Validation of biomarkers for social function in autism

The Autism Biomarkers Consortium for Clinical Trials

This study is trying to find reliable signs that can help understand social skills in children with autism and see how they compare to typically developing kids.

Quick facts

• Study type: Observational
• Enrollment: 400 (estimated)
• Ages: 6 Years to 11 Years
• Sex: All
• Sponsor: Yale University (Academic / other)
• Locations: 5 sites (Los Angeles, California and 4 other locations)
• Trial ID: NCT05294705 on ClinicalTrials.gov

What this trial studies

This multicenter longitudinal study aims to validate promising candidate biomarkers and objective measures of social function in children with Autism Spectrum Disorder (ASD). The study will recruit 200 participants with ASD and 200 typically developing children aged 6-11 years, assessing them across three time points to gather data on their social functioning. By repeating data collection and analysis protocols from a previous study, the goal is to establish reliable outcome measures and biomarkers that can enhance clinical trials for ASD treatments.

Who should consider this trial

Good fit: Ideal candidates are children aged 6-11 years with a diagnosis of Autism Spectrum Disorder or typically developing peers.

Not a fit: Patients outside the age range of 6-11 years or those with IQs below the specified thresholds may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could lead to more effective treatments for improving social function in children with autism.

How similar studies have performed: Other studies have shown promise in validating biomarkers for autism, making this approach both relevant and potentially impactful.

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.

Where this trial is running

Los Angeles, California and 4 other locations

• Children's Hospital Los Angeles — Los Angeles, California, United States (Recruiting)
• Yale Child Study Center — New Haven, Connecticut, United States (Recruiting)
• Boston Children's Hospital — Boston, Massachusetts, United States (Recruiting)
• Duke — Durham, North Carolina, United States (Recruiting)
• Unviersity of Washington — Seattle, Washington, United States (Recruiting)

Study contacts

• Principal investigator: James McPartland, PhD — Yale University
• Study coordinator: James McPartland, PhD

Conditions Autism Spectrum Disorder; Biomarkers