Autism Spectrum Disorder Clinical Trials

Difficulties in reciprocal social interaction are hallmark features of several neuropsychiatric disorders, most notably autism spectrum disorder (ASD) and schizophrenia spectrum disorder (SSD). While recent studies have demonstrated substantial overlap in genetic etiology between ASD and SSD, little is known about common versus unique neural mechanisms that may underlie these downstream social deficits that cross diagnostic boundaries. Thus, a comprehensive imaging study examining social deficits in youth with ASD and adolescent- onset SSD at the neurochemical, connectivity, as well as functional activation level will be crucial in furthering our understanding of these underlying neural mechanisms. Specifically, the current project aims to examine how targeted social skills interventions may impact the organization of large-scale functional brain networks implicated in social cognition in these disorders, leading to improved outcomes. Thirty adolescents with ASD and 30 adolescents with SSD will undergo the Program for the Education and Enrichment of Relational Skills (PEERS), which is a 16-week parent-assisted social skills intervention that aims to improve friendship quality and social skills in teens with social difficulties. All participants will receive pre- and post-treatment MRI scans including functional MRI and magnetic resonance spectroscopy to quantify neural changes resulting from the intervention. All participants will also receive behavioral and social cognition assessments pre- and post-intervention to quantify real- world gains in social behaviors resulting from the intervention. Additionally, 30 typically developing adolescents will be recruited to serve as control participants and undergo two MRI and behavioral assessment sessions 16-weeks apart with no intervention in between. Specific aims include (1) examining inter-group disruptions in connectivity patterns, activation levels, and neurometabolite concentrations in key social brain regions pre-treatment in ASD and SSD groups, (2) examining inter-group changes in connectivity patterns, activation levels, and neurometabolite concentrations in key social brain regions in response to treatment in ASD and SSD groups, and, (3) dimensionally identifying intra-group differences in brain responses and how they relate to real-world treatment outcomes. Read Less

The goal of this clinical trial is to compare two school based interventions to manage anxiety in autistic students. The main question\[s\] it aims to answer are: 1) Compare the effectiveness of Facing Your Fears-School Based Program (FYF-SB) with Zones of Regulation (ZOR) on anxiety reduction and emotion regulation; and 2) examine the feasibility and satisfaction of FYF-SB and ZOR, according to students, caregivers and school providers. Autistic students with anxiety between 8-14 years will be randomized to either FYF-SB and ZOR. They will participate in one of the programs for 12 weeks and pre, post and follow-up measures will occur. Read Less

The goal of this early Phase 1 clinical trial is to assess if the social content of a story impacts autistic children's listening comprehension of stories. The main questions this study aims to answer are: * Does removing social content from a story improve listening comprehension in autistic children? * Does listening comprehension of more social versus less social stories differentially predict performance on a standardized reading comprehension measure? Participants will listen to more social and less social stories while viewing accompanying pictures and answer comprehension questions about the stories and complete a standardized assessment of reading comprehension. In addition, participants complete measures of their nonverbal cognition, hearing status, autism severity, language abilities, and social communication abilities to help characterize individual differences in participants. Read Less

The purpose of this study is to systematically evaluate the results of medical investigations to identify symptom and biological patterns and common etiologies of neurodevelopmental disorders. Read Less

This Phase 2 study examines the safety, tolerability, and preliminary efficacy of pimavanserin in individuals with Autism Spectrum Disorder. Male or female participants aged 16 to 40 years of age will be randomized to receive single doses of either placebo or pimavanserin in this randomized, placebo-controlled, cross-over designed study, followed by open label extension. Read Less

The objective of this proposed study is to collect initial efficacy data on a telehealth parent-based behavioral program for children with autism and overweight or obesity (PBT-A), compared with health education (HE). Read Less

The increased prevalence of autism spectrum disorder (1 in 36 youth) in the United States along with the exorbitant cost of care of supporting one autistic individual with and without intellectual disability across their lifespan ($2.4 and $1.4 million, respectively) creates a sense of urgency to improve outcomes for autistic youth. Schools are the primary setting in which autistic children receive intervention. Despite a growing array of evidence-based interventions (EBI) such as behavioral management, EBIs often are unavailable in schools due to misalignment of existing implementation strategies (IS) with that setting. Large numbers of IS have been described for the education sector, but there is a dire need to identify the most efficient IS to support use of EBI in resource-strapped public schools. This project will apply the local Discover, Design/Build and Test (DDBT) framework to redesign and pilot a multifaceted IS to support educator use of a behavioral skills EBI for autistic children in public schools. Read Less

ML-004-002 is a multi-center, randomized, double-blind, parallel-group, placebo-controlled study that will enroll approximately 150 adolescent and adult subjects with ASD. The primary objective is to evaluate the efficacy of ML-004 compared with placebo in the improvement of social communication deficits in subjects with ASD. Read Less

The social cognitive deficits associated with autism spectrum disorder (ASD) are related to an imbalance in excitatory and inhibitory neurotransmission, specifically a deficit in the inhibitory neurotransmitter GABA. The investigators have used magnetic resonance spectroscopy (MRS) techniques to measure GABA in specific brain regions and have demonstrated that a single dose of gabapentin increases GABA in brain regions associated with social cognition. This study will use a biomarker-driven approach to investigate gabapentin to correct the underlying imbalance of neurotransmitters and improve the core social cognitive deficits in ASD. By using a brain-based biomarker (GABA) that is quantifiable and measurable, the investigators can target this biomarker directly and measure the impact of the treatment. This will help with the future development of targeted therapies for ASD and provide an early marker of response to aid in the selection of individuals more likely to respond to various treatments. The specific aims of this study are to: 1) determine if treatment with gabapentin sustainably increases GABA in the right anterior insula (RAI; an area of the brain involved in social cognition), 2) determine if response of RAI GABA levels to a single dose challenge of gabapentin predicts a sustained response after treatment, and 3) determine if the increase in GABA levels with gabapentin treatment translates into clinically measurable improvement in social cognition. The investigators will conduct an 8-week open-label clinical trial of gabapentin in 40 adolescents (age 13-17 years) with ASD, using MRS before and after treatment to measure GABA in the RAI (the primary outcome for the study). Before the trial, a single dose challenge of gabapentin will be used to evaluate the immediate response of GABA levels in the RAI, to determine if this predicts later response. A secondary outcome will be the clinical effects of gabapentin on social cognition. This study can demonstrate for the first time that neuroimaging biomarkers can be used to guide treatment of social cognition deficits seen in ASD and that the excitatory-inhibitory imbalance in neurotransmitters in ASD can be pharmacologically targeted. This can provide a rational basis for pharmacological treatment of the core social deficits of ASD, providing direct benefit to participants in the study as well as indirect benefit to countless patients in the future. Read Less

A within-subjects design will be used for this preliminary investigation of four biomarkers across two contexts of use: prediction of treatment response (i.e., stratification) and quantification of response (i.e., change). Read Less

Anxiety is prevalent in young children, under 7 years of age, with autism. Yet, few studies have examined anxiety interventions for this age range, and only one anxiety treatment study has included young children with cognitive and language delays. Anxiety treatment models utilizing cognitive-behavioral therapy (CBT), adapted for children with autism, are empirically supported in school-age autistic children. Further, preliminary evidence suggests CBT approaches may reduce intolerance of uncertainty (IU), a mechanistic construct that may contribute to the maintenance of anxiety in autistic children. This study seeks to address the existing gap in anxiety treatment by examining the feasibility and preliminary efficacy of a novel, telehealth CBT intervention, DINO Strategies for Anxiety and intolerance of Uncertainty Reduction (DINOSAUR), which targets both anxiety and IU in young autistic children. Read Less

Research teams in Boston University, UCLA, and Weill Cornell will recruit 90 participants in 3 years (10 participants per site per year) and evaluate JASPER, play-based intervention, using the BOSCC and ELSA-T. Participants will be randomized to receive JASPER facilitated either by a clinician or the caregiver. After 10 weeks, the participants will be evaluated using the CGI to determine if they are "responders" or "non-responders". Nonresponders will be given a mix of clinician and caregiver-facilitated JASPER and responders will remain the course for the following 10 weeks. Coding of the BOSCC and ELSA-T will be the outcome measures and change will be evaluated throughout the study. Read Less