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 trials — Testing medications or technologies aimed at improving social interaction, eye contact, and communication skills in autistic children and adults.
- Behavioral symptom trials — Studies of drugs for irritability, aggression, hyperactivity, or repetitive behaviors that can interfere with daily life.
- Anxiety and mood trials — Testing treatments for anxiety, depression, and mood symptoms, which are common in autistic people.
- Biomarker studies — Research looking for biological markers that might predict treatment response or identify subtypes of autism.
- Technology and device studies — Testing apps, wearable devices, and other technologies that support communication, emotion recognition, or daily living skills.
Recently added Autism Spectrum Disorder trials
Effects of Transcranial Direct Current Stimulation for Enhancing Cognitive Function in Individuals With Autism Spectrum Disorder - A Pilot fMRI Study
Autism Spectrum Disorder (ASD) is a pervasive neurodevelopmental disorder that currently affects 1 in 32 children. ASD is characterized by persistent deficits in social communication and restricted, repetitive behaviors. For instance, people with ASD often show great difficulty in recognizing social cues and understanding others' emotions, as well as having a tendency to act inflexibly and inappropriately in social situations, all of which contribute to lifelong social and occupational dysfunctions.To date, there is no cure for ASD. Existing treatments are primarily behavioral or educational based. Although some treatments show positive therapeutic effects in reducing patients' core autistic symptoms, the interventions are often labor- and time-intensive. Recently, a non-invasive painless brain stimulation technique, "transcranial direct current stimulation (tDCS)," has received increasing interest in clinical settings as a potential tool for cognitive enhancement and neurorehabilitation for various cognitive dysfunctions. This study aims to examine the effectiveness of tDCS treatment combined with cognitive training for individuals with ASD. We will study the effect before and after treatment.
Evaluation of the TAP-Preschool (TAP-P) for Tele-assessment of Autism in Preschool-aged Children
Use of tele-assessment for evaluation and diagnosis of autism in toddlers has demonstrated success in increasing service access and reducing clinical wait times, with high levels of diagnostic accuracy, clinician confidence, and family satisfaction; however, these models have not yet been rigorously evaluated within the preschool age range. The TAP-Preschool (TAP-P) was developed by the investigative team for tele-assessment of autism in preschool-aged children, with preliminary studies demonstrating utility with many children with limited verbal language and some children with more flexible verbal language. The proposed study will recruit a larger sample of children with varied language and phenotypic profiles to (1) thoroughly evaluate the psychometric properties of the TAP-P and (2) investigate child and family factors associated with diagnostic ascertainment and family satisfaction with tele-assessment to inform the creation of a decision support tool intended to recommend families for either tele- or in-person assessment, with the ultimate goal of optimizing clinical care models to serve patients in efficient ways while routing families to diagnostic care pathways that best fit their needs.
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