Autism Research Studies at Stanford

Study Title/ Age 


SPARK (Simons Powering Autism Research) Study

all ages

If you or your child has a professional diagnosis of autism, Stanford University invites you to learn more about SPARK, a new online research study sponsored by the Simons Foundation Autism Research Initiative. The mission of SPARK is clear: speed up research and advance understanding of autism by creating the nation’s largest autism study. Joining SPARK is simple – register online and provide a DNA sample via a saliva collection kit in the comfort of your own home. Together, we can help spark a better future for all individuals and families affected by autism.

Register in person at Stanford University by contacting us at or online at

Effectiveness of a Developmental Reciprocity Treatment Program in Autism

2-5 years

This is a research study examining the effectiveness of a Developmental Reciprocity Treatment Program in treating social deficits in children with Autism Spectrum Disorders. Developmental Reciprocity Treatment is an early intervention that applies developmentally-informed teaching methods in naturalistic settings in order to target social and communication deficits.

Researchers have begun to develop strategies to investigate the effectiveness of combining a parent training program teaching parents how to implement  Developmental Reciprocity Treatment with in-home, therapist-implemented treatment. To determine the effectiveness of theDevelopmental Reciprocity Treatment Program, it will be compared to a delayed treatment group by conducting a randomized controlled 24-week trial. This research will allow us to help in the development of therapeutic approaches that can meet the increasing service demands for families. We hope that investigating interventions that aim to improve core deficits will aid in providing better care for children with autism.

Neuroimaging Predictors of Improvement to Pivotal Response Treatment (PRT) in Young Children With Autism


2-4 years

Neuroimaging Predictors of Improvement to Pivotal Response Treatment (PRT) in Young Children With Autism

Stanford University researchers are recruiting children with autism to identify brain imaging predictors of benefits from Pivotal Response Treatment (PRT) targeting language abilities.

In order to participate in this research study, your child must:

  • Be between the ages of 2 and 4 years
  • Be able to complete an MRI of the brain during natural sleep
  • Participate in a 16-week parent training program
  • Meet inclusion based on testing.

Natural History Study of Individuals with Autism and Germline Heterozygous PTEN Mutations       

3-21 years

The goal of this study is to gain a better understanding of PTEN mutation syndromes to identify early markers and ultimately effective interventions for autism spectrum disorder. Individuals between the ages of 3 and 21 years are eligible to participate if they have been diagnosed with PTEN hamartoma tumor syndrome or autism spectrum disorder with macrocephaly. The study involves five visits over a two year period. Three of the visits occur on-site at a study location. The other two visits occur as phone calls. The on-site visits include a blood draw, physical/neurological exams and behavioral testing.


Study Flyer 

Clinical Trial of Everolimus in Children and Adolescents with PTEN Mutations

6-21 years

Phosphatase and TENsin homolog (PTEN) gene mutations (mistakes in the gene) are associated with neurocognitive deficits, intellectual disability, autism symptoms, skin lesions, macrocephaly (large head size), overgrowth of tissues, and an increased risk of cancers. Investigators are conducting research to evaluate the potential safety and efficacy (effectiveness) of the drug, RAD001 (everolimus), in patients with PTEN Hamartoma Tumor Syndrome, and the potential neurocognitive benefits from treatment with RAD001 or placebo for a six month period. The investigators hope this trial will lead to a better understanding of PTEN and to new forms of treatment that may benefit children and adults with PTEN in the future.

Clinical Trials Website  

Study Webpage

Oxytocin: Relationships with Autism and Social Functioning      

3-45 years

Pregnenolone Randomized Controlled Trial


14 - 21 years

Neurosteroid Pregnenolone Treatment for Irritability in Adolescents with Autism

Medication treatments for core symptoms of autism spectrum disorder (ASD) continue to be unmet medical needs. The only medications approved by the U.S. Food and Drug Administration (FDA) for the treatment of individuals with ASD are effective in treating irritability and associated aggressive behaviors, but these medications can also cause severe long-term side effects such as diabetes and involuntary motor movements. Therefore, effective medications with more tolerable side effect profiles are highly desirable. This profile is consistent with pregnenolone (PREG). PREG belongs to a new class of hormones known as neurosteroids, which have been shown to be effective in treating various psychiatric conditions including bipolar depression and schizophrenia. As compared to currently FDA-approved medications, our preliminary data suggested that PREG may represent a potentially effective and well-tolerated agent for treating irritability in individuals with ASD. In addition, our experience suggests that PREG might be helpful in improving selected core symptoms such as social deficits and sensory abnormalities of ASD. This study provides the opportunity to further explore the usefulness of PREG in the treatment of irritability and some core symptoms of ASD. We are performing a 12-week randomized double-blind controlled pilot trial to examine the effectiveness of orally administered PREG in reducing irritability and associated behaviors in adolescents with ASD. In this study, we also aim to examine the usefulness of biomarkers (blood levels of neurosteroids, eyetracking and brain wave recording) in predicting treatment response and assessing biologic changes with PREG treatment.

Study Flyer 

Link to study in Stanford's Clinical Trials Directory


Parent Training to Enhance Social Success for Children with Autism Spectrum Disorder

4-6 years

Children with autism spectrum disorder (ASD) exhibit characteristic deficits in social initiation and there are few empirically validated treatments specifically for this core deficit. While parent training is known to be a critical intervention component, few social skills programs involve parents. This study will capitalize on an existing social skills group already established at the community agency Abilities United, with the goal of enhancing the program’s effectiveness by adding a parent component. Specifically, the proposed study will investigate whether a novel parent-mediated social skills program (Social SUCCESS), which provides parent training in conjunction with a social skills group intervention, will result in more frequent initiations during play with typically developing peers. We are currently recruiting verbal children with Autism Spectrum Disorder, aged 4:0 to 6:11 years, who have average to above average cognitive ability. Children will be randomly assigned to either Social SUCCESS or waiting list. Treatment will be provided for 12 weeks and include participation in a social skills group and a separate parent training group. The study will evaluate the effects of Social SUCCESS on areas of core social deficit including frequency of peer initiations using parent ratings, observational

measures, standardized questionnaires, and a social eyetracking task. Outcomes will be compared to a waiting list control group. Findings will aid clinicians in providing more effective social skills treatment for children with ASD and enhance the scientific knowledge-base related to evidence-based social skills treatments.