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.

Link to study in Stanford's Clinical Trials Directory

oRBiting Study

We know that no two people with autism are the same. And of course we wouldn’t want them  to be. But people with autism often have behaviors that they repeat, known as repetitive and restricted behaviors, or RRBs. So we are conducting the oRBiting study to better understand the different scales that have been developed to measure RRBs in autism, and to explore the use of wearable technologies for gaining important insights into daily behaviors.

oRBiting is a non-drug observational study which we hope will help to inform later studies as to whether investigational drugs could make life with autism a little easier.  We’re looking for children and adults with autism to join the  oRBiting study who are aged 5–45 years and have someone they see and speak to regularly who can be their study partner and accompany them to all clinic visits (for example, a parent or close friend).

For more information, please contact Rachel Schuck at 650-736-1235 or autismdd@stanford.edu

Stanford University researchers are currently recruiting children and adolescents with autism (i.e. typically developing) to participate in a research study. The study compares individuals with Autism Spectrum Disorder to typically developing individuals so we can better understand the causes of autism.

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 Webpage    

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

Stanford University researchers are currently recruiting same-sex twin pairs with autism and twin pairs without autism (i.e. typically developing) to participate in a research study. The study compares twins with Autism Spectrum Disorder to typically developing twins so we can better understand the causes of autism.

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

The goal of this study is to examine the effectiveness of a novel resilience training program for parents of children with Autism Spectrum Disorder (ASD), the AMOR Method. The program is designed to teach parents of children with ASD (4-10 years old) various resilience techniques including mindfulness, optimism, and acceptance. This treatment approach is based on several principles of resilience training, mindfulness practice, Grief and Loss Processing Therapy (GLPT), Acceptance and Commitment Therapy (ACT), and optimism training. Specifically, we are looking to see the different parent factors that may contribute to resilience development. 

The 8-week program includes both a parent group and an individual component. Parents will be asked to participate in weekly group parent training sessions at Stanford University to learn the science, foundation, and skills of the treatment from study staff as well as receive feedback on weekly homework assignments of practicing the skills outside of group. In addition, parents will enroll in three individual sessions with research staff on specific treatment content including grief and loss processing, optimistic thinking strategies, and resilience maintenance over time. We hope that by investigating what treatments are most effective in improving resilience it will aid clinicians in providing better care for parents of children with ASD