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Condition Guide

New Treatments & Clinical Trials for Autism Spectrum Disorder

Last updated September 2026Data from ClinicalTrials.gov600 active trials
← Browse all Autism Spectrum Disorder trials

Autism spectrum disorder affects about 1 in 36 children in the United States. There are no medications that treat the core features of autism — social communication differences and repetitive behaviors — though some drugs help with related symptoms like anxiety or irritability. Most interventions focus on behavioral therapies, educational support, and accommodations.

What's actually going on in research

Trials are testing drugs that target specific brain pathways involved in autism, including vasopressin and oxytocin for social communication. Researchers are also studying cannabis-derived compounds for irritability and aggression, anti-inflammatory approaches for children with immune activation, and technologies that help with communication. Many studies now include autistic adults as partners in designing research questions.

Vasopressin pathway drugs

Drugs that affect vasopressin, a brain chemical involved in social behavior, are being tested to see if they improve social communication in autistic children. Early trials showed mixed results, but research continues with different doses and age groups.

Cannabinoid compounds

Cannabidiol and related compounds are being tested for irritability, aggression, and anxiety in autism. Some small studies suggest they may help, but larger trials are needed to confirm safety and effectiveness.

Targeted treatments by subtype

Researchers are testing whether specific genetic or biological subtypes of autism respond to tailored treatments. This includes trials for people with fragile X syndrome, tuberous sclerosis, and other conditions that often occur with autism.

What to know before you search

Eligibility typically depends on age, autism severity, presence of specific symptoms like irritability, and sometimes genetic testing results or biomarker status.

What types of trials are currently open

  • Social communication trialsTesting medications or technologies aimed at improving social interaction, eye contact, and communication skills in autistic children and adults.
  • Behavioral symptom trialsStudies of drugs for irritability, aggression, hyperactivity, or repetitive behaviors that can interfere with daily life.
  • Anxiety and mood trialsTesting treatments for anxiety, depression, and mood symptoms, which are common in autistic people.
  • Biomarker studiesResearch looking for biological markers that might predict treatment response or identify subtypes of autism.
  • Technology and device studiesTesting apps, wearable devices, and other technologies that support communication, emotion recognition, or daily living skills.

Recently added Autism Spectrum Disorder trials

RecruitingInterventional study

Try a behavioral activation program designed for autistic teens with depression

Autism spectrum disorder is characterized by differences in social communication/interaction and the presence of restricted/repetitive behaviors, with significant heterogeneity in terms of autistic features, language ability, and cognitive characteristics. About 2-3% of individuals in the United States are autistic, and most autistic individuals have co-occurring mental health conditions. Autistic people are 3-4 times more likely to experience depression than non-autistic people. Of the emerging depression treatment research with autistic people, none to date has focused on developing and testing a depression treatment for autistic youth with below average cognitive abilities - a group which comprises over 50% of the autistic community. Therefore, this study seeks to evaluate the feasibility (Aim 1), acceptability (Aim 2), and preliminary treatment effects (Aim 3) of an extension of Behavioral Activation for Depression in Autistic Adolescents (BA-A), adapted to meet the needs of cognitively lower functioning autistic youth.

Stony Brook, New York, United States
RecruitingObservational study

Complete fitness tests to help researchers understand autism and cardiometabolic health

The goal of this observational study is to evaluate cardiorespiratory and muscular fitness in children with obesity, with and without autism spectrum disorder (ASD), and examine how fitness relates to body measurements, body composition, and cardiometabolic health. The main questions it aims to answer are: * Do children with and without ASD differ in cardiorespiratory fitness and handgrip strength? * Are fitness levels related to body composition and measures of cardiometabolic health? Researchers will compare children with ASD to children without ASD to see if there are differences in fitness, body composition, and cardiometabolic health. Participants will have information from their medical records reviewed, including: * Cardiorespiratory fitness and handgrip strength test results * Height, weight, BMI, and body composition measurements * Blood test results, including HbA1c and cholesterol levels * Age, sex, and other demographic information

Charlottesville, Virginia, United States
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