Autism spectrum disorder (ASD) is a neurodevelopmental condition characterized by differences in social communication and repetitive or restricted behaviors, with a very wide range of abilities and support needs across individuals. Research is increasingly focused on co-occurring conditions and quality of life rather than trying to eliminate autistic traits.
What's actually going on in research
No medication changes the core features of autism, but several drugs are in trials for specific co-occurring challenges — irritability, repetitive behaviors, and gastrointestinal problems. Oxytocin, arbaclofen, and bumetanide have each shown promise in subgroups and are being tested in larger, more carefully designed trials. Gene therapies are entering early trials for specific single-gene autism syndromes like SYNGAP1 and SHANK3, where the genetic cause and potential correction point are known.
Targeted drug trials
Drugs including mGluR5 modulators, arbaclofen, and bumetanide are being tested for specific core and associated features of ASD, with trials stratified by genetic subtype.
Gene therapy for rare subtypes
For autism caused by single-gene mutations, gene therapy trials are beginning — targeting conditions like SYNGAP1 deficiency and Phelan-McDermid syndrome with gene replacement strategies.
Behavioral intervention research
Large trials are comparing the intensity, timing, and digital delivery of naturalistic developmental behavioral interventions to identify the most effective and accessible approaches for young children.
What to know before you search
Eligibility requires a confirmed ASD diagnosis, often a specific genetic subtype for gene therapy trials, and age and IQ ranges that vary by study.
What types of trials are currently open
- Behavioral therapy trials — Comparing early intervention models, intensity levels, and delivery formats for core ASD features.
- Drug trials — Testing medications for irritability, repetitive behaviors, sleep, and gastrointestinal co-occurring conditions.
- Gene therapy trials — Evaluating gene replacement and editing strategies for specific single-gene autism subtypes.
- Caregiver support trials — Testing parent-mediated interventions and caregiver training programs.
- Quality of life trials — Measuring outcomes that autistic individuals and families prioritize, including autonomy and community participation.
Recently added Autism Spectrum Disorder trials
A Digital Health Parent-Training Intervention for Children With Social Communication Delays
Early intervention, during a time of optimal brain plasticity, is critical for autistic children to develop functional skills such as communication and daily living abilities. However, many families face barriers to accessing timely autism-specific services due to delays in autism diagnosis (which is often a prerequisite to autism-specific intervention), long waitlists, high costs, and the necessity to travel far distances to service providers. Self-directed parent-mediated digital health interventions (i.e., programs that parents complete online without provider coaching or feedback) offer a scalable solution to reduce challenges accessing intervention by providing evidence-based strategies that parents can implement without relying on traditional service pathways. The proposed pilot randomized controlled trial (RCT) will test a revised self-directed parent-training program, that teaches parents naturalistic developmental behavioral intervention (NDBI) strategies, which have proven effective for young children with autism. The study will include young children who have already been diagnosed with autism and those who screen positive on an autism screener but have not yet been evaluated, enabling earlier intervention before a formal diagnosis. The study will also explore parents' willingness to engage in the intervention. The specific research aims are: (1) test the preliminary efficacy of the parent-training program vs. treatment as usual (TAU) for 50 children with autism traits and their parents; (2) examine parent engagement in the intervention; and (3) explore predictors of engagement.
Recording Stress Biomarkers in Autism Spectrum Disorders
The goal of this observational study is to learn how stress affects children and young people with autism spectrum disorder (ASD). Many individuals with autism experience strong stress reactions that may lead to challenging behaviours such as agitation, withdrawal, aggression, or self-injury. These behaviours can be difficult to predict, especially in people who have limited communication abilities. Researchers want to better understand how the body reacts to stress in real-life situations. The study focuses on two main biological systems involved in the stress response: the autonomic nervous system, which produces fast reactions such as changes in heart rate and sweating, and the hypothalamic-pituitary-adrenal axis, which produces slower hormonal responses such as cortisol. The main questions the study aims to answer are: * Do physiological stress signals differ between individuals with ASD and those without ASD? * Are there differences in physiological stress responses between individuals with ASD and non-ASD participants? * Can physiological markers help identify stress earlier in people with autism? Researchers will compare children and young people with autism to a control group of participants without autism to see whether their stress responses differ. Participants will take part in monitoring during their normal daily activities. This allows researchers to observe stress responses in natural environments such as school, home, or specialized care institutions. Participants will: * Wear a wrist device during the day that measures heart rate, heart rate variability, skin conductance, skin temperature, and movement * Provide saliva samples in the morning and afternoon to measure stress hormones such as cortisol and alpha-amylase * Have additional saliva samples collected after behavioural crises or stressful events when possible * Be observed by a trained researcher who records behavioural events and the surrounding context Researchers will combine physiological data, behavioural observations, and contextual information such as physical activity, environmental conditions, and daily routines. This will help identify patterns of stress in everyday life. The results of this study may help researchers better understand the physiology of stress in autism and support the future development of wearable systems that could detect stress early and help prevent behavioural crises.
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