal Children's Hospital, McGill University Health Centre, 
Montreal 
Dr. Janet Rennick – McGill University & Montreal Children’s Hospital, McGill University Health Centre, 
Montreal 
Dr. Michael Shevell – McGill University & Montreal Children's Hospital, McGill University Health Centre, 
Montreal 
Dr. Lehana Thabane – McMaster University, Hamilton  
Dr. Lonnie Zwaigenbaum – University of Alberta, Glenrose Rehabilitation Hospital, Edmonton 
 
Current Active Members of Patient-Family Advisory Committee (PFAC): 
Donna Thomson, Parent/Family Advisor 
Roger Stoddard, Parent/Family Advisor 
JoAnne Mosel, Parent/Family Advisor 
Connie Putterman, Parent/Family Advisor 
Kinga Pozniak, Parent/Family Advisor 
Nathan Tasker, Young Adult/Patient Advisor 
Julia Hanes, Young Adult/Patient Advisor 
Kyle Chambers, Young Adult/Patient Advisor 
Jessica Havens, Young Adult/Patient Advisor 
Claire Dawe-McCord, Young Adult/Patient 
Advisor 
Dana Arafeh, Young Adult/Patient Advisor 
Musa Arafeh, Young Adult/Patient Advisor 
Michael Frost, Young Adult/Patient Advisor
 
Project Staff: 
MyREADY Design & Development Project Manager: Alicia Via-Dufresne Ley, McGill University 
Qualitative/Stakeholder Engagement Research Coordinator: Sonya Strohm, McMaster University 

NOTE: CTO#1666 Amendment approved by HiREB on January 9, 2023. Target sample was 264 
participants; however, we randomized 52 participants, and did not complete the full-scale RCT. Linking 
health card numbers with provincial administrative data is labour intensive and costly, and the small 
number would not provide meaningful results. As approved by ethics, we dropped the health economic 
outcome analysis (Health card number linking, Health Utilities Index (HUI), Resource Use Questionnaire 
(RUQ)) and destroy stored health card number data. 
Page 2 of 74                                         Protocol - READYorNotTM Brain-Based Disabilities Trial (version date: April 13, 2022) 
 
 
Randomized Controlled Trial (RCT) Research Coordinator: Barb Galuppi, McMaster University; Nadilein 
Mahlberg, McMaster University. 
 
Current Active Trainees: 
Linda Nguyen, PhD Candidate, Rehabilitation Science, McMaster University (Supervisor: Dr. Gorter)  
 
Sponsor: The READYorNotTM Brain-Based Disabilities Trial is funded by the CHILD-BRIGHT Network, 
under the Canadian Institutes of Health Research (CIHR) Strategy for Patient-Oriented Research (SPOR) 
initiative, and with partner support from McMaster University Faculty of Health Sciences, Hamilton 
Health Sciences, McMaster Children’s Hospital Foundation, Montreal Children's Hospital Foundation, 
and New Brunswick Health Research Foundation.  
 
PLAIN LANGUAGE DESCRIPTION:  
Youth with brain-based disabilities (BBD) see a variety of pediatric doctors and health care providers 
during their childhood years. Pediatric doctors and care teams are trained to manage the health of 
children, including physical, behavioural, and mental health issues. Typically, by their eighteenth 
birthday, youth in Canada will need to leave their pediatric doctors and health care providers and go to 
adult providers instead. Generally, there are more expectations for youth to take charge of their own 
care when they see an adult care provider. Yet, if youth are not ready for this responsibility, or it is not 
clear where youth should go for care as adults, their health can sometimes be affected (for example 
when appointments or medications are missed). We also know that this change can be especially 
difficult and stressful for youth with BBD and for their families.   
In the first part of this project, researchers, healthcare professionals, technology designers, youth and 
families have worked together, to co-create an e-health application called MyREADY TransitionTM BBD 
App for Brain-Based Disability. In this next part of the project, we will ask pediatric health care providers 
to share it with their patients who are between 15 and 17 years of age, and who have one of the 
following conditions: autism spectrum disorder, cerebral palsy, epilepsy, spina bifida, or fetal alcohol 
spectrum disorder. The MyREADY TransitionTM BBD App is designed to help youth with health care 
transition planning, in preparation for their transfer out of the child health system and into the adult 
health system. We want to see how youth will use the MyREADY TransitionTM BBD App as they are 
getting ready to go from pediatric to adult health care services. And, we want to see if it will help them 
to be more prepared and knowledgeable to manage their own health. We hope to see youth taking 
steps to be better managers of their health. For example, this would include knowing about their 
condition or knowing when to ask for help from parents/caregivers and health care providers.  
BACKGROUND AND RATIONALE:  
Large care gaps during pediatric to adult transition years have been shown in half of Canadian patients 
with increases in emergency room visits and resulting costs.1 The goal of transition is to maximize 
“lifelong functioning and potential through the provision of high-quality, developmentally-appropriate 
health care services and coordination that continue uninterrupted as the individual moves from 
adolescence to adulthood”.2 Youth with BBD, as well as their parents/caregivers, often feel ill-prepared 

NOTE: CTO#1666 Amendment approved by HiREB on January 9, 2023. Target sample was 264 
participants; however, we randomized 52 participants, and did not complete the full-scale RCT. Linking 
health card numbers with provincial administrative data is labour intensive and costly, and the small 
number would not provide meaningful results. As approved by ethics, we dropped the health economic 
outcome analysis (Health card number linking, Health Utilities Index (HUI), Resource Use Questionnaire 
(RUQ)) and destroy stored health card number data. 
Page 3 of 74                                         Protocol - READYorNotTM Brain-Based Disabilities Trial (version date: April 13, 2022) 
 
 
for the transfer from pediatric to adult health care services, typically at a system-defined age of 18 years 
in Canadian jurisdictions.  
Pediatric health care providers are encouraged to talk to children and youth about taking on more 
responsibility for their own health as they mature. Yet many providers report that they do not have the 
time, resources or helpful tools, nor do they have engaging ways to talk about making a smooth move to 
the adult system. This study proposes to overcome these barriers by applying interventions based on 
individual functional needs to promote transition readiness (self-management and self-advocacy skills) 
through an innovative e-health intervention.  The overall objective of this project is to improve 
readiness for transition of care in youth with BBD who are between 15 and 17 years of age, are 
beginning to manage their own or have the potential for self-management.  
We acknowledge the input from youth, parent/caregiver and health care stakeholders, who emphasized 
that a youth’s transition to adulthood involves many systems beyond health care. In this study, 
however, we focus primarily on health care transition. Transition from pediatric to adult health care 
systems is a critical turning point for a growing population of youth with lifelong conditions. When the 
transition to adult health care is unplanned or poorly supported, patients’ and parents’/caregivers’ 
adverse experiences of care are higher, as is risk for poor health outcomes, costly hospitalizations, 
inequity of health care services, and “other” costs such as missed school or work 3.  
Patient-centred care and patient engagement have become central components of the modern clinical 
encounter. Enhancing patient engagement via information technology is shown to directly impact 
patient behaviour that promotes positive health outcomes, patient satisfaction, care delivery efficiency, 
reduces costs and improves quality of care and patient safety.4-5 
This project proposes to provide an e-health intervention to youth patients to improve education, 
empowerment and navigation of their health and the health care system, with the goal of improving 
transition readiness. Specifically, the intervention includes important elements such as personal 
identity, knowledge and understanding of living with a health condition, personal control, decision-
making, and knowing when and how to seek support from others.6 Our study responds to the needs of 
families, and the need for systematically well-designed research approaches to co-create evidence-
based e-health transition interventions through ongoing youth, parent/caregiver and health care 
stakeholder engagement. As well, we have formed partnerships with e-health industry, so that our 
intervention can be affordable, easily implemented in health care systems, and applicable to the 
majority of all youth with BBD. We have engaged a core group of youth, parent/caregiver and health 
care stakeholders, including a Patient and Family Advisory Council (PFAC), composed of representative 
youth (adolescents and young adults) and parents/caregivers. We have collaborated with the PFAC 
during the first part of the project to inform the design and tailor the MyREADY TransitionTM BBD App. 
We plan to continue to leverage our stakeholder partnerships throughout the RCT and Knowledge 
Transition (KT) parts of the project to promote uptake and benefit.  
SYNOPSIS: 

NOTE: CTO#1666 Amendment approved by HiREB on January 9, 2023. Target sample was 264 
participants; however, we randomized 52 participants, and did not complete the full-scale RCT. Linking 
health card numbers with provincial administrative data is labour intensive and costly, and the small 
number would not provide meaningful results. As approved by ethics, we dropped the health economic 
outcome analysis (Health card number linking, Health Utilities Index (HUI), Resource Use Questionnaire 
(RUQ)) and destroy stored health card number data. 
Page 4 of 74                                         Protocol - READYorNotTM Brain-Based Disabilities Trial (version date: April 13, 2022) 
 
 
Our overall project consists of several parts. In Part 1 of the project, we have developed the MyREADY 
TransitionTM BBD App intervention designed to improve transition readiness of youth prior to transfer of 
care.  This current protocol is focused on Part 2, a mixed methods design featuring an RCT to test 
whether the MyREADY TransitionTM BBD App intervention improves readiness for transition. Our RCT 
will use a pragmatic approach designed to test whether the intervention works under the usual 
conditions. The PRECIS-2 website7 (PRECIS – PRagmatic Explanatory Continuum Indicator Summary) is a 
tool for designing clinical trials on the pragmatic/explanatory continuum. The PRECIS-2 tool has nine 
domains: eligibility criteria, recruitment, setting, organization, flexibility-delivery, flexibility-adherence, 
follow-up, primary outcome, and primary analysis. All domains are scored from 1 (very explanatory) to 5 
(very pragmatic). We will use the PRECIS-2 planning tool to facilitate discussion and consensus and to 
ultimately match design decisions with how the trial results are intended to be used.  Integrated 
knowledge translation throughout the years of the project will draw on patient, parent/caregiver and 
health care stakeholder partnerships. 
Outcome metrics are based on the Institute for Health care Improvement (IHI)’s Triple Aim framework8 
(better health, better experience, at lower cost) with analysis stratified by region to account for local 
contextual factors. This comprehensive framework is poised for the creation of effective and efficient 
systems to optimize child and family outcomes.  
CHILD-BRIGHT intentionally addresses a wide range of chronic diseases that share common functional 
limitations and health care needs. This non-categorical approach (i.e. including more than one medical 
condition or disability) enables interventions that can impact on larger populations of children above 
and beyond any diagnostic specific needs. The focus on BBD is strategically more integrative than 
diagnosis-specific research, thus promoting broader applicability.  
STUDY AIMS AND HYPOTHESIS: 
The MyREADY TransitionTM BBD App intervention includes elements of transition planning to empower 
youth with BBD as they prepare for transfer into the adult health care system.  
The primary aim of the study is to determine whether the MyREADY TransitionTM BBD App intervention 
will result in improved self-management and self-advocacy skills (major components of transition 
readiness), compared to usual care for youth with BBD between 15 and 17 years of age. The MyREADY 
TransitionTM BBD App intervention has the potential to improve perception of quality of care and youth 
and parent/caregiver experiences during the transition process; and a potential also for cost efficiencies 
across pediatric and adult provincial health care systems. Analysis for the primary aim of the study will be 
led by Dr. Gorter at McMaster University. 
Hypothesis: Youth who use the MyREADY TransitionTM BBD App intervention will have higher self-
management change scores (a major component of transition readiness), over a 6-month period 
compared with youth receiving usual care. Self-management change scores will be measured using the 
Transition Readiness Assessment Questionnaire (TRAQ).9 
Secondary aims of the study include exploring the contextual experiences of youth using the MyREADY 
TransitionTM BBD App (e.g., their satisfaction with the App and its content, how it is being used and 

NOTE: CTO#1666 Amendment approved by HiREB on January 9, 2023. Target sample was 264 
participants; however, we randomized 52 participants, and did not complete the full-scale RCT. Linking 
health card numbers with provincial administrative data is labour intensive and costly, and the small 
number would not provide meaningful results. As approved by ethics, we dropped the health economic 
outcome analysis (Health card number linking, Health Utilities Index (HUI), Resource Use Questionnaire 
(RUQ)) and destroy stored health card number data. 
Page 5 of 74                                         Protocol - READYorNotTM Brain-Based Disabilities Trial (version date: April 13, 2022) 
 
 
integrated during transition, and perceived value); as well as the interactive processes of youth, their 
parents/caregivers and health care providers around use of the intervention (e.g., that support/hinder 
use). Analysis for the secondary aim of the study will be led by Dr. Marelli at McGill University. 
In addition, both Dr. Gorter and Dr. Marelli will work with the Health Economics team within the CHILD-
BRIGHT network who will support the data collection and analysis for the health economics outcomes, 
with a few key objectives across all projects: 
• 
To compare the patterns of health service use for the intervention compared to current standard of 
care. 
• 
To compare the incremental cost of the intervention compared to current standard of care per unit 
of effectiveness (using standardized measures across the network including the Health Utilities 
Index®10, PedsQLTM 11, Resource Use Questionnaire12).  
RESEARCH PLAN AND METHODOLOGY: 
METHODOLOGY  
We will take a mixed methods approach that utilizes both quantitative and qualitative research methods 
and techniques. This methodology is commonly used in patient-centred care research as these methods 
combined can serve to answer complex research questions and allow for stronger and richer evidence 
than could be accomplished by a single method alone. Contextual qualitative data is important where 
the complexity of different sites throughout Canada might create challenges for evaluating the 
effectiveness of the intervention and will be essential for eventually understanding and explaining any 
potential variations that emerge from the RCT.        
DESIGN 
We will use an embedded experimental model design13, which will involve embedding a qualitative 
component within the randomized controlled trial (RCT). We will seek to collect qualitative data from a 
subset of participants in the intervention group, at the end of exposure or after 6 months (whichever 
comes first) and after the 6 Month quantitative data collection point. The qualitative data will be 
supplementary to help explain or interpret the quantitative results of the RCT (primary aim) as well as to 
understand user experience (secondary aim). The two data types will complement each other to provide 
a comprehensive understanding of the effectiveness of the app, both from the quantitative 
questionnaires and from the perspectives of youth, parents/caregivers and health care providers 
through qualitative interviews/open-ended questions.  The qualitative sampling, data collection and 
analysis will be guided by the principles of interpretive description – an applied qualitative methodology 
that provides a structure for answering questions that arise in clinical practice.14  
Usability Testing (completed during the App Development phase) 
Usability testing aims to ensure that interactive systems are adapted to the users and their tasks and is 
considered a fundamental component of the user-centred design process.15  Throughout the 
development of the MyREADY TransitionTM BBD App, iterative cycles of participatory design sessions 
were used to elicit patient, parent/caregiver and health care provider feedback on elements of the tools 

NOTE: CTO#1666 Amendment approved by HiREB on January 9, 2023. Target sample was 264 
participants; however, we randomized 52 participants, and did not complete the full-scale RCT. Linking 
health card numbers with provincial administrative data is labour intensive and costly, and the small 
number would not provide meaningful results. As approved by ethics, we dropped the health economic 
outcome analysis (Health card number linking, Health Utilities Index (HUI), Resource Use Questionnaire 
(RUQ)) and destroy stored health card number data. 
Page 6 of 74                                         Protocol - READYorNotTM Brain-Based Disabilities Trial (version date: April 13, 2022) 
 
 
(i.e. to learn what was working, what wasn’t, and where gaps existed). This rigorous process was 
instrumental in refining the App intervention prior to the start of the RCT, thus enhancing the likelihood 
of success of the intervention itself.  
Internal Pilot Phase  
The British Medical Research Council explicitly recommends the use of feasibility studies prior to Phase 
III clinical trials.16 In order to guide the planning and to enhance the likelihood of success of our full scale 
RCT, the first three months of recruitment will comprise an internal pilot phase. In this internal pilot 
phase, we will aim to recruit the first 12 patients in each of the four regions, up to 48 participants across 
all regions. 
During the internal pilot phase, we will observe the study procedures and consider key implementation 
aspects such as recruitment (including refusal rates and screening process), multi-site 
coordination/collaboration (including communication, documentation and provision of support) and 
intervention uptake and adherence (including technical support needs among app users). The results 
will be used to refine and enhance the research design. The RCT will proceed with procedural 
modifications based on the findings of the internal pilot study and final study analyses will incorporate 
all data. As long as the alpha-level is controlled, internal pilot designs have, at most, a small adverse 
effect on the significance level and may greatly improve the power.17  
After data collection has concluded for the main RCT, we will conduct Optional Research to enhance our 
pilot and feasibility exploration and reporting. Participants will include health care providers and 
research assistant staff who were involved in recruiting youth participants for the RCT. 
Health care providers who were involved in study recruitment will be invited to complete an electronic 
survey. The purpose is to help us learn about their experience recruiting youth during the pandemic and 
about their perspective on integrating the eHealth intervention into practice. Each health care provider 
participant will have the opportunity to be entered into a draw to win a $250 meal allowance for their 
next staff meeting. Rather than a guaranteed gift card incentive of low monetary value (e.g., $10 for 
each of 25 respondents), we have selected a lottery model which will give survey respondents a chance 
at a more appealing incentive. We feel this incentive is appropriate and reasonable in scope in order to 
encourage participation without undue inducement. 
Research Assistant (RA) staff who were involved in study recruitment will be invited to participate in a 
focus group interview by Zoom and to complete a short demographic survey. The purpose of the focus 
group is to help us learn more about the processes, resources, and management of the study. As a token 
of appreciation for their time to participate, all research assistant participants will be given a $25 gift 
card. We expect to include 10-12 participants.  
SAMPLE 
Our primary sample will be comprised of 264 youth with BBD, between 15 and 17 years of age, who are 
recruited through a participating clinic in one of the four study regions (Alberta, Ontario, Quebec, and 
Maritimes) and who are at least 6 months pre-transfer to the adult health care system. A 
parent/caregiver of the youth will also be recruited to the study. The study aims to have an equal 

NOTE: CTO#1666 Amendment approved by HiREB on January 9, 2023. Target sample was 264 
participants; however, we randomized 52 participants, and did not complete the full-scale RCT. Linking 
health card numbers with provincial administrative data is labour intensive and costly, and the small 
number would not provide meaningful results. As approved by ethics, we dropped the health economic 
outcome analysis (Health card number linking, Health Utilities Index (HUI), Resource Use Questionnaire 
(RUQ)) and destroy stored health card number data. 
Page 7 of 74                                         Protocol - READYorNotTM Brain-Based Disabilities Trial (version date: April 13, 2022) 
 
 
number of participants in each of two groups: intervention group (132) and control group (usual care) 
(132). Since participants will be recruited from only four regions, we will model the effect of region as a 
fixed effect rather than a random effect.18 This will allow region-specific intervention effects to be 
modelled (i.e. region by intervention interaction effects). If no region by intervention interactions are 
found, the interaction terms will be dropped from the model and we will estimate an overall 
intervention effect. Since the region by intervention interaction is unknown, we take a conservative 
approach with a sample size calculation that will enable detection of an intervention effect within each 
site. This will allow for a properly powered analysis of the site by treatment interaction. The primary 
outcome measure (Transition Readiness Assessment Questionnaire, TRAQ)9 has been validated on a 
sample of youth with congenital heart disease and in the absence of literature specific to BBD, we will 
base our sample size calculations on these findings. We anticipate a mean TRAQ self-management 
baseline score of 3.01 (SD 1.02) (out of a possible 5.0) as reported for youth (with congenital heart 
disease) without a transition intervention and an anticipated mean score of 4.0 at 6 months post-
intervention resulting in a change score of 1 (i.e., 1 SD), with a=0.05 and 90% power.19 Therefore, in 
each region, we will require 23 youth in each of the 2 arms (total of 46) x 4 regions = 184. Estimating 
30% attrition, we will enroll 264 participants.  
Inclusion Criteria 
• 
Youth with chronological age between 15 and 17 years of age (i.e. before 18th birthday), in one of 
the four study regions, followed in pediatric care and for whom a discharge from pediatric care is 
planned but not for at least 6 months. 
• 
A diagnosis of one of the following neurological BBD: autism spectrum disorder, cerebral palsy, 
epilepsy, spina bifida, or fetal alcohol spectrum disorder.  
• 
Cognitive ability to provide informed consent and the ability to read and understand English or 
French 
• 
Access to internet and a smartphone, iPad/tablet or desktop computer. 
• 
TRANSITION-Q20 score >40 (as a screen to define a minimum threshold for transition readiness 
based on our earlier work).   
In a validation sample of Ontario youth aged 12 -25 years old with chronic health conditions 
including our target population, the average TRANSITION-Q score for 13 year olds was 40; for 15 
year olds was 53; and for 17 year olds was 59.21 The decision to set the threshold at > 40 aligns with 
clinical judgement and is conservative given that the youngest age group in the validation sample 
demonstrated this minimum level of readiness. At the point of screening, we will evaluate several 
components of cognitive function as well as youth capacity to share the management of their own 
health. The TRANSITION-Q screen will inherently not include youth who have severe intellectual 
disability, and/or those who rely significantly on parents/caregivers in most areas of daily 
functioning, self-care and/or communication.  
Exclusion Criteria 

NOTE: CTO#1666 Amendment approved by HiREB on January 9, 2023. Target sample was 264 
participants; however, we randomized 52 participants, and did not complete the full-scale RCT. Linking 
health card numbers with provincial administrative data is labour intensive and costly, and the small 
number would not provide meaningful results. As approved by ethics, we dropped the health economic 
outcome analysis (Health card number linking, Health Utilities Index (HUI), Resource Use Questionnaire 
(RUQ)) and destroy stored health card number data. 
Page 8 of 74                                         Protocol - READYorNotTM Brain-Based Disabilities Trial (version date: April 13, 2022) 
 
 
• 
Youth is in “acute crisis” with unstable physical or mental health that would interfere with the 
ability to participate in the study. 
• 
Sensory impairments, such as uncorrected vision or hearing loss, which interfere with use of the 
App. 
• 
Enrolled in a potentially confounding trial (e.g., a different transition intervention study). 
Qualitative Subsample 
From among the sample of 132 youth participants in the intervention group, we will purposefully 
sample a subset of up to 50 youth participants to describe and understand primary outcomes, as well as 
to capture process and user experience (secondary aim). Interviews will be conducted after the 6-month 
quantitative data collection point. To further describe and understand primary outcomes after 6 
months, we will also seek to interview 10 parents/caregivers and 10 health care providers.   
METHODS 
The current study timeline is described in Appendix A. 
Recruitment 
Our recruitment approach was modified (prior to recruitment start) to add strategies for the study to 
proceed even during periods of social distancing due to the COVID-19 global pandemic.  
Step 1 – Initial Contact. Someone in the patient’s circle of care at the clinic site will approach potential 
participants who meet the minimum eligibility requirements in order to ascertain interest and obtain 
permission for the RA to contact them.  They may obtain this permission in person (after COVID-19 
restrictions are lifted), by telephone or by letter (e.g. using the Invitation Letter and Initial Interest 
form). Recruitment materials will also be shared on websites and social media so there may be some 
participants who self-refer to the RA. 
Step 2 – RA Contact. Prior to being in contact with study participants, each study RA will complete e-
learning module training provided by the research team. When a potential participant learns about the 
study online or through social media, they will self-refer by contacting the RA directly. Otherwise, after 
someone in the circle of care has made first contact and obtained permission for the RA to contact, the 
RA will contact eligible participants by phone to complete the Screening Checklist and confirm eligibility. 
If there is a clinic appointment scheduled, contact will be made in advance so that they have an 
opportunity to learn about the study and ask questions. The consent and assent form will be sent to the 
participants in advance of the scheduled visit. Due to the COVID-19 pandemic, we have added the 
option for the participant to choose to have these sent to them by email or by mail.  
Step 3 – Consent/Assent. The visit will be arranged with the participants in advance and will be done 
either by phone or in person. Due to the COVID-19 pandemic, we have added a telephone verbal 
consent procedure. While on the phone with the participant, the RA asks the participants to watch the 
Assent Video by clicking on the link sent to them.  Watching the video together by Zoom meeting link 
may be offered if the participants prefer. The RA will stay on the phone while the participants watch the 
video and then will proceed with the Verbal Consent/Assent Telephone scripts and logs. Youth 

NOTE: CTO#1666 Amendment approved by HiREB on January 9, 2023. Target sample was 264 
participants; however, we randomized 52 participants, and did not complete the full-scale RCT. Linking 
health card numbers with provincial administrative data is labour intensive and costly, and the small 
number would not provide meaningful results. As approved by ethics, we dropped the health economic 
outcome analysis (Health card number linking, Health Utilities Index (HUI), Resource Use Questionnaire 
(RUQ)) and destroy stored health card number data. 
Page 9 of 74                                         Protocol - READYorNotTM Brain-Based Disabilities Trial (version date: April 13, 2022) 
 
 
participants will provide assent and a parent/caregiver will also provide consent both for their child and 
for themselves. Ideally the participants will then complete the baseline study visit at the same 
appointment time. If the participant is unable to complete the baseline visit, the RA will arrange another 
study appointment that is convenient for the youth and parent/caregiver. 
Consent/Assent for Optional Research. Youth participants: The RA will explain to participants that the 
researchers doing this study are interested in doing additional optional research.  Participants will be 
given an additional optional study consent form to read and sign if they wish to give permission to take 
part in an optional research project.  Participants may decide not to participate in any optional research 
and still participate in this main study. Up to 50 youth in the intervention group will be contacted about 
their interest to participate in optional qualitative interviews to describe and understand primary study 
outcomes, as well as to capture process and user experience related to the secondary aim.) All 
interviews will take place following the 6 month quantitative data collection point. .  We will recruit a 
heterogeneous sample of youth to explore the experiences of a diverse range of users. We will 
purposefully sample youth of different genders, different diagnoses, from different geographic regions, 
and who demonstrate a range of transition readiness as indicated by initial TRANSITION-Q scores. Once 
the RCT has concluded, and to help with explaining the quantitative results, purposeful sampling may 
also involve seeking unique cases with respect to study outcomes (e.g., individuals who complete high 
percentage of app content but who score low on measures of transition readiness, individuals who 
complete low percentage of app content but who score high on measures of transition readiness, etc.).  
Parent/caregiver participants: We will also contact a group of 10 parents/caregivers about their interest 
to participate in qualitative interviews. We will invite parents/caregivers who RAs have recommended 
are good candidates for an interview. Prior to collecting any qualitative data, verbal and written 
informed consent will be obtained.  
Health care provider participants: We will invite 25-30 health care providers who were involved in 
recruiting study participants to complete a short electronic survey to help us collective qualitative 
information about their experience recruiting youth during the pandemic and about their perspective 
on integrating the intervention into practice. The survey will begin with an information and consent 
statement. They will also be asked if they consent to being contacted for a follow-up interview if 
necessary. 
RA participants: We will invite 10-12 Research Assistant (RA) staff who were involved in study 
recruitment to participate in a focus group interview by Zoom. The purpose is to help us learn more 
about the processes, resources, and management of the study. In order to ensure accuracy, and with 
the participants’ agreement, the focus group interview will be recorded on Zoom and the conversation 
will be transcribed. Prior to collecting any qualitative data, verbal and written informed consent will be 
obtained.  
Participants have the right to withdraw their data from the study. There are no limitations on the 
withdrawal of quantitative data during the data collection phase. However, due to the iterative nature 
of qualitative research, participants will have up to 2 weeks following the date of an interview or focus 
group interview to request withdrawal of their data. In contrast to quantitative data analysis, qualitative 

NOTE: CTO#1666 Amendment approved by HiREB on January 9, 2023. Target sample was 264 
participants; however, we randomized 52 participants, and did not complete the full-scale RCT. Linking 
health card numbers with provincial administrative data is labour intensive and costly, and the small 
number would not provide meaningful results. As approved by ethics, we dropped the health economic 
outcome analysis (Health card number linking, Health Utilities Index (HUI), Resource Use Questionnaire 
(RUQ)) and destroy stored health card number data. 
Page 10 of 74                                         Protocol - READYorNotTM Brain-Based Disabilities Trial (version date: April 13, 2022) 
 
 
analysis begins as soon as data are collected in a study. This iterative approach is imperative, as early 
and continuous coding drives ongoing data collection (e.g., new questions that arise). The two-week 
period reflects the average time it would take for a transcriptionist to complete transcription of an audio 
file. Once a participant’s interview becomes part of the analysis, it begins to shape interpretation and 
meaning of the data. If someone were to request withdrawal of their data after it has been coded, it 
would be hard to distinguish if something from their interview might have already contributed to our 
collective understanding of the data. Once the study analysis has been completed, we cannot withdraw 
data in order to protect the integrity of the research. 
Randomization 
Randomization will be stratified by region with a 1:1 allocation ratio for patients: intervention group 
(receiving MyREADY TransitionTM BBD App intervention) or control group (continuing with usual care). 
The unit of randomization is the patient. We will use variable block randomization with block sizes of 2, 
4, 6 and 8. Allocation will be done via REDCap (Research Electronic Data Capture)22 using a centralized 
allocation system generated by the Biostatistics Unit at St. Joseph’s Hospital in Hamilton, Ontario.  
Participants who meet the eligibility requirements at the point of screening, and who give consent to 
participate will be randomized to a group. Once a patient has been randomized to the intervention 
group, the Research IT team at McGill University will receive the assigned study identification number to 
associate with the user activation code in the MyREADY TransitionTM BBD App. The RA at the 
recruitment site will use the study identification number and user activation code to facilitate the 
download and user orientation to the App.  The RCT Research Coordinator at McMaster University and 
the Research IT team at McGill University will support the use of the App and troubleshoot issues as 
they arise. This will be done using a designated email to capture and respond to queries and will include 
an automated response, indicating receipt and response time.  
Participants allocated to the usual care group will not know the details of the electronic intervention 
being offered to the intervention group. Due to the nature of the intervention, participants cannot be 
blinded to group allocation, however outcome assessment and data analysis will be blinded. Our RCT 
protocol is registered with ClinicalTrials.gov (NCT03852550).  
In order to track participants according to the CONSORT guidelines23-24, a de-identified recruitment log 
of potential candidates at all participating sites will be kept, recording inclusion/exclusion criteria and 
reasons for eligible youth patients not being recruited or randomized. 
Intervention and Control Conditions 
Participants randomized into the control group will receive their clinic’s standard of care. Any support 
the youth (and parents/caregivers) receive as part of any ongoing transition programs in usual care will 
be documented by youth, parents/caregivers and health care providers. Documentation will include an 
inventory checklist with a section to add any specific information or individualized approach relevant to 
the transition process. We will also ask participants and their parents/caregivers what they perceive as 
supports and if they were received.  

NOTE: CTO#1666 Amendment approved by HiREB on January 9, 2023. Target sample was 264 
participants; however, we randomized 52 participants, and did not complete the full-scale RCT. Linking 
health card numbers with provincial administrative data is labour intensive and costly, and the small 
number would not provide meaningful results. As approved by ethics, we dropped the health economic 
outcome analysis (Health card number linking, Health Utilities Index (HUI), Resource Use Questionnaire 
(RUQ)) and destroy stored health card number data. 
Page 11 of 74                                         Protocol - READYorNotTM Brain-Based Disabilities Trial (version date: April 13, 2022) 
 
 
Participants randomized into the intervention group will be provided the link to download the MyREADY 
TransitionTM BBD App.  The RA will be trained to facilitate the App download and registration. The RA 
will demonstrate the App, ensuring that the participant understands how to access all the features. The 
MyREADY TransitionTM BBD App intervention is fully described in Appendix B. The App will be translated 
into French, and the translation will be done by professional translators of the firm Essentiel Plus. 
During the course of study participation, participants will receive regular reminder messages and/or 
monthly phone calls to promote the use of the MyREADY TransitionTM BBD App and to assist with 
troubleshooting any problems accessing the intervention. The App uses local storage within the device 
and synchronizes each participant’s progress information with the Content Management System (CMS) 
which is hosted by Amazon and managed jointly by a software company (RootQuotient) and the 
Research IT team at McGill University. The Research IT team at McGill University will send regular 
reports to the RAs and/or the RCT Coordinator to monitor when key time points are achieved (e.g. when 
a participant has completed at least 25%, 50%, 75% and 100% of the App content, or when it has been 
more than 2 weeks since a participant has accessed the App). 
In terms of exposure time, there is planned flexibility to allow participants to proceed through the 
MyREADY TransitionTM BBD App intervention at their own pace. The App has 19 individual sections 
organized within 5 chapters that are provided within a set order. A timer has been built into the App to 
ensure that participants will have at least one day between sections. This will allow for time to process 
and reflect on the take-away message in each section and/or engage in one of the suggested between-
session practice activities. The timer will help to moderate pace and align with how young people learn 
and digest information. We estimate that there are approximately 5-7 hours of content within the 5 
chapters of the App, depending on individual pace. For the RCT, the recommended exposure to the 
intervention is somewhere between 1 section per day (19 days) and 1 section per week (19 weeks). As 
an example, if a participant completed one of the five chapters per week, they would complete the App 
in 5 weeks. Games and fun activities are incorporated to encourage youth to visit the App between 
sections. The tools and resources within the App remain available for users to access throughout the 
intervention exposure time. The researchers aim to supply the MyREADY TransitionTM BBD App to 
participants in both the intervention and control group for a limited time after their participation in the 
study.  However, if the App intervention turns out not to be effective, or if significant issues are found, 
study participants may not be able to access the App. We will continually review and update our 
securities and controls as technology changes to ensure ongoing personal information security. In case 
of substantial impactful changes of the main technical elements (components and services) including 
but not limited to iOS, Android, Web or the CMS; the team may discontinue the support of the 
application and therefore withdraw the App. Any changes we may make to our privacy statement in the 
future will be posted on the platform page and, where appropriate, notifications will be sent to users by 
e-mail. 
We know that the experiences of youth, parents/caregivers and service providers are interconnected 
and interdependent.25 We expect that in both groups, the parent(s)/caregiver(s) and health care 

NOTE: CTO#1666 Amendment approved by HiREB on January 9, 2023. Target sample was 264 
participants; however, we randomized 52 participants, and did not complete the full-scale RCT. Linking 
health card numbers with provincial administrative data is labour intensive and costly, and the small 
number would not provide meaningful results. As approved by ethics, we dropped the health economic 
outcome analysis (Health card number linking, Health Utilities Index (HUI), Resource Use Questionnaire 
(RUQ)) and destroy stored health card number data. 
Page 12 of 74                                         Protocol - READYorNotTM Brain-Based Disabilities Trial (version date: April 13, 2022) 
 
 
provider(s) naturally may provide support to participants. Youth, parent(s)/caregiver(s) and health care 
provider(s) will be provided reference handouts. These guidelines will help to standardize the nature 
and extent of the App support provided to participants in the intervention group. 
Data Collection 
Data source triangulation, or the use of multiple data sources (e.g., youth, parents/caregivers, health 
care providers) is a strategy used to gain understanding, ensure completeness, and confirm the 
credibility of findings 26. Triangulation also enables the researcher to ensure construct validity by 
providing multiple measures of the same phenomenon. In this study, both quantitative and qualitative 
data types will be collected, including: 1) in-depth semi-structured interviews; and 2) quantitative 
implementation and outcome data. 
All data collection forms will be available in both English and French.  
Baseline Visit 
In case the baseline visit is not done in-person (e.g. due to COVID-19 pandemic), we have added the 
option to conduct the baseline visit via Zoom meeting. In order to better establish rapport with 
participants, the RA may conduct the Zoom meeting with their own camera on. Participants will have 
the option to turn on their camera or keep it off. Zoom meetings will not be recorded. 
Following consent, all participants will be asked to complete a questionnaire package. All participants 
will complete the same baseline measures regardless of group assignment. All youth will be provided 
with sufficient time and support to complete the questionnaire package. Parents/caregivers will also 
complete a separate questionnaire package. Participants will complete the questionnaire package 
electronically or in printed form.  
Participants randomized into the intervention group will also have an introduction to the MyREADY 
TransitionTM BBD App intervention at the baseline visit. They will be provided with brief verbal and 
written instructions about how to use and engage with the intervention. The RA will navigate through 
the MyREADY TransitionTM BBD App intervention with the participant to give a short demonstration. The 
baseline visit for participants in the intervention group will take an estimated one and a half hours to 
complete in order to ensure that the participants feel able to use the MyREADY TransitionTM BBD App 
intervention. All RAs will receive intervention-facilitation training and treatment fidelity will be 
monitored.  
Follow-Up Visits 
Participants will repeat the questionnaire package at 6-months following baseline. The RA will arrange 
these visits at a mutually convenient time during follow-up appointments in clinic if possible, or 
otherwise outside of clinic time. If an in-person visit is not possible, the questionnaire package can be 
completed online, by mail or by phone interview according to the participant’s preference.   
Qualitative Data Collection 
In addition to the quantitative follow-up at 6-months, qualitative data will be collected for a subset of 
participants using interviews.  Up to 50 youth in the intervention group will be interviewed after the 6- 

NOTE: CTO#1666 Amendment approved by HiREB on January 9, 2023. Target sample was 264 
participants; however, we randomized 52 participants, and did not complete the full-scale RCT. Linking 
health card numbers with provincial administrative data is labour intensive and costly, and the small 
number would not provide meaningful results. As approved by ethics, we dropped the health economic 
outcome analysis (Health card number linking, Health Utilities Index (HUI), Resource Use Questionnaire 
(RUQ)) and destroy stored health card number data. 
Page 13 of 74                                         Protocol - READYorNotTM Brain-Based Disabilities Trial (version date: April 13, 2022) 
 
 
month quantitative data collection point to describe and understand primary study outcomes, as well as 
to capture process and user experience related to the secondary aims. We will purposefully select some 
participants who have completed only a low percentage of App content to learn about the experiences 
of those participants.  
One-on-one, semi-structured interviews will be carried out for the collection of in-depth description. 
These sessions lasting approximately 30-60 minutes will be conducted with a subset of youth 
participants, and separately with parents/caregivers and health care providers. All interviews will take 
place following the 6-month quantitative data collection point, will be conducted over virtual 
teleconference or phone, and will be audio-recorded. Qualitative interviews will be conducted by 
project staff supervised by Dr. Marelli at McGill University and/or by project staff supervised by Dr. 
Gorter at McMaster University. 
Data Safety Monitoring 
Due to the nature of the intervention as short term and low-risk, a formal Data Safety Monitoring Board 
is not necessary. In accordance with the core principles in the Tri-Council Policy Statement (TCPS 2) 
guidelines (http://www.pre.ethics.gc.ca), our safety monitoring plan will aim to ensure that (a) 
foreseeable risks to participants are minimized, and appropriately evaluated alongside potential 
benefits, (b) participants are clearly informed as to the nature of these foreseeable risks and potential 
benefits, (c) participant safety is monitored and accurately reported, and (d) any new information that 
may impact on the welfare of participants, or their decision to remain involved in a trial, be shared 
appropriately. The plan balances patient safety with study operations and doesn’t influence the care 
given to a patient if an adverse event, such as a mental health issue, occurs during the study.   
Data Storage and Transfer 
Prior to starting recruitment, McMaster University will put a data transfer agreement in place with each 
participating centre (listed in Appendix C) to govern the collection, storage, transfer, analysis, 
ownership, archiving, and destruction of study data.  
This project is part of a CHILD-BRIGHT’s pan-Canadian research program, studying new diagnostic tests, 
therapies, service models, and technologies to optimize the physical and mental health of Canadian 
children born with brain-based disorders as well as the well-being of their caregivers and families. 
Within CHILD-BRIGHT’S twelve patient-oriented research projects, there are several common ‘network-
wide’ data collection forms with the intention to pool results across projects. Relevant health economics 
data will be shared with the CHILD-BRIGHT network’s health economics team. A statement is included in 
the study consent forms to ensure that participants have consented to this transfer of anonymized data.  
Prior to sharing data, data transfer agreements will be established for the secure transfer of data from 
McMaster University to the Hospital for Sick Children (Dr. Wendy Ungar, CHILD-BRIGHT Health 
Economics) and; to the University of Alberta (Dr. Rick Watts, CHILD-BRIGHT Data Coordinating Centre. 
CORE Investigators will have access to anonymized data consistent with institutional guidelines. 

NOTE: CTO#1666 Amendment approved by HiREB on January 9, 2023. Target sample was 264 
participants; however, we randomized 52 participants, and did not complete the full-scale RCT. Linking 
health card numbers with provincial administrative data is labour intensive and costly, and the small 
number would not provide meaningful results. As approved by ethics, we dropped the health economic 
outcome analysis (Health card number linking, Health Utilities Index (HUI), Resource Use Questionnaire 
(RUQ)) and destroy stored health card number data. 
Page 14 of 74                                         Protocol - READYorNotTM Brain-Based Disabilities Trial (version date: April 13, 2022) 
 
 
Data storage, collection and access/transfer is described below for the various types of data being 
collected for this study.  
Data from surveys or questionnaires (related to primary and secondary aims)  
Data Collection: Data can be entered into REDCap22 by designated users or survey respondents 
from any computer with an internet connection. Surveys will be: a) completed on paper by a 
study participant and entered into the REDCap form by the RA at the recruitment site, or b) 
completed online by a study participant directly into the REDCap form. Since no identifying 
information is stored in REDCap, the link to electronic survey forms will be sent to the RA and the 
RA will email it to the participant. 
Data Storage: Data will be entered online and stored in REDCap22. REDCap is a secure web 
application for building and managing online surveys and databases (www.project-redcap.org). 
As in several Canadian pediatric academic centers, REDCap has been set up at McGill University 
and at McMaster University to support Faculty members’ research. REDCap questionnaire data 
for this project will be hosted by the Department of Pediatrics at McMaster University.  De-
identified data is stored on a secure, firewall protected server with regular backup in the Faculty 
of Health Sciences Computer Services Unit with only the https port available to the internet.   
Data Access/Transfer: Research staff in the Department of Pediatrics at McMaster University will 
be designated to the ‘Super Administator’ role in the REDCAp system.  Together with Dr. Gorter 
and Dr. Marelli, this person will establish user accounts and user rights for the project team.  
User accounts include electronic signatures comprised of a username and password and an audit 
trail is generated for all activity within each REDCap project.  The Computer Services Unit at 
McMaster University has access to the database, only for the purposes of IT support including 
regular server maintenance and software updates. A de-identified and encrypted database file of 
variables for the secondary health economics aim will be uploaded to MacDrop 
(https://drop.mcmaster.ca/login), an Enterprise File Storage and Synchronization Solution that is 
endorsed by McMaster, and that is managed by Computer Services Unit to securely house data 
onsite. Research staff at the Hospital for Sick Children (Dr. Wendy Ungar, CHILD-BRIGHT Health 
Economics) will be given access to a shared folder where they can upload and access the 
database file via MacDrop link. Similarly, a de-identified and encrypted database file of variables 
from the ‘network-wide’ forms will be shared via MacDrop link with research staff at the 
University of Alberta (Dr. Rick Watts, CHILD-BRIGHT Data Coordinating Centre). The license with 
Insignia Health for the Patient Activation Measure or PAM does have a data-sharing requirement 
for scoring purposes. The PAM survey scoring form is an Excel-based application that uses 
macros and requires an Internet connection to calculate PAM Score and Level using non-
personally identifiable, individual data captured through participants’ use of the PAM. 
Data from qualitative interviews   
Data Collection: Qualitative interviews will be conducted by project staff supervised by Dr. 
Marelli at McGill University and/or Dr. Gorter at McMaster University. All interviews will be 

NOTE: CTO#1666 Amendment approved by HiREB on January 9, 2023. Target sample was 264 
participants; however, we randomized 52 participants, and did not complete the full-scale RCT. Linking 
health card numbers with provincial administrative data is labour intensive and costly, and the small 
number would not provide meaningful results. As approved by ethics, we dropped the health economic 
outcome analysis (Health card number linking, Health Utilities Index (HUI), Resource Use Questionnaire 
(RUQ)) and destroy stored health card number data. 
Page 15 of 74                                         Protocol - READYorNotTM Brain-Based Disabilities Trial (version date: April 13, 2022) 
 
 
conducted over Zoom or by phone, and will be audio-recorded. Audio recordings will be 
transcribed as soon as possible after being collected, and recordings will be destroyed following 
transcription and once the transcripts have been checked for accuracy. 
Data Storage: For interview data collected at McMaster University, interview recordings and 
transcripts will be collected and uploaded to MacDrop (https://drop.mcmaster.ca/login), an 
Enterprise File Storage and Synchronization Solution that is endorsed by McMaster, and that is 
managed by Computer Services Unit to securely house data onsite. For interview data collected 
at McGill University, interview recordings and transcripts will be collected and uploaded to 
McGill OneDrive for Business, a Microsoft Enterprise File Storage and Synchronization Solution 
that is provided by Microsoft to McGill. The McGill OneDrive for Business solution is protected by 
a negotiated a contract between McGill and Microsoft that respects Canadian and Quebec 
privacy laws and protects the intellectual property of data stored on OneDrive for Business; 
these commitments are not guaranteed by the consumer class version of OneDrive, or other self-
provisioned cloud storage services. McGill OneDrive for Business is managed by McGill IT 
Resources to securely house data onsite. In addition, McGill data could be entered online and 
stored in REDCap and administered by the McGill team and CIM (Centre for Intelligent Machines, 
the inter-departmental inter-faculty research group which was formed in 1985 to facilitate and 
promote research on intelligent systems). De-identified data will be stored on a secure, firewall 
protected server with regular backup managed by CIM with only the https port available to the 
internet. Data can be entered by designated users or survey respondents from any computer 
with an internet connection. 
Data Access/Transfer: For interview data collected at McMaster University, McGill project staff 
will be given access to a shared folder where they can upload and access de-identified transcripts 
or qualitative reports via MacDrop link. For interview data collected at McGill University, 
McMaster project staff will be given access to a secure encrypted file transfer system, endorsed by 
the Research Institute of the McGill University Health Centre. Recordings will not be shared between 
the two sites.  
Data from the App (primary and secondary aims) 
Data Collection: Intervention User Metrics data will be collected and stored within the MyREADY 
TransitionTM BBD App hosted and maintained by RootQuotient.  
Data Storage: In order for the application to sync with the content management system, and in 
order to provide technical support as needed, the MyREADY TransitionTM BBD App retrieves the 
device brand and model, its Operating System (Apple vs Android), and the name of the device 
(e.g. "Bert's iPhone"). If the user uses the support request feature it will send an email to the 
support email address. The email will include the information mentioned above as well as the 
version number of the application (e.g. RCT2019BBD) that the user has installed. The application 
also records the level of completion of the sections as the user goes along, and the information 
the user may have provided during registration process. This information is securely stored in the 
content management system. The MyREADY TransitionTM BBD App will have access to the 

NOTE: CTO#1666 Amendment approved by HiREB on January 9, 2023. Target sample was 264 
participants; however, we randomized 52 participants, and did not complete the full-scale RCT. Linking 
health card numbers with provincial administrative data is labour intensive and costly, and the small 
number would not provide meaningful results. As approved by ethics, we dropped the health economic 
outcome analysis (Health card number linking, Health Utilities Index (HUI), Resource Use Questionnaire 
(RUQ)) and destroy stored health card number data. 
Page 16 of 74                                         Protocol - READYorNotTM Brain-Based Disabilities Trial (version date: April 13, 2022) 
 
 
camera and microphone on the device when the user is completing the selfie-audio and selfie-
video recording challenges. It will only have access when the application is open, and when 
activated by the user. These recordings will be stored locally on the phone and only a 
confirmation that the challenge has been completed will be sent to the content management 
system, not the audio-video files themselves. 
Datacenter Security Policies: RootQuotient is using services of Amazon Web Services, and their 
datacenter is hosted in Montreal, Quebec, Canada. Datacenter policies are described in a 
document “Amazon Web Services: Overview of Security Processes (May 2017)” created by 
Amazon (https://d1.awsstatic.com/whitepapers/Security/AWS_Security_Whitepaper.pdf)  
Service Provider Security Policies:  
• SSH access to server is restricted to access only through our corporate VPC or through 
corporate static IP address. 
• CMS and API’s are secured with https protocol and authentication with different roles. 
• Users have different security levels in S3 Bucket to upload and read the application materials. 
• Client App (iOS & Android) authenticate the users, request the content and meta files to 
download the application materials. 
• All the persistent data are stored in the application container inside the mobile apps and it’s 
not accessible anywhere outside the App. 
Backup Management: Backups are scheduled to run on daily, weekly and monthly basis over the 
persistent data such as database, files & folders. 
Configuration Management:  
 
 
Hardware 
Environment 
Details 
Test Server 
cms.childbright.360medlink.net 
& 
api.childbright.360medlink.net 
RAM (in MB) 
8000 
CPUs 
2 vCPUs 
Virtual/Physical 
Virtual 
Server Type 
Web /App server 
 
OS 
OS Type 
Ubuntu 
Vendor (for OS) 
Open Source 
OS version 
16.04 LTS 
Patch 
- 

NOTE: CTO#1666 Amendment approved by HiREB on January 9, 2023. Target sample was 264 
participants; however, we randomized 52 participants, and did not complete the full-scale RCT. Linking 
health card numbers with provincial administrative data is labour intensive and costly, and the small 
number would not provide meaningful results. As approved by ethics, we dropped the health economic 
outcome analysis (Health card number linking, Health Utilities Index (HUI), Resource Use Questionnaire 
(RUQ)) and destroy stored health card number data. 
Page 17 of 74                                         Protocol - READYorNotTM Brain-Based Disabilities Trial (version date: April 13, 2022) 
 
 
Application/Web Server 
Details 
Application Server version 
nginx/1.10.3 
Application Server 
Nginx 
 
Database 
Type 
MySQL 
Vendor (Database vendor) 
Oracle 
Version 
5.7 
Storage 
Amazon Elastic Block Store 
(Amazon EBS) 
Persistent block storage 
volumes for the server instance 
- 50 GiB 
 
AWS S3 
Object storage service 
Data Access/Transfer: Intervention User Metrics data from the MyREADY TransitionTM BBD App 
will be exported to Excel and will also be presented in a website dashboard (“Compliance 
Dashboard”). The Content Management System (CMS) is hosted by Amazon and managed jointly 
by a software company (RootQuotient) and the Research IT team at McGill University. To 
support the primary aims analysis that will take place at McMaster University, McMaster project 
staff will be given access to the intervention user metrics data. Research staff in the Research 
Institute of the McGill University Health Centre will be designated to the ‘Super Administator’ 
role in the CMS system dashboard.  Together with Dr. Gorter and Dr. Marelli, this person will 
establish user accounts and user rights for project staff.  
Using Zoom for Research Involving Human Participants 
Due to the COVID-19 pandemic, we have added the option for the RA to use the Zoom platform 
to conduct study visits remotely with participants as well as to carry out qualitative interviews. 
Zoom gives the ability for people to easily join meetings using either a telephone and/or 
computer connection, with a shared digital whiteboard. Zoom provides information about 
compliance with PIPEDA / PHIPA (see Zoom privacy statement for Canadian customers: 
https://zoom.us/docs/doc/PIPEDA_PHIPA Canadian Public Information Compliance Guide.pdf). 
McMaster accounts only route traffic through Canadian data centres. We note that Zoom does 
use 256-bit encryption, however information is not encrypted on the Zoom servers themselves. 
The RA will not record Zoom meetings so any concerns about how recordings may be stored to 
do not apply to this study. When security settings for meetings are not properly configured, it is 
possible for uninvited guests to join meetings. This is called “Zoom bombing” and in some 
situations, guests have joined meetings with the intent of being disruptive, which could be very 
troubling for research participants. Therefore, the RA will be trained to take cautionary measures 
when configuring Zoom meeting options. For example:  
• 
Meeting ID: Generate Automatically 
• 
Meeting Password: Require a meeting password 
• 
Enable waiting room (to prevent uninvited guests) 

NOTE: CTO#1666 Amendment approved by HiREB on January 9, 2023. Target sample was 264 
participants; however, we randomized 52 participants, and did not complete the full-scale RCT. Linking 
health card numbers with provincial administrative data is labour intensive and costly, and the small 
number would not provide meaningful results. As approved by ethics, we dropped the health economic 
outcome analysis (Health card number linking, Health Utilities Index (HUI), Resource Use Questionnaire 
(RUQ)) and destroy stored health card number data. 
Page 18 of 74                                         Protocol - READYorNotTM Brain-Based Disabilities Trial (version date: April 13, 2022) 
 
 
• 
Video: Both host and participant video should be set to ‘off’ on entry.  
• 
Audio: Participants should be given the option to join by telephone only. Provide a toll-free 
dial in number to make sure there are no accidental costs to participants. 
Health Card Number Data (secondary health economics aim) 
Data Collection: The youth participant’s Health Card Number is requested and recorded on the local 
Consent form by parents/caregivers who agree to share it. Participants can choose not to share the 
child’s Health Card Number and still participate in the study.   
 
Data Storage: The youth participant’s Health Card Number is requested and recorded on the Consent 
form by parents/caregivers who agree to share it. They can choose not to share the child’s Health Card 
Number and still participate in the study.  Health Card Numbers will not be entered into REDCap. The RA 
at each recruitment site will record the participant’s study identification number and Health Card 
Number into an excel file which will be stored on a secured drive, separate from other study data.   
 
Data Access/Transfer: The RCT Research Coordinator at McMaster University will create a designated 
folder with limited privileges on MacDrop (https://drop.mcmaster.ca), an Enterprise File Storage and 
Synchronization Solution that is endorsed by McMaster, and that is managed by Computer Services Unit 
to securely house data onsite. An excel file containing only youth participant study identification 
number and Health Card Number will be encrypted and uploaded to the designated folder by the RA at 
each recruitment site.  We will work with appropriate organizations in each province (Alberta, Ontario, 
Quebec, New Brunswick, and Nova Scotia) to gain approval to access provincial Health Services 
Administrative Data repositories. Health care services data recorded in the provincial administrative 
databases will be linked deterministically to the study data using the Health Card Number, partial date 
of birth and sex. 
 
The RCT will be centrally managed by the RCT Coordinator at McMaster University’s CanChild. Research 
files will be stored on the CanChild Active Directory at McMaster, on a secure network that is in a tier 3 
data facility. The CanChild Active Directory is a firewall protected server to which only the PIs and 
Research Coordinators will have access. Remote access to the CanChild directory is via VPN. Any 
personal information collected will be entered into password-protected SPSS or excel files and stored on 
the CanChild Active Directory, separate from other study data. Qualitative data will be stored on the 
CanChild Active Directory and managed electronically using NVivo, a qualitative data analysis software 
system. Research staff will password protect their electronic and audio digital files from the interview 
sessions, and can transmit these into the secure cloud storage provided through McMaster's MacDrop 
(https://drop.mcmaster.ca/login), with final storage on the CanChild Active Directory. 
Incentives for Participation:  
As a token of appreciation for their time to participate, all youth participants will be given a $20 gift card 
and parents/caregivers will have their parking or travel costs covered at the baseline and 6-month visit. 

NOTE: CTO#1666 Amendment approved by HiREB on January 9, 2023. Target sample was 264 
participants; however, we randomized 52 participants, and did not complete the full-scale RCT. Linking 
health card numbers with provincial administrative data is labour intensive and costly, and the small 
number would not provide meaningful results. As approved by ethics, we dropped the health economic 
outcome analysis (Health card number linking, Health Utilities Index (HUI), Resource Use Questionnaire 
(RUQ)) and destroy stored health card number data. 
Page 19 of 74                                         Protocol - READYorNotTM Brain-Based Disabilities Trial (version date: April 13, 2022) 
 
 
With the adaptations due to the COVID-19 pandemic, when study visits are not done in-person, the 
parking or travel cost no longer applies. The parent/caregiver will receive a $20 gift card at each visit 
instead as a small token of thanks. E-gift cards will be offered to participants for ease of administration 
and use due to COVID-19 closures. In addition to the gift card at each visit, we will offer youth 
participants who are in high school, 1 hour of volunteer community service (which can be used towards 
the graduation requirement if the high school allows). Those who participate in the qualitative data 
collection will also be given a $20 gift card as a token of appreciation. All participants will be given the 
option to receive a plain language report at the end of the study, after the RCT results have been 
analyzed.  
STUDY MEASURES:  
The quantitative study measures are briefly described here and the full set of data collection booklets 
are provided separately. All data collection forms will be available in English and French. 
Demographic Information  
We will collect detailed demographic information from parents/caregivers at the Baseline visit in order 
to describe and compare our sample to other youth with BBD in Canada. The CHILD-BRIGHT network 
has developed a set of standard forms for common reporting among network projects: “Network-wide 
Demographic Information Form” and “Network-wide Profile Information Form: Child and Parent(s)”. 
Primary Measures  
Transition Readiness Assessment Questionnaire (TRAQ) 9  
While TRAQ measure refinement is ongoing, and other versions are now available, our sample size 
calculation is based on findings from an intervention trial 19 where the 29-item version of the TRAQ was 
used. The 29-item version has a Self-management domain (16 items) and a Self-advocacy domain (13 
items). Each item is scored 1-5, where 1 = “No, I do not know how” and 5 = “Yes, I always do this when I 
need to.” The TRAQ will be completed by youth participants in both groups at Baseline and at 6-Months. 
It takes 5-10 minutes to complete.  
Utilization of Intervention (Usage, Adherence, and Fidelity) 
We will use mixed methods to assess MyREADY TransitionTM BBD App usage, adherence and fidelity, and 
to identify the barriers and facilitators to using the IT platform for users. 
Quantitative Data: We will track and analyze quantitative measures of App use for all components of 
the IT platform using two approaches: 
1. MyREADY TransitionTM BBD App will include support for utilization data gathering that can be 
used during the intervention and evaluation phases of the project. For example, it will log data 
related to end user login to the system (times and dates), section/session/video views (including 
sub-section features), challenge completion, access to games at the arcade, time spent on a 
section/session and trends over time. In addition, we will track and assess 
sections/sessions/videos progress by patient condition and/or other user variables by 
referencing information from the user profile. Within the App, users are asked to provide 

NOTE: CTO#1666 Amendment approved by HiREB on January 9, 2023. Target sample was 264 
participants; however, we randomized 52 participants, and did not complete the full-scale RCT. Linking 
health card numbers with provincial administrative data is labour intensive and costly, and the small 
number would not provide meaningful results. As approved by ethics, we dropped the health economic 
outcome analysis (Health card number linking, Health Utilities Index (HUI), Resource Use Questionnaire 
(RUQ)) and destroy stored health card number data. 
Page 20 of 74                                         Protocol - READYorNotTM Brain-Based Disabilities Trial (version date: April 13, 2022) 
 
 
optional feedback at the end of each section. Thus, we can track and analyze the extent and 
frequency of use, and the profiles of patients who did or did not use the IT platform. This 
information will be exported to Excel and will also be presented in a website dashboard 
(“Compliance Dashboard”). 
2. For all participants in the intervention group, self-reported usage of the IT platform will also be 
assessed at the end of the intervention. This assessment will focus on users' utilization of the 
application and its features, the perceived value and their experience and satisfaction with the 
intervention. The self-reported survey will provide additional information about the users' 
adherence, behavior, motivation and experience with the IT platform, as well as about the main 
reasons for using or not using it. The System Usability Scale (SUS) will be administered to youth 
in the Experimental Arm at the 6 Month visit. The measure focus is on users' utilization of the 
App and its features, the perceived value and their experience and satisfaction with the 
intervention. The self-reported survey will provide additional information about the users' 
adherence, behavior, motivation and experience with the IT platform, as well as about the main 
reasons for using or not using it.  
The mHealth App Usability Questionnaire (MAUQ) will be administered after end of exposure 
(completion of the intervention) as well as at the 6-month follow-up interview. While the SUS 
has been more widely used, the MAUQ is the first reliable usability questionnaire specifically 
designed to assess the usability of mHealth Apps created for patients and providers.  
We will administer the MAUQ immediately following completion of the App in an effort to reduce 
potential recall bias of users’ experiences with the App. There may be variation in the length of time that 
youth take to complete the App; Participants are given 6 months to complete the App, however they may 
choose to complete it more quickly. Having participants answer the items on the MAUQ as soon as 
possible after completing the App, will allow for important “in-the-moment” feedback. Since other 
outcome measures will be administered at the 6-month follow-up visit, we will also re-administer the 
MAUQ at that time. This will allow for user experience with the App to be captured at the same moment-
in-time as the other outcomes, and for clearer interpretation of data analysis that includes variables of 
user experience. 
Qualitative Data: After the intervention period, we will conduct semi-structured interviews with a 
subset of youth in the intervention group, and parents/caregivers. The purpose will be to better 
understand how the App was used initially and over time, barriers and facilitators to its use, and 
participants’ experience and satisfaction with the App intervention. Our qualitative evaluation will 
also focus on their perceptions of the MyREADY TransitionTM BBD App design and content; 
implementation; most and least beneficial aspects; and the main challenges and opportunities for 
improvements. We will develop and use interview guides to facilitate discussion topics and 
standardize data collection. All interviews will be transcribed verbatim, coded, and themes will be 
extracted. We will synthesize lessons learned from all these activities into best practices for 
implementation, adoption, and use of the intervention. 
Secondary Measures 

NOTE: CTO#1666 Amendment approved by HiREB on January 9, 2023. Target sample was 264 
participants; however, we randomized 52 participants, and did not complete the full-scale RCT. Linking 
health card numbers with provincial administrative data is labour intensive and costly, and the small 
number would not provide meaningful results. As approved by ethics, we dropped the health economic 
outcome analysis (Health card number linking, Health Utilities Index (HUI), Resource Use Questionnaire 
(RUQ)) and destroy stored health card number data. 
Page 21 of 74                                         Protocol - READYorNotTM Brain-Based Disabilities Trial (version date: April 13, 2022) 
 
 
Population Health 
Canadian Occupational Performance Measure (COPM)27 
The COPM is an evidence-based, generic, and individualized outcome measure used to capture a client’s 
self-perception of performance and satisfaction in everyday living, over time.27 The measure 
accomplishes this by identifying problems in performing activities of daily living. The patient is 
encouraged to think about things that they want to do, need to do or are expected to do but can’t do, 
don’t do or aren’t satisfied with the way they do. Concrete examples are provided for youth: things 
related to their health and personal care, getting ready for the day, getting around, working, household 
chores, school, recreation, socializing. The patient is asked to rate current performance of each using a 
10-point scale from ‘not able to do it’ to ‘able to do it very well’. The patient is also asked to rate 
satisfaction with performance on a 10-point scale from ‘not satisfied at all’ to ‘extremely satisfied’ with 
higher scores reflecting better performance and satisfaction with performance as perceived by the 
patient. The performance and satisfaction can be re-assessed following a period of treatment.28 The 
COPM will be administered to all youth participants.  
TRANSITION-Q20 
The TRANSITION-Q is a 14-item transition readiness/self-management ability scale.20, 29 This short, 
clinically meaningful and psychometrically sound scale can be used in research and in pediatric and 
adolescent clinics to help evaluate readiness for transition.20 Item responses (“never” = 0, “sometimes” 
= 1, and “always” = 2) are summed to create a raw score, with a possible range from 0 to 28. Raw scores 
are transformed using a table provided by the developers and the transformed scores range from 0-100. 
A higher score indicates greater transition readiness; exhibiting more self-management skills with higher 
frequency.20, 30  
A TRANSITION-Q score > 40 will be used as an eligibility screen to define a minimum threshold for 
transition readiness based on our earlier work. At the point of screening and recruitment, we will 
evaluate several components of cognitive function as well as youth capacity to share the management 
of their own health. The TRANSITION-Q screen will inherently exclude youth who have severe 
intellectual disability, and/or those who rely significantly on parents/caregivers in most areas of daily 
functioning, self-care and/or communication. We will repeat the TRANSITION-Q at the 6-Month Follow-
Up Visit.  
PedsQLTM Pediatric Quality of Life Instrument, Generic Core, Teen Report (13-18 years) 11 
The PedsQLTM Measurement Model (http://www.pedsql.org/about_pedsql.html) is a modular approach 
to measuring health-related quality of life (HRQOL) in healthy children and adolescents and those with 
acute and chronic health conditions.  
In this study the PedsQLTM Pediatric Quality of Life Instrument, Generic Core, Teen Report (13-18 years) 
will be completed by youth participants at Baseline and 6-Months. The form is brief (23 items), practical 
(less than 4 minutes to complete), multidimensional (Physical, Emotional, Social, School Functioning), 
reliable (Child Self-Report; 0.90) and valid (Distinguishes between healthy children and children with 

NOTE: CTO#1666 Amendment approved by HiREB on January 9, 2023. Target sample was 264 
participants; however, we randomized 52 participants, and did not complete the full-scale RCT. Linking 
health card numbers with provincial administrative data is labour intensive and costly, and the small 
number would not provide meaningful results. As approved by ethics, we dropped the health economic 
outcome analysis (Health card number linking, Health Utilities Index (HUI), Resource Use Questionnaire 
(RUQ)) and destroy stored health card number data. 
Page 22 of 74                                         Protocol - READYorNotTM Brain-Based Disabilities Trial (version date: April 13, 2022) 
 
 
acute and chronic health conditions; distinguishes disease severity within a chronic health condition), 
and responsive to clinical change over time. 
Patient Activation Measure (PAM) (health-related self-efficacy)31 
The Patient Activation Measure (PAM) (http://www.insigniahealth.com/products/pam-survey) is a valid, 
reliable, unidimensional, Guttman-like scale based on a developmental model of activation. Activation 
appears to involve four stages: (1) believing the patient role is important, (2) having the confidence and 
knowledge necessary to take action, (3) actually taking action to maintain and improve one’s health, and 
(4) staying the course even under stress. The measure has good psychometric properties indicating that 
it can be used at the individual patient level to tailor intervention and assess changes.31 
PAM originally included 22 questions, and through subsequent validation of the short-version now has 
13 questions. The PAM assesses a patient's confidence in self-managing their condition, and the total 
score allows individuals to be categorized into four levels:  
Level 1 - disengaged and overwhelmed 
Level 2 - becoming aware, but still struggling  
Level 3 - taking action  
Level 4 - maintaining behaviours and pushing further  
PAM has been used as a measure of health-related self-efficacy in a relevant study of transition to adult 
care among adolescents with chronic disease utilizing a technology program.32  Hibbard et al. 33 reported 
on the relationship between increased patient activation and improved self-management.  
The license with Insignia Health for the PAM Measure does have a data-sharing requirement for scoring 
purposes. This is necessary to score each survey as there is not a scoring algorithm or rubric that is a 
static formula. 
Health Care Experience 
Measure of Process of Care (MPOC) (family-centred care) 34  
The Measure of Processes of Care (https://canchild.ca/en/resources/47-measure-of-processes-of-care) 
is a well-validated and reliable self-report measure of parents' perceptions of the extent to which the 
health services they and their child(ren) receive are family-centred. The original version of MPOC is a 56-
item questionnaire; as of 1999 there is a shorter, 20-item version. MPOC has been used internationally 
in many evaluations of family-centred service. Parents/caregivers will complete the MPOC-20 at 
Baseline and 6-Months. Additional mixed methods questions related to satisfaction with service will be 
complement the information collected within the MPOC.  
Newest Vital Sign 35 
As a complement to our Baseline measures, we will include a health literacy measure called Newest 
Vital Sign (NVS) http://www.pfizer.com/health/literacy/public-policy-researchers/nvs-toolkit. It is 
appropriate for our population and research purpose and can be easily administered in 3 minutes. The 
NVS measures words and numbers, not just word recognition. Since the NVS was published in December 

NOTE: CTO#1666 Amendment approved by HiREB on January 9, 2023. Target sample was 264 
participants; however, we randomized 52 participants, and did not complete the full-scale RCT. Linking 
health card numbers with provincial administrative data is labour intensive and costly, and the small 
number would not provide meaningful results. As approved by ethics, we dropped the health economic 
outcome analysis (Health card number linking, Health Utilities Index (HUI), Resource Use Questionnaire 
(RUQ)) and destroy stored health card number data. 
Page 23 of 74                                         Protocol - READYorNotTM Brain-Based Disabilities Trial (version date: April 13, 2022) 
 
 
2005, it has appeared in more than 25 peer-reviewed studies. It has been used to assess health literacy 
in populations with varying age, among racial/ethnic minorities, and applied to a wide variety of health 
conditions. The NVS will allow us to describe our population at baseline and explore determinants of 
change in self-management, as well as tailoring the intervention in the knowledge translation phase. 
Cost Utility/Cost Effectiveness 
The measure of effectiveness used in the cost effectiveness analysis will be consistent with the primary 
outcome measures. Additional measures will examine health-related quality of life using the PedsQLTM 11 
and the Health Utilities Index (HUI)®10. The use of a preference-based measure such as the HUI will 
permit the calculation of quality-adjusted life years (QALYs) for a cost-utility analysis. 
Health Utilities Index® (Hui2/3) Proxy-Assessed (health-related quality of life) 10 
The HUI is a generic health status instrument developed in Canada for use with children and has been 
incorporated in numerous clinical studies as well as the Canadian Community Health Survey, allowing 
the generation of norms for most age groups.10 The HUI Mark II includes 7 attributes: Sensation, 
Mobility, Emotion, Cognition, Self-care, Pain and Fertility with each attribute divided into 3 to 5 levels. 
The HUI III includes 8 attributes: Vision, Hearing, Speech, Ambulation, Dexterity, Emotion, Cognition and 
Pain. Each attribute of the HUI III consists of 5 to 6 levels. The organization of attributes by level of 
functioning is referred to as the HUI classification system. The HUI II and III use the same questionnaire 
to map individual responses to classification levels. As the mapping is done at the analysis phase, there 
is no additional response burden for completing the HUI. A multi-attribute utility function is used to 
assign weights to each level for each attribute. The weights for each attribute are combined statistically 
to derive a single utility using a scoring formula. Separate pre-established and validated utility functions 
and scoring formulae exist for the HUI II and III. Since the attributes in the HUI II and III are structurally 
independent, the user can produce attribute scores, in addition to overall utility for a health state that 
range from 0 to 1. To reduce burden on youth participants in this study, we will use the Health Utilities 
Index® (Hui2/3) Proxy-Assessed which will be self-administered by parent/caregiver proxies. The 
completion time is 5-7 minutes. 
Resource Use Questionnaire (RUQ) 12 
The RUQ 12 is typically an interviewer-administered questionnaire for parents of children aged 11 to 18 
years. The original RUQ measures the family resource use of condition-related treatments, services and 
programs, as well as parent time losses and family out-of-pocket costs. It also documents condition-
related government subsidies and funding that families receive. Resources measured include those 
delivered by a parent, by other providers (e.g. behavioural specialist) or a combination of both. In this 
project, we will use a modified subset of RUQ questions, self-completed by the parent/caregiver. We 
have also included some questions to capture information about serious illnesses during the study 
including information about hospitalizations, ICU admissions, etc.  
Provincial Data on Use of Health Care Services 
We will request consent to obtain youth participant’s Health Card Number to answer questions about 
use of health services like physician office visits, visits to emergency rooms and hospitalizations during 

NOTE: CTO#1666 Amendment approved by HiREB on January 9, 2023. Target sample was 264 
participants; however, we randomized 52 participants, and did not complete the full-scale RCT. Linking 
health card numbers with provincial administrative data is labour intensive and costly, and the small 
number would not provide meaningful results. As approved by ethics, we dropped the health economic 
outcome analysis (Health card number linking, Health Utilities Index (HUI), Resource Use Questionnaire 
(RUQ)) and destroy stored health card number data. 
Page 24 of 74                                         Protocol - READYorNotTM Brain-Based Disabilities Trial (version date: April 13, 2022) 
 
 
the study period (between the time a participant joins the study up until March 31, 2021). Participants 
have the option of participating in the study without sharing the youth’s Health Card Number. The 
Health Card Number will allow us to link the information provided in the study with provincial data 
regarding the participant’s use of health services. We will work with appropriate organizations in each 
province (Alberta, Ontario, Quebec, New Brunswick, and Nova Scotia) to gain approval to access 
provincial Health Services Administrative Data repositories. Health care services data recorded in the 
provincial administrative databases will be linked deterministically to the study data using the Health 
Card Number, partial date of birth and sex. 
STATISTICAL ANALYSIS: 
We will use regression analysis to adjust for baseline function as a sensitivity analysis to address any 
residual imbalance from the randomization.  
The analysis and reporting of the internal pilot phase and main RCT results with follow CONSORT 
guidelines23-24. Patient demographics and baseline outcome variables (both primary and secondary) will 
be summarized using descriptive summary measures. All analyses will be performed using SAS 9.4 (Cary, 
NC). Analysis will be intention to treat. The table below provides a summary of study objectives, 
corresponding outcomes, hypotheses and methods of analyses. For subgroup analysis we will use 
interaction test (interaction of subgroup variable with the treatment or intervention variable). 
 
Table 1: Study Objectives, Corresponding Outcomes, Hypotheses and Methods of Analyses 
 
Objective 
Outcome 
Criterion for 
Success 
Method of 
Analysis 
Primary:  
To determine 
whether the 
MyREADY 
TransitionTM BBD 
App intervention 
will result in 
improved transition 
readiness 
Primary: 
• Change in TRAQ self-
management score from 
Baseline to 6 months. 
Intervention > 
Control 
T-Test; p<0.05 
Secondary: 
• Change in TRAQ self-advocacy 
score from Baseline to 6 
months. 
Intervention > 
Control 
T-Test; p<0.05 
Secondary 
• Health care transition 
experience 
Individual semi-
structured 
interviews  
Qualitative 
Methods  
Secondary: 
What is the effect of 
the MyREADY 
TransitionTM BBD 
App intervention for 
Primary:  
Population Health 
• Serious illness (hospitalizations, 
ICU admission questions from 
Resource Use Questionnaire 
Intervention > 
Control 
 
T-Test for 
continuous 
outcomes 
 
Chi-square test  

NOTE: CTO#1666 Amendment approved by HiREB on January 9, 2023. Target sample was 264 
participants; however, we randomized 52 participants, and did not complete the full-scale RCT. Linking 
health card numbers with provincial administrative data is labour intensive and costly, and the small 
number would not provide meaningful results. As approved by ethics, we dropped the health economic 
outcome analysis (Health card number linking, Health Utilities Index (HUI), Resource Use Questionnaire 
(RUQ)) and destroy stored health card number data. 
Page 25 of 74                                         Protocol - READYorNotTM Brain-Based Disabilities Trial (version date: April 13, 2022) 
 
 
improving health 
and use of health 
systems? 
• PedsQLTM Pediatric Quality of 
Life 
• Patient Activation Measure 
(PAM) (health-related self-
efficacy) 
• TRANSITION-Q  
for hospitalization 
Secondary:  
• Utilization MyREADY 
TransitionTM BBD App 
User metrics built 
into MyREADY 
TransitionTM BBD 
App intervention to 
assess the extent to 
which various 
components of the 
intervention are 
accessed 
Descriptive 
Secondary:  
Cost utility/cost-effectiveness 
• Health Utilities Index® (Hui2/3) 
• Resource Use Questionnaire  
Evaluate changes in 
patients’ health in 
relation to changes 
in cost to assess if 
the intervention 
represents an 
efficient allocation 
of health care 
resources 
Descriptive;  
Cost-effectiveness 
analysis with 
support from 
Child-Bright 
health economics 
network team  
 
 
Secondary: 
• Achievement of health/life 
goals 
• Canadian Occupational 
Performance Measure (COPM)  
Intervention > 
Control 
 
Paired Student 
t-tests to compare 
mean ratings for 
performance and 
satisfaction on the 
COPM scoring 
system (10-point 
scale) with > 2 
points difference 
as clinically 
meaningful 
difference  
 
Qualitative Data Analysis 
In this study, individual interviews will be audio-recorded and transcribed verbatim. Data will be stored 
and managed electronically using NVivo® Version 11 36, a qualitative research data management 

NOTE: CTO#1666 Amendment approved by HiREB on January 9, 2023. Target sample was 264 
participants; however, we randomized 52 participants, and did not complete the full-scale RCT. Linking 
health card numbers with provincial administrative data is labour intensive and costly, and the small 
number would not provide meaningful results. As approved by ethics, we dropped the health economic 
outcome analysis (Health card number linking, Health Utilities Index (HUI), Resource Use Questionnaire 
(RUQ)) and destroy stored health card number data. 
Page 26 of 74                                         Protocol - READYorNotTM Brain-Based Disabilities Trial (version date: April 13, 2022) 
 
 
software program to facilitate the organization and coding of large quantities of data. Conventional 
content analysis 37 will be used to code, categorize and synthesize the data to contextualize the analysis 
of the primary aim of the RCT. In addition, data related to usability of the App will be monitored and 
analyzed by the Research IT team at McGill to inform ongoing App development. 
Health Economic Evaluation 
Cost-effectiveness analyses will be conducted from the perspective of the provincial payer, as well as 
from the perspectives of society and the family, data permitting. Costing relates to the cost to develop 
and resources to support the intervention as well as resources used for treatment/management of 
participants’ health conditions over the study. Health resource use data include things such as the 
intervention, physician services, rehabilitation services, emergency services, hospital admissions, and 
medications. We anticipate that the health economic evaluation in this project will be exploratory since 
we are engaging youth during the timeframe prior to transition. However, there will also be opportunity 
to explore provincial jurisdiction patterns. There may be interest to submit a proposal to follow up this 
cohort in the future once they have landed in adult care, which would give us more of an opportunity to 
explore these health economics questions. 
A decision tree that models the comparison groups will be constructed. The model will include all 
relevant outcomes and events that may occur with each group throughout the study period. A 3% 
discount rate will be applied to outcomes and costs extending beyond one year. All measurement and 
analytic assumptions made for the base case analysis will be clearly stated. The 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. Subgroups of patients based on baseline demographic factors may be 
analyzed separately, if appropriate. Extensive sensitivity analysis including probabilistic sensitivity 
analysis will be undertaken to test the robustness of the results to variations in underlying assumptions. 
PATIENT ENGAGEMENT: 
Patient-oriented research refers to a continuum of research that engages patients as partners, focuses 
on their priorities and ultimately improves the value of research. Under this model, patients and 
parents/caregivers with "lived experiences," together with health professionals, join researchers as 
members of the research team. Most importantly, what patients and parents/caregivers contribute is 
the sharing of their values and priorities. Doing so ensures that the research conducted is meaningful 
and will have impact on those most affected by its outcomes. The entire team collaborates to plan and 
conduct the research, and to disseminate its findings. Researchers, research staff, and stakeholders 
involved in this project are encouraged to participate in the CIHR training curriculum in patient-oriented 
research to forefront the meaningful engagement of key stakeholders throughout our project.  
READYorNotTM Brain-Based Disabilities Trial is a patient-oriented project of CHILD-BRIGHT, a pan-
Canadian research program generally aimed at optimizing physical and mental health of youth with BBD 
and their families. In the READYorNotTM Brain-Based Disabilities Trial, the MyREADY TransitionTM BBD 

NOTE: CTO#1666 Amendment approved by HiREB on January 9, 2023. Target sample was 264 
participants; however, we randomized 52 participants, and did not complete the full-scale RCT. Linking 
health card numbers with provincial administrative data is labour intensive and costly, and the small 
number would not provide meaningful results. As approved by ethics, we dropped the health economic 
outcome analysis (Health card number linking, Health Utilities Index (HUI), Resource Use Questionnaire 
(RUQ)) and destroy stored health card number data. 
Page 27 of 74                                         Protocol - READYorNotTM Brain-Based Disabilities Trial (version date: April 13, 2022) 
 
 
App has been developed and evaluated to promote transition readiness (self-management and self-
advocacy skills) in preparing youth for the transition from pediatric to adult health care. 
In Part 1 of the project, Heath Information Technology (HIT) development was based on Agile 
Programming Best Practices. 38 This gave us flexibility and helped us to integrate feedback from 
researchers, research staff, as well as youth, parent/caregiver and health care stakeholders. The 
research team worked in partnership with PFAC to plan interviews, focus groups and other interactive 
testing activities to ensure the MyREADY TransitionTM BBD App matches the needs and interests of 
youth. In Part 2 of the project, the PFAC will continue to collaborate and guide the research team in 
planning this RCT to study the MyREADY TransitionTM BBD App in health care settings across Canada. 
Then, in the Part 3 of the project, patients, parents/caregivers and health care stakeholders will 
continue to be involved to ensure findings of the project are communicated in innovative and 
understandable ways, and can reach those who need it most. All partners will continue to work together 
to define future research directions.  
Members of our team are working together with CHILD-BRIGHT’s Citizen Engagement Council, Ontario 
SPOR Support Unit (OSSU) and Maritime SPOR Support Unit (MSSU) on some tailored content to be 
delivered online for all members of our project including researchers, PFAC, clinicians and the research 
staff who will be part of the RCT. The training is especially timely as we expand the group for the RCT, to 
give everyone a shared understanding of the partnership, and set the stage for engagement and 
interaction throughout the project. 
DISSEMINATION AND KNOWLEDGE TRANSLATION (KT): 
KT activities directed at the academic and stakeholder community will include presentations at scientific 
meetings, publications in academic journals and dissemination of teaching and training tools through 
patient associations, and patient and family advocacy groups. All participants will be given the option to 
receive a plain language report at the end of the study, after the RCT results have been analyzed. We 
will leverage CHILD-BRIGHT’S Knowledge Translation Program and our PFAC partners to find novel 
integrated and end-of-study KT strategies for patients, parents/caregivers and health care providers 
who participated in the RCT and beyond. After the completion of this RCT, our team will explore the 
potential to make the App more widely available.  
REFERENCES: 
1. A Guideline for Transition from Paediatric to Adult Care. Canadian Association of Paediatric Health 
Centres (CAPHC). http://www.caphc.org/. 
2. Rosen DS, Blum RW, Britto M, Sawyer SM, Siegel DM. Transition to adult health care for adolescents 
and young adults with chronic conditions: position paper of the Society for Adolescent Medicine. 
The Journal of adolescent health: official publication of the Society for Adolescent Medicine, 2003. 
33(4): p. 309-311. 
3. Tuchman L, D'Angelo L, Harris R, McCarter R. (2015). 32. Cost and Health Care Utilization for 
Adolescent and Young Adult Patients With Special Health Care Needs One Year Before and One Year 

NOTE: CTO#1666 Amendment approved by HiREB on January 9, 2023. Target sample was 264 
participants; however, we randomized 52 participants, and did not complete the full-scale RCT. Linking 
health card numbers with provincial administrative data is labour intensive and costly, and the small 
number would not provide meaningful results. As approved by ethics, we dropped the health economic 
outcome analysis (Health card number linking, Health Utilities Index (HUI), Resource Use Questionnaire 
(RUQ)) and destroy stored health card number data. 
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After Enrollment into a Randomized Health Care Transition Care Coordination Trial. Journal of 
Adolescent Health, 2015. 56(2), S17. 
4. Rozenblum R, Miller P, Pearson D, Marielli A. Patient-centered healthcare, patient engagement and 
health information technology: The perfect storm. In: Grando M, Rozenblum R, Bates D, editors. 
Information technology for patient empowerment in healthcare. Berlin: Walter de Gruyter Inc; 2015. 
p. 3–22. 
5. Grando M, Rozenblum R, Bates D, editors. Information Technology for Patient Empowerment in 
Healthcare. Berlin: Walter de Gruyter Inc; 2015. 
6. Acuna Mora M, Sparud-Lundin C, Bratt EL, Moons P. Person-centred transition programme to 
empower adolescents with congenital heart disease in the transition to adulthood: a study protocol 
for a hybrid randomised controlled trial (STEPSTONES project). BMJ Open 2017; 17; 7(4):e014593. 
doi: 10.1136/bmjopen-2016-014593. 
7. PRECIS-2 – PRagmatic Explanatory Continuum Indicator Summary. https://www.precis-2.org/.  
8. Prior M, McManus M, White P, Davidson L. Measuring the “triple aim” in transition care: a 
systematic review. Pediatrics 2014; 134(6):e1648-61. doi: 10.1542/peds.2014-1704.  
9. Sawicki GS, Lukens-Bull K, Yin X, Demars N, Huang IC, Livingood W, Reiss J, Wood D. Measuring the 
transition readiness of youth with special healthcare needs: validation of the TRAQ—transition 
readiness assessment questionnaire. J Pediatr Psychol 2011; 36(2):160-71. doi: 
10.1093/jpepsy/jsp128. Epub 2009 Dec 29. 
10. Horsmann J, Furlong W, Feeny D, Torrance G. The Health Utilities Index (HUI): concepts, 
measurement properties and applications. Health and quality of life outcomes 
2003;1:54doi:10.1186/1477-7525-1-54. 
11. Varni JW, Seid M, Rode CA. The PedsQLTM: Measurement Model for the Pediatric Quality of Life 
Inventory. Medical Care, 1999; 37(2):126-139.   
12. Ungar WJ, Tsiplova K, Millar N, Smith IM. Development of the Resource Use Questionnaire (RUQ–P) 
for families with preschool children with neurodevelopmental disorders: Validation in children with 
autism spectrum disorder. Clinical Practice in Pediatric Psychology. 2018 Jun;6(2):164. 
13. Creswell JW, Plano Clark VL. Designing and conducting mixed methods research. 2017; Thousand 
Oaks, CA: SAGE.   
14. Thorne S. Interpretive description: Qualitative research for applied practice (2nd ed.). 2016; New 
York, NY: Routledge. 
15. Bastien JM. Usability testing: a review of some methodological and technical aspects of the method. 
Int J Med Inform. 2009;79(4):e18-23. 
16. Craig N, Dieppe P, Macintyre S, Michie S, Nazareth I, Petticrew M. Developing and evaluating 
complex interventions: the new Medical Research Council guidance. BMJ. 2008, 337: a1655-
10.1136/bmj.a1655. 
17. Wittes J, Brittain E. The role of internal pilot studies in increasing the efficiency of clinical trials. Stat 
Med. 1990, 9: 65-71. 10.1002/sim.4780090113. 

NOTE: CTO#1666 Amendment approved by HiREB on January 9, 2023. Target sample was 264 
participants; however, we randomized 52 participants, and did not complete the full-scale RCT. Linking 
health card numbers with provincial administrative data is labour intensive and costly, and the small 
number would not provide meaningful results. As approved by ethics, we dropped the health economic 
outcome analysis (Health card number linking, Health Utilities Index (HUI), Resource Use Questionnaire 
(RUQ)) and destroy stored health card number data. 
Page 29 of 74                                         Protocol - READYorNotTM Brain-Based Disabilities Trial (version date: April 13, 2022) 
 
 
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NOTE: CTO#1666 Amendment approved by HiREB on January 9, 2023. Target sample was 264 
participants; however, we randomized 52 participants, and did not complete the full-scale RCT. Linking 
health card numbers with provincial administrative data is labour intensive and costly, and the small 
number would not provide meaningful results. As approved by ethics, we dropped the health economic 
outcome analysis (Health card number linking, Health Utilities Index (HUI), Resource Use Questionnaire 
(RUQ)) and destroy stored health card number data. 
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