Clinical trial NCT05456139: Effectiveness Trial of Mobile ESI for Toddlers With Autism Identified by Early Screening in Primary Care
Official title: Effectiveness Trial of the Early Social Interaction (ESI) Model Using Mobile Technology for Toddlers With Autism Identified From Early Screening in Primary Care
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: NA
Conditions: Autism Spectrum Disorder
Keywords: Pediatrics
Telehealth
Treatment
Early Intervention
Community-based
Lead sponsor: Florida State University
Sponsor class: OTHER
Collaborators: name: National Institute of Mental Health (NIMH)
name: University of California, Los Angeles
name: Emory University
name: Children's Healthcare of Atlanta
name: University of Pennsylvania
name: Children's Hospital of Philadelphia
name: Weill Medical College of Cornell University
name: Rush University
name: Seattle Children's Hospital
name: Children's Hospital Medical Center, Cincinnati
First posted: 2022-07-13
Enrollment: 240
Sex: ALL
Minimum age (years): 15
Maximum age (years): 20
Standard ages: CHILD
Accepts healthy volunteers: yes
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Intervention model description: The investigators will conduct an effectiveness trial of ESI-MC to address the question of whether starting evidence-based intervention earlier leads to better outcomes than starting later. The investigators will address this question by using a modified stepped wedge design and blended implementation research to analyze data obtained with ESI-MC start at 18, 24, or 30 months. he investigators will diagnostically ascertain 240 children from a pool of 360 18-month-olds with early signs of autism. The 240 children will be randomly assigned to one of three ESI-MC timing groups. The investigators will measure child active engagement and social communication change every 6 months as the primary outcome variables. Outcome measures of developmental level, autism symptoms, and adaptive behavior will be examined to measure differential treatment effects.
Primary purpose: HEALTH_SERVICES_RESEARCH

Brief summary: The goal of this collaborative R01 is to demonstrate the therapeutic value and community-wide implementability of an early intervention (EI) platform for toddlers with autism spectrum disorder (ASD) that is completely virtual, from recruitment through intervention. This platform-Early Social Interaction Mobile Coaching (ESI-MC) deploys individual telehealth sessions with coaching and feedback to help families embed intervention in everyday activities. Specifically, the investigators will conduct an effectiveness trial of ESI-MC to address the important question of whether starting evidence-based intervention earlier leads to better outcomes than starting later. The investigators will address this question by using a modified stepped wedge design and blended implementation research to analyze data obtained with ESI-MC start at 18, 24, or 30 months. The investigators will diagnostically ascertain 240 children from a pool of 360 18-month-olds with early signs of autism, 30 in each of 8 US regions (Central and SW Florida; Atlanta, GA; suburbs of Philadelphia, PA; New York City, NY; Cincinnati, OH; Chicago, IL; Seattle, WA; and Los Angeles, CA). Research participants will be recruited using a new virtual platform-My Baby Navigator-linking a new surveillance and screening tool, an app to upload video-recorded home observations and telehealth intervention sessions, and a package of educational resources. The 240 children will be randomly assigned to one of three ESI-MC timing groups. ESI-MC will be delivered by community-based early intervention providers (EIPs) currently working within the the early intervention system in the recruitment regions. The investigators will measure child active engagement and social communication change every 6 months as the primary outcome variables. Outcome measures of developmental level, autism symptoms, and adaptive behavior will be examined to measure differential treatment effects. Maximizing the use of mobile technology, ESI-MC offers the prospect of a community-viable, scalable and sustainable treatment to improve EI services for toddlers with ASD, particularly among minority and low-resource communities.

Detailed description: This is a collaborative R01 application (RFA-MH-18-700) with Dr. Amy Wetherby as PI at Florida State University (FSU), Drs. Ami Klin and Jennifer Stapel-Wax as Co-PIs at Emory University (EU), and Dr. Catherine Lord as Co-PI at the University of California, Los Angeles (UCLA). This effectiveness trial will address the important question of whether starting evidence-based intervention earlier leads to better outcomes than starting later. The investigators will compare the timing of Early Social Interaction Mobile Coaching (ESI-MC) -individual telehealth sessions with coaching and feedback to help families embed intervention in everyday activities, starting at 18, 24, or 30 months, with a modified stepped wedge design and blended implementation research. This effectiveness trial will be conducted using a virtual platform from recruitment through intervention implementation to reach a national community-based sample of toddlers with autism spectrum disorder (ASD).

Overview of Effectiveness Trial. The investigators will recruit 360 children with early signs of autism at 18 months using a new virtual platform-My Baby Navigator-linking surveillance and screening tools, an app for uploading video-recorded home observations and mobile intervention sessions, and a package of educational resources. From the pool of 360 children with early signs, the investigators will identify 240 children with a clinical best estimate diagnosis of ASD, 60 in each of four regions (Northeast, Southeast, Midwest, West Coast), and randomly assign parent-child dyads to one of three ESI-MC timing groups (18, 24, 30) to address the following research aims:

Aim 1. Compare the effectiveness of ESI-MC implemented for 6 months on proximal outcome measures of child active engagement, child social communication change, parent transactional supports, and parent evidence-based strategy use (1A) with Treatment-as-Usual (TAU) at 24 and 30 months and (1B) across treatment timing groups initiated at 18, 24, or 30 months. Hypotheses 1) Parent-child dyads in ESI-MC will (1A) show significantly better child and parent outcomes compared to TAU and (1B) those starting ESI-MC earlier will show successively better child outcomes than those starting later.

Aim 2. Examine (2A) change in parent transactional supports and evidence-based strategy use as the mechanism for change in proximal child outcomes and (2B) individual child and family characteristics that moderate response to treatment.

Aim 3. Compare the effectiveness of ESI-MC implemented for 6 months on secondary outcome measures of child developmental level, autism symptoms, and adaptive behavior (3A) with TAU at 24 and 30 months and (3B) across treatment timing groups initiated at 18, 24, or 30 months.

Aim 4. Explore outcomes at 36 months, individual patterns of change from 18-36 months, and predictors of change across treatment timing groups by estimating child growth trajectories.

Aim 5. Examine barriers and promotive factors impacting widespread dissemination, implementation and sustainability across racial, socioeconomic and geographic lines, including parental uptake, engagement and satisfaction during and after intervention, treatment fidelity, reduction of disparities in access to services and in outcomes, and cost-benefit analyses.

The investigators expect ESI-MC will result in better outcomes than TAU and that initiating treatment earlier leads to better outcomes. This effectiveness trial of ESI-MC will address concerns raised by the US Preventive Services Task Force (USPSTF), advance knowledge for community-uptake, and provide needed evidence to support early universal screening and referral at 18-24 months. Documenting the therapeutic value of ESI-MC to teach parents in natural environments will offer a community-viable and affordable treatment to improve services for toddlers with ASD, which in light of ongoing COVID-19 pandemic restrictions, places a premium on remote tools and methods. The recent spotlight on healthcare disparities and civil injustices underscores the urgency with which inequalities faced by minority and low-resource communities need to be vigorously addressed. By maximizing the use of mobile technology and community-based care teams, this treatment will be ready for immediate, rapid, scalable, and sustainable deployment across the US that can lead to transformative changes in healthcare services for toddlers with autism and their families.

Overview of Collaborative Study. A collaborative study is necessary to accommodate a large, representative community-based sample of very young children with ASD with geographic spread for an effectiveness trial. Recruitment and implementation across 8 sites in 4 national regions will provide racial/ethnic and socioeconomic diversity, ample urban/rural areas, and different health service systems. The research team includes the complementary expertise needed and has history of collaboration. The PIs will establish a means of coordinating communication and shared decision-making before the start of research. FSU will provide the virtual platform for recruitment and random assignment, train the ESI mobile coaches, monitor intervention fidelity, and oversee data management and statistical analysis. UCLA will oversee reliability of autism symptom measures and calibration of diagnosis. Emory will oversee documentation and dissemination of research-based solutions to address health disparities. Each site will conduct diagnostic evaluations, ensure reliability on measures, and use manualized procedures conducted with fidelity to ensure scientific integrity for pooling data.

Eligibility criteria: Inclusion Criteria:

* Parent(s) complete the SoCo CheckUp by the time the child is 18 months of age and results will indicate positive risk for autism
* Parent(s) submit a home observation video of the parent engaging their child for an hour in 6 everyday activities by the time the child is 18 months of age
* Results of the SORF rated from the home observation indicate risk for autism
* The child receives a clinical best estimate diagnosis of autism spectrum disorder based on a diagnostic and developmental evaluation by 18 months (+/-2 months) of age, which includes completion of the ADOS
* The child will have sufficient trunk stability and control of arm and hand movements to hold objects and make simple gestures (give, show, reach)
* Parent(s) agree to be in the intervention study by the time the child is 18 months (+/- 2 months) of age
* The primary language at home is English or Spanish (the SoCo CheckUp and all Baby Navigator tools are available in these languages)
* The family lives within 45 miles of one of the 8 recruitment sites.

Exclusion Criteria:

* Child does not meet diagnostic criteria for autism
* Family does not agree to be video recorded
* Family does not agree to receive the intervention via mobile technology
* Enrollment in EI at the time of screening
* A sibling is already enrolled in the trial
* Children with significant motor, visual, or hearing impairments
* Children born with very low birth weight or significant medical complications (e.g., seizure disorder)

Primary outcomes:
measure: Parent-Child Interaction Change at Home
timeFrame: Baseline and 3, 6, 9, 12, 15, and 18 months thereafter
measure: Social Communication Change
timeFrame: Baseline and 6, 12, and 18 months thereafter
measure: Intervention Strategy Use
timeFrame: Baseline and 6, 12, and 18 months thereafter
measure: Observation of Diagnostic Features of Autism Change
timeFrame: Baseline and 3, 6, 9, 12, 15, and 18 months thereafter
Secondary outcomes:
measure: Autism Symptoms
timeFrame: Baseline and 6, 12, and 18 months thereafter
measure: Developmental Level
timeFrame: Baseline and 6, 12, and 18 months thereafter
measure: Adaptive Behavior
timeFrame: Baseline and 6, 12, and 18 months thereafter
measure: Brief Observation of Autism Symptoms
timeFrame: Baseline and 6, 12, and 18 months thereafter
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: University of California Los Angeles
state: California
city: Tallahassee
country: United States
facility: Florida State University Autism Institute
state: Florida
city: Atlanta
country: United States
facility: Emory University
state: Georgia
city: Chicago
country: United States
facility: Rush University
state: Illinois
city: White Plains
country: United States
facility: Weill Cornell Medical College
state: New York
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia
state: Pennsylvania
city: Seattle
country: United States
facility: Seattle Children's Hospital
state: Washington
Study officials:
affiliation: Florida State University
name: Amy Wetherby, PhD
role: PRINCIPAL_INVESTIGATOR
References: