Can a Novel Telemedicine Tool Reduce Disparities Related to the Identification of Preschool
Phase: NA
Conditions: Autism Spectrum Disorder
Sponsor: Vanderbilt University Medical Center

 
 
 
 
 
 
 
 
Can a novel telemedicine tool reduce disparities related to the 
identification of preschool children with autism?  
 
NCT05373173 
 
Protocol/Statistical Analysis Plan adapted from IRB application approved on 
4/20/2022 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 

 

Background 
 
With the rising prevalence of Autism Spectrum Disorder (ASD), accurate identification 
and meaningful engagement of children and families in evidence-based services 
represents a pressing public health and clinical care challenge. Considerable emphasis 
has been placed on strategies to boost early ASD diagnosis in toddlers. However, early 
identification is hindered in part by vast phenotypic heterogeneity, wherein subtle ASD 
symptoms may not be readily identified via screening, surveillance, or parent concerns 
during the toddler years. Specifically, children without significant language delays, 
cognitive delays, or externalizing behaviors may not be flagged as for ASD concerns until 
the preschool years or later. In addition, pervasive service system limits such as a lack 
of trained providers and significant wait times for evaluation can delay definitive 
diagnosis for years. Despite advances in screening and awareness, the average age of 
diagnosis in many locations in the US remains later than four years of age. This is 
especially true for children from traditionally underserved communities (e.g., families in 
poverty, rural communities, racial/ethnic minorities, linguistically diverse families), who 
are much less likely to be diagnosed at young ages. These delays contribute to 
substantial family stress and restrict access to ASD intervention services critical for 
optimal short-term and lifespan functioning. 
 
Ideally, universal ASD screening in toddlerhood would identify most children at-risk for 
ASD and then facilitate prompt diagnostic evaluation by a multi-disciplinary team of 
specialists. In reality, such diagnostic teams, or individual experts, are not readily 
available in most areas of the U.S. The standard of care in many community pediatric 
settings is to perform developmental surveillance, screen for risk, and then refer children 
to tertiary diagnostic centers with lengthy waitlists; meanwhile, without diagnostic 
confirmation, children may receive low levels of non-specific intervention or preschool 
services, or fail to engage with intervention and school services at all. Data suggest that 
the wait for ASD evaluation services dramatically increases as children age, with 
extended waits (i.e. over a year) common in the preschool period. Delays in diagnosis 
may contribute to more pronounced impairments and challenges accessing services (i.e. 
diagnostic delay associated more limited private/school-based intervention, higher rates 
of psychotropic medication use). Unfortunately, even when an appointment becomes 
available, multi-stressed, rural, or traditionally underserved families may not be able to 
make it to a tertiary care center.  Powerful recent work suggests that addressing care 
disparities and systemic structural racism within models of ASD identification will require 
novel identification tools and processes that 1) include the preschool age span and 2)  
specifically target underserved groups at risk for tremendous diagnostic delays. For 
example, a recent study investigating a large cohort of African-American children in the 
U.S. found an average 3-year delay between first parental developmental concern and 
ASD diagnosis; half of parents reported seeing multiple providers before receiving an 
ASD diagnosis, and 31% reporting a lack of available professionals as a major 
contribution to this delay. 
 
Study Purpose and Description  
 
Innovative telemedicine practices could address many existing traditional barriers to ASD 
identification. Telemedicine has the potential to put linguistically and culturally 

competent clinical expert virtual providers directly into communities during a critical 
window in which many vulnerable families may approach, or re-approach, care systems 
about developmental concerns that become more prominent with age. In the current 
proposal, we will develop and evaluate the potential clinical and familial value of a 
telemedicine-based ASD assessment tool, the TAP-Preschool (TELE-ASD-PEDS-
Preschool), designed to overcome traditional barriers to diagnosis and service access in 
underserved preschool populations that may not be readily identified or engaged by 
early screening and intervention systems.  
 
A growing body of literature supports the use of telemedicine-based approaches to ASD 
assessment and intervention. This includes remote activities to assess infant social 
communication skills in the first year of life, coaching parents through administration of 
gold standard diagnostic tests, and provider coding and analysis of interactions that are 
video-recorded by caregivers. Results reflect high levels of caregiver and provider 
satisfaction and satisfactory agreement with traditional in-person evaluations, illustrating 
both the promise and feasibility of tele-assessment. However, most existing approaches 
are limited by a focus on screening rather than diagnosis, protocols requiring specific 
materials, or asynchronous analysis of submitted videos, which require resources that 
preclude broader use. Moreover, in current form, these tools are not designed or 
intended to yield a quantitative formal assessment of core ASD symptoms to support 
diagnostic decision-making. Further, although providers are increasingly exploring 
telemedicine approaches to ASD assessment in the context of the COVID-19 pandemic, 
there is limited published work to date on the use of real-time, caregiver-led ASD 
assessment in home settings with diverse populations. Our own work has focused 
almost exclusively on toddlers in this regard. Ultimately, very few viable tools with 
validated psychometric properties are available for use in current models of telehealth 
assessment and care.  
 
Study Aims 
 
In Specific Aim 1, we will generate optimally performing models and key behavioral 
targets for tool development. We will do this by applying machine learning models and 
the principles of feature engineering to a phenotypically rich clinical research data set of 
preschoolers with ASD and other developmental concerns (N=914: 594 ASD and 320 
Non-ASD). 
 
In Specific Aim 2, we will engage in a rigorous adaptation and translation approach to 
optimize design of the TAP-Preschool. We will include leading ASD assessment experts, 
providers dedicated to caring for underserved populations, and parents of preschool 
children with ASD from underserved racial/ethnic and linguistic groups. We will lead 
these groups in a design process in order to yield a set of interactive and play-based, 
parent-coached tele-assessment activities designed to 1) elicit observations tied to key 
computational features, 2) be deployed within a 30-minute timeframe, 3) employ 
inexpensive materials found in most homes, and 4) use accessible assessment 
instructions for real-time coaching of parents. 
 
We will recruit 30 parent/child dyads (children ages 36 to 72 months) with existing 
diagnoses of ASD (n = 20) or other developmental concerns (n = 10) from our clinical 

research database. Each participant will have recent data available from comprehensive 
evaluation tools (ADOS-2, cognitive functioning, adaptive skills). Children with and 
without ASD are included to provide information about TAP-Preschool usage across 
diverse phenotypic profiles. The sample size of 30 is deemed adequate for gathering 
detection of feasibility/acceptability issues and key feedback regarding measure 
modification for further validation. 
 
Inclusion/Exclusion Criteria: Aim 2 
Inclusion 
- English/Spanish Speaking families 
- Children 36-72 months of age 
- access to a device capable of supporting Zoom 
- already has participated in a diagnostic evaluation 
 
Exclusion 
- severe sensorimotor impairments 
 
Specific Aim 3: Initial deployment, formative evaluation, and refinement of 
the TAP-Preschool: 
 
We will deploy the preliminary TAP-Preschool with a small sample (n = 30) to assess 
acceptability/feasibility, potential clinical value for remote observation, and challenges 
that warrant revision. We will then use this data to modify the TAP-Preschool and deploy 
the refined tool with a new sample of clinically referred children (n=120). We will 
evaluate its ability to facilitate accurate telemedicine supported diagnostic decision-
making.  
 
We will recruit a novel sample (n = 120)  parent/child dyads (children 36 - 72 months of 
age) referred for evaluation of ASD or developmental delays. These children and a 
primary caregiver will participate in a home-based tele-assessment session and a 
subsequent blinded in-person evaluation. We will again include English/Spanish 
speaking families with access to a device that will support Zoom.  
 
Inclusion/Exclusion Criteria: Aim 3 
 Inclusion 
- English/Spanish Speaking families 
- Children 36-72 months of age 
- access to a device capable of supporting Zoom 
- has not participated in a diagnostic evaluation 
 
-Exclusion 
- severe sensorimotor impairments 
 
 
Study Procedures 
 
Initial deployment, evaluation, and refinement of the TAP-Preschool:  
Consenting families will be scheduled for a single tele-assessment session with a 
consented licensed psychologist (“remote clinician”) from our clinical research center 

(n=10). This clinician will coach parents through the TAP-Preschool procedures. 
Although families will receive standard, basic support regarding tele-assessment 
procedures, they will not receive extensive training on the TAP-P prior to the session in 
order to mimic real-world use. All remote clinicians will be experts in ASD with training 
on the original TAP and ADOS-2 research reliability. Each clinician will participate in 3 
sessions (2 children with ASD, 1 other developmental concerns). Clinicians will be 
blinded to child diagnostic status. Participating telemedicine clinicians in this aim will not 
be aware of the ratio (2:1 ASD vs. other DD) or recruitment status prior to evaluation 
ratings. As children will have existing diagnoses, no diagnostic feedback will be 
provided.  
 
We will collect user data (caregiver, clinicians) on satisfaction, ease of implementation, 
and diagnostic certainty (clinicians only). Based on this feedback, we will modify the 
preliminary TAP-Preschool instructions and procedures as needed. To systematically 
measure acceptability and feasibility of use we will utilize an adapted Acceptability, 
Likely Effectiveness, Feasibility, and Appropriateness Questionnaire (ALFA-Q).54 The 
ALFA-Q asks caregivers and clinicians to use a 5-point Likert scale to rate the instrument 
acceptability, effectiveness, feasibility, and appropriateness for ASD decision making. We 
will also solicit free-form input. Team leads will briefly interview each clinician and 
caregiver about their experiences. After each telemedicine evaluation, participating 
clinicians will view data from previous comprehensive evaluations. Clinicians will provide 
concrete task evaluation data regarding whether they were able to elicit such behaviors 
or information in the telemedicine evaluation process. It is important to note that 
diagnostic agreement rankings will include dichotomous (agree/disagree) and 
uncertainty data (Likert ratings). As in our previous preliminary feasibility/effectiveness 
trial, we will target >60% of providers agreeing with existing risk classification for ASD, 
<10% of ASD inaccurately identified with ASD, and >50% non ASD DD with certainty as 
key benchmarks for understanding potential meaningful clinical value. Based on this 
data, our investigative team will collaborate with our clinical design team to suggest 
instrument modifications. 
 
The tele-assessment will take <45 minutes to complete.  
 
The parent forms will take approximately 20 minutes to complete.  
 
Assessment on psychometrics, clinical, and familial value of the TAP-
Preschool (Novel Sample): 
All consented families will complete an initial home-based tele-assessment visit via Zoom 
that includes the TAP-Preschool and a brief symptom-focused developmental interview 
with a consented clinician. The initial tele-assessment session includes interviewing and 
developmental assessment to mimic real-world use of TAP-Preschool. The visit is 
designed to take less than 90 minutes, with TAP-Preschool evaluation lasting <30 
minutes. Although we will not limit length, we will time all sessions. After the session, 
the examiner will record the clinical diagnosis issued (ASD, other developmental 
concerns, or typical development) and complete two diagnostic certainty ratings, one 
dichotomous, the other continuous. All initial TAP-Preschool administrations will be 
recorded via Zoom, and 50% of administrations (randomly selected) will be co-scored by 
a blinded examiner, unaware of prior assessment results and diagnostic decision, to 

evaluate inter-rater reliability. Within 7 days of the remote assessment, families will 
participate in an in-person diagnostic assessment including common comprehensive 
measures of ASD, cognitive skills, and adaptive behavior. We will randomly select 25% 
of families to participate in a remote home-based re-test with TAP-Preschool two weeks 
following initial administration. 
 
Tele-assessment measures: Prior to the appointment, caregivers will complete a 
Demographics Questionnaire (i.e., age, gender race/ethnicity, zip code, parent 
education) and a Medical History Form through a secure HIPAA-compliant web portal 
(REDCap). To reduce technology-related disruptions, research staff will talk to families 
before the visit about connectivity settings and recommended materials. 
 
These will take approximately 20 minutes to complete.  
 
In a single tele-assessment session modeled after our previous work, licensed clinicians 
will administer the TAP-Preschool, the Developmental Profile, 4th edition (DP-4), and a 
DSM-5 ASD interview. The DP-4 is a caregiver interview (birth – 21 years) that identifies 
developmental strengths and weaknesses in five core areas. The DSM-5 interview 
provides symptom-focused questions with developmental anchors pertinent to this age 
range. Immediately after the session, the clinician will complete a DSM-5 checklist 
indicating symptoms present and clinical diagnosis issued (ASD, other developmental 
concerns, 
or 
typical 
development). 
This 
form 
also 
contains 
dichotomous 
(certain/uncertain) and continuous (10-point Likert scale) certainty ratings. Clinicians will 
describe their satisfaction with the tele-assessment process (CSQ: Clinician Satisfaction 
Questionnaire).   
 
The initial tele-assessment appointment with the psychologist will last approximately 90 
minutes. 
 
Blinded in-person assessment measures: The in-person examiner will be blinded to the 
tele-assessment diagnosis until after the in-person assessment. The diagnostic 
confirmation battery will include the Autism Diagnostic Observation Schedule, Second 
Edition (ADOS-2) (one module is chosen based on language ability), a cognitive measure 
(Mullen Scales of Early Learning or Differential Ability Scales 2nd Edition, the Vineland 
Adaptive Behavior Scales, Third Edition, and a DSM-5 ASD Interview. Immediately after 
the in-person session, the examiner will also complete a DSM-5 checklist and certainty 
ratings. 
 
In-person appointments will last approximately 2-3 hours and will consist of traditional 
diagnostic evaluations for autism spectrum disorder. 
 
Parent measures: Parents will complete the Parent Perceptions of Telehealth (PPT) and 
the Parent Service Satisfaction (PSS) surveys, used in our previous work, to assess 
perceptions of tele-assessment procedures after the telemedicine and in-person 
evaluations. 
 
These parent forms will take approximately 10 minutes to complete.   
 

Reporting of Adverse Events or Unanticipated Problems involving Risk to 
Participants or Others 
 
All members of the research team will be trained and instructed to immediately report 
any adverse events to the PI. Adverse events will be reported to the IRB within seven 
days. The PIs, in communication with the study teams, will distinguish serious adverse 
events (SAE) from non-serious adverse events (AE). The PIs and Co-Is are licensed 
clinical psychologists with experience establishing adverse event and safety protocols in 
research projects. All project staff will have training in identifying potential adverse 
events and reporting procedures.  
 
Should any study participant express concerns about the study or their participation or 
appear distressed during any study activities, the witnessing research team member will 
bring the matter to the attention of the PI, or designate at that time, to determine the 
appropriate course of action, which may include contacting local authorities in the case 
of imminent danger during a telemedicine appointment (suicidality, child neglect or 
abuse). Although not anticipated, serious adverse events would be documented and 
discussed with the IRB as soon as possible and reported to the IRB and NIH project 
officer within 48 hours. 
 
Study Withdrawal/Discontinuation 
 
Participants can withdraw from the study at any time. If a participant withdraws from 
the study, research team members will offer other options for having the child evaluated 
for ASD. If a clinician withdraws from the study they will not be asked to participate 
again in the current protocol.  
 
Study participation may also be discontinued in the event of repeated technology 
failures during tele-assessment. Should technology fail (e.g., lost connection that cannot 
be re-established), we will contact families by phone to schedule a second attempt. If 
this is also unsuccessful, will document this as part of our ongoing data collection 
processes while also routing families to appropriate clinical referral pathways.   
 
If a child is in distress (i.e., crying, falling to the floor, shouting) then research staff will 
take a break and/or consult parents for input/help on how to best soothe their child. 
The parent will always be given the option to take a break or end the evaluation if they 
think their child is too distressed to continue.  
If a clinician thinks that the child appears to be too distressed to complete the 
evaluation, opportunities will be given to reschedule the assessment. If the parent 
declines, the clinician will note that the child was unable to complete the assessment 
and only data obtained will be included in the study while also routing families to 
appropriate clinical referral pathways.  
 
Statistical Design and Power 
 
In Specific Aim 2, we will collect user data (caregiver, clinicians) on satisfaction, ease of 
implementation, and diagnostic certainty (clinicians only). We will examine these data 

descriptively (frequencies for satisfaction items; mean/SD for diagnostic certainty) and 
use them to inform modifications to TAP-P procedures.  
 
In Specific Aim 3, we will benchmark acceptability/usability and preliminary clinical value 
and then perform rigorous evaluation of the TAP-Preschool relative to in-person 
evaluation. Our sample size has been determined to power primary analyses around 
estimates of accuracy, specific psychometric properties, as well as family experience. We 
will use receiver operating characteristic (ROC) curve analyses to determine a cut score 
for the optimal scoring method and evaluate characteristics utilizing the entire sample. 
We will examine agreements and corresponding sensitivity, specificity, and positive and 
negative predictive value.  After estimated attrition of approximately 10%, 108 subjects 
(projected 65 ASD; 43 non-ASD) will be available for analysis.  PPV and sensitivity will 
have a margin of error no larger than +/- 9.7%, and NPV and specificity will have a 
margin of error no larger than +/- 12.0%. Margin of error (half the width of a 95% 
confidence interval) calculations are based on preliminary data from prior data from tele-
assessment of ASD and conservatively assuming 80% PPV and 80% NPV. For inter-rater 
and test-retest reliability, we will be able to measure percent agreement with a margin 
of error no larger than +/- 7.5% and Cohen’s kappa within +/- 0.12. For estimating 
associations among factors potentially related to clinician diagnostic certainty and 
caregiver satisfaction, we will have 80% power to detect odds ratios larger than 1.37. All 
calculations use a two-sided significance level of 0.05 (See Statistical Design and Power 
for more detail).