The Autism Biomarkers Consortium for Clinical Trials: Confirmation Study

NCT ID: NCT05294705

Last Updated: 2025-09-18

Basic Information

• Recruitment Status: COMPLETED
• Total Enrollment: 565 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2022-04-11
• Study Completion Date: 2025-08-31

Brief Summary

This is a multicenter longitudinal study that aims to validate a set of measures that were previously identified as promising candidate biomarkers and/or sensitive and reliable objective measures of social function in ASD for potential use in clinical trials. The confirmation study will repeat the data collection and analysis protocols from the original ABC-CT study. This confirmation study will recruit 200 ASD and 200 TD comparison participants who are 6-11 years old, matching the overall sample size but providing a larger normative reference sample and greater statistical power for group comparisons.

Detailed Description

The goal of this consortium is to establish tools that can be used as biomarkers and/or sensitive and reliable objective assays of social function in autism spectrum disorder (ASD) clinical trials. Specifically, we aim to accelerate the development of effective treatments for social function in ASD by validating (a) outcome measures that will be sensitive and reliable assessments of response to treatment and (b) biomarkers in the domains of electroencephalography (EEG), eye-tracking (ET) and behavioral measures of social function to reduce heterogeneity of samples via stratification. The consortium will conduct a naturalistic, longitudinal study of school-aged (6-11 years) children with ASD and typical development (TD) with IQ ranging from 60-150 (ASD) and 80-150 (TD). Children will be assessed across three time points (T1: Baseline, T2: 6 weeks, T3: 24 weeks) using clinician and caregiver assessments along with a battery of conceptually related EEG and ET tasks and independent ratings of clinical status.

Conditions

Autism Spectrum Disorder

Study Design

• Observational Model Type: CASE_CONTROL
• Study Time Perspective: PROSPECTIVE

Study Groups

Autism Spectrum Disorder

During Screening Visits, the Autism Diagnostic Observational Schedule (ADOS) will be administered to confirm diagnosis. Criteria for group inclusion is: diagnosis of Autism Spectrum Disorder based on Diagnostic and Statistical Manual of Mental Disorders (DSM-5), the Autism Diagnostic Observation Schedule (ADOS-G), Brief Observation of Symptoms of Autism (BOSA), Childhood Autism Rating Scale-2(CARS-2) and the Autism Diagnostic Interview-Revised, short form (ADI-R). Diagnostic evaluations will be completed by research staff and supervised by a licensed psychologist.

No interventions assigned to this group

Typical Development

Typically Developing (TD) participants from each site will be roughly matched by age and sex to the ASD group.

No interventions assigned to this group

Eligibility Criteria

Inclusion Criteria

For All Subjects:

1. Males and Females Age 6 - 11 (less than 11 years and 6 months old at T1D1 unless all study procedures will be completed before the participant turns 12.0 and prior approval by the Principal Investigator is obtained).

2. Written parental permission, and child assents when applicable, obtained prior to any study procedures.

3. IQ 60-150 (ASD) and 80-150 (TD) as assessed by the Differential Ability Scales - 2nd Edition.

4. Participant and parent/guardian must be English speaking.

For ASD Participants (only):

1. Diagnosis of ASD based on Diagnostic and Statistical Manual of Mental Disorders (DSM-5), the Autism Diagnostic Observation Schedule (ADOS-2) or BOSA and the Autism Diagnostic Interview-Revised, short form (ADI-R). Diagnostic evaluations will be completed by research staff and supervised by a licensed psychologist.

2. If parents are biological, a minimum of the child and one parent will be required to consent to the blood draw procedure. It is preferred that the child and both biological parents participate in the blood draw procedure. The inability to obtain blood samples will not be exclusionary.

Exclusion Criteria

For All Subjects:

1. Known genetic or neurological syndrome with an established link to autism (in addition to ASD for ASD participants)

1. This does not include events in which the link to ASD is less well known/established (e.g., 16p11.2 CNVs, CHD8 mutations, Trisomy 21, 22q deletion syndrome, Dup 15q Syndrome).

2. Specific cases will be discussed with the clinical team who will make a final determination, as needed.

2. History of epilepsy or seizure disorder

a. This does not include history of simple febrile seizures or if the child is seizure free (regardless of the seizure type) for the past year.

3. Motor or sensory impairment that would interfere with the valid completion of study measures including significant hearing or vision impairment not correctable by a hearing aid or glasses/contact lenses. Children who wear bifocal or progressive lenses are not eligible.

4. Children who are taking neurological or psychiatric medications that are not stable on prescription or dose for 8 weeks prior to T1D1.

a. Medication is not exclusionary. Children taking neurological or psychiatric medications, including anti epileptics and psychopharmacological agents, must be stable on the medication and dose for 8 weeks prior to T1D1.

5. History of significant prenatal/perinatal/birth injury as defined by birth <36 weeks AND weight <2000 grams (approximately 4.5.lbs).

6. History of neonatal brain damage. (e.g., with diagnosed hypoxic or ischemic event).

7. Any other factor that the investigator feels would make assessment or measurement performance invalid.

8. Participation in the original ABC-CT study.

For ASD Participants (only):

1. Any known environmental circumstance that is likely to account for autism in the proband.

For TD Participants (only):

1. Known historical diagnosis of ASD or a sibling with ASD.

2. Criteria score in the ASD range on the ADOS/BOSA at T1

3. Active psychiatric disorder (depression, anxiety, ADHD, etc.) and/or any current treatment (medication or other treatment) for a psychiatric condition.

1. Participants will be screened using the Child/Adolescent Symptom Inventory (CASI- 5). Due to the instrument's high sensitivity and potential for false positives, any score in the clinical range will be reviewed by research staff for determination of eligibility.

• Minimum Eligible Age: 6 Years
• Maximum Eligible Age: 11 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Boston Children's Hospital

OTHER

Sponsor Role: collaborator

Duke University

OTHER

Sponsor Role: collaborator

Children's Hospital Los Angeles

OTHER

Sponsor Role: collaborator

University of Washington

OTHER

Sponsor Role: collaborator

Food and Drug Administration (FDA)

FED

Sponsor Role: collaborator

University of Alabama at Birmingham

OTHER

Sponsor Role: collaborator

University of California, Los Angeles

OTHER

Sponsor Role: collaborator

Seattle Children's Hospital

OTHER

Sponsor Role: collaborator

National Institutes of Health (NIH)

NIH

Sponsor Role: collaborator

National Institute of Mental Health (NIMH)

NIH

Sponsor Role: collaborator

Yale University

OTHER

Sponsor Role: lead

Responsible Party

James C. McPartland

Professor - Child Study Center

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

James McPartland, PhD

Role: PRINCIPAL_INVESTIGATOR

Yale University

Locations

Children's Hospital Los Angeles

Los Angeles, California, United States

Yale Child Study Center

New Haven, Connecticut, United States

Boston Children's Hospital

Boston, Massachusetts, United States

Duke

Durham, North Carolina, United States

Unviersity of Washington

Seattle, Washington, United States

Countries

United States

Other Identifiers

U19MH108206

Identifier Type: NIH

Identifier Source: secondary_id

View Link

2000031989

Identifier Type: -

Identifier Source: org_study_id