Neuroimaging Predictors of Improvement to Pivotal Response Treatment (PRT) in Young Children With Autism (PRT-I)

Autism spectrum disorder (ASD) is a very heterogeneous disorder with limited empirically validated behavioral and biological interventions. The goal of this pilot investigation is to apply a biologically-based approach to identify predictors of treatment response in children with ASD who are receiving Pivotal Response Treatment (PRT), an evidence-based behavioral intervention. Specifically, the investigators propose to identify neuroimaging biomarkers of treatment response to a PRT program (PRT-P) targeting language deficits in young children with ASD who will be randomized to either PRT-P or to a delayed treatment group (DTG).

Study Overview

• Status: Recruiting
• Conditions: Autism Spectrum Disorder; Autism
• Intervention / Treatment: Behavioral: Pivotal Response Treatment Program (PRT-P)

• Study Type: Interventional
• Enrollment (Estimated): 36
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Estefania Millan, MA; Phone Number: (650) 736-1235; Email: mmillan2@stanford.edu
• Study Contact Backup: Name: John Hegarty, PhD; Phone Number: (650) 736-1235; Email: hegartyj@stanford.edu

Study Locations

• United States
  • California
    • Stanford, California, United States, 94305-5719
      • Recruiting
      • Stanford University
      • Principal Investigator: Antonio Y. Hardan, MD
      • Contact: Estefania Millan, MA; Phone Number: 650-736-1235; Email: mmillan2@stanford.edu
      • Contact: Robin Libove, BS; Phone Number: (650) 736-1235; Email: rlibove@stanford.edu

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 7 months to 2 years (Child)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Diagnosis of Autism Spectrum Spectrum Disorder (ASD) based on clinical interview and Diagnostic and Statistical Manual, 5th edition (DSM-5) and confirmed using the Autism Diagnostic Interview Revised (ADI-R) and the Autism Diagnostic Observation Schedule (ADOS) and/or Brief Observation of Symptoms of Autism (BOSA) and/or Childhood Autism Rating Scale- Second Edition (CARS-2).
• Outpatients between 2.0 and 4.11 years of age of either gender,
• Children of all cognitive levels will be included as long as they are able to participate in the testing procedures to the extent that valid standard scores can be obtained
• Language delay as measured by the Preschool Language Scale, 5th Edition (PLS-5) [at least 1 standard deviation behind for children age 2 and 3 years; and 2 standard deviations behind for children age 4],
• Stable psychotropic medication(s) or biomedical intervention(s) for at least 1 month prior to baseline measurements with no anticipated changes during study participation,
• Stable treatment [Applied Behavior Analysis (ABA), Floortime, or other interventions], speech therapy, and school placement for at least 1 month prior to baseline measurements with no expected changes during study participation,
• No more than 60 minutes of 1:1 speech therapy per week,
• The child's exposure to the English language must be sufficient that administration of standardized tests in English is appropriate for measuring progress,
• The availability of at least one parent who can consistently participate in the training sessions and related activities, and
• Successful completion of baseline brain scan.

Exclusion Criteria:

• Current or life-time diagnosis of severe psychiatric disorder (e.g., bipolar disorder),
• Genetic abnormality (e.g., Fragile X)
• Presence of active medical problem (e.g., unstable seizure disorder),
• Receiving more than 15 hours of in home 1:1 Applied Behavior Analysis (ABA) per week
• Magnetic Resonance (MR) contraindication (e.g., the presence of ferrous metal), or
• Previous adequate Pivotal Response Treatment (PRT) trial.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Single

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Pivotal Response Treatment Program (PRT-P); The Pivotal Response Treatment Program (PRT-P) will consist of 3 parent-only sessions (60-90 min) and 13 family sessions with the parent and child (60-90 min). These 16 sessions are once per week over a 16 week period.	Behavioral: Pivotal Response Treatment Program (PRT-P); The Pivotal Response Treatment Program (PRT-P) will consist of 3 parent-only sessions (60-90 min) and 13 family sessions with the parent and child (60-90 min). These 16 sessions are once per week over a 16 week period.; Other Names: Pivotal Response Treatment
No Intervention: Delayed Treatment Group (DTG); Child continues stable treatments as usual in the community.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Change in Number of Child Utterances During a Structured Lab Observation (SLO)	Baseline, 16 Weeks

Secondary Outcome Measures

Outcome Measure	Time Frame
Change on MacArthur-Bates Communication Development Inventory (CDI)	Baseline, 16 Weeks

Other Outcome Measures

Outcome Measure	Time Frame
Change on Preschool Language Scale, 5th Edition (PLS-5)	Baseline, 16 Weeks
Change on Mullen Scales of Early Learning	Baseline, 16 Weeks
Change on Vineland Adaptive Behaviors Scales, 3rd Edition	Baseline, 16 Weeks
Change on Clinical Global Impressions Scale (CGI)	Baseline, 16 Weeks
Change on the Brief Observation of Social Communication Change (BOSCC) direct child observation assessment	Baseline, 16 Weeks
Change on Parent Stress Index (PSI)	Baseline, 16 Weeks
Change on Family Empowerment Scale (FES)	Baseline, 16 Weeks
Change on General Self Efficacy Scale (GSES)	Baseline, 16 Weeks

Collaborators and Investigators

• Sponsor: Stanford University
• Collaborators: National Institute on Deafness and Other Communication Disorders (NIDCD)
• Investigators: Principal Investigator: Antonio Hardan, MD, Stanford University

Study record dates

Study Major Dates

• Study Start (Actual): December 7, 2018
• Primary Completion (Estimated): November 30, 2024
• Study Completion (Estimated): November 30, 2024

Study Registration Dates

• First Submitted: June 13, 2018
• First Submitted That Met QC Criteria: June 28, 2018
• First Posted (Actual): July 11, 2018

Study Record Updates

• Last Update Posted (Estimated): December 14, 2023
• Last Update Submitted That Met QC Criteria: December 13, 2023
• Last Verified: December 1, 2023

More Information

Terms related to this study

• Keywords: Imaging; Pivotal Response Treatment
• Additional Relevant MeSH Terms: Mental Disorders; Neurodevelopmental Disorders; Child Development Disorders, Pervasive; Autistic Disorder; Autism Spectrum Disorder
• Other Study ID Numbers: IRB-46131; 1R21DC016089-01A1 (U.S. NIH Grant/Contract)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: We will submit de-identified clinical data to the NIMH Data Archive (NDA) data repository.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No