Autism Spectrum Disorder Clinical Trials

This is a pivotal, prospective, double-blind, study to evaluate the sensitivity and specificity of the SenseToKnow app for the detection of autism spectrum disorder in children 16-36 months of age. Read Less

This study will assess clinical and behavioral measures along with electroencephalogram (EEG), event-related potentials (ERPS), and eye-tracking (ET) prior to and following a single intermittent Theta Burst Stimulation (iTBS) session to provide preliminary insight into the potential of TMS as an intervention for depression in individuals with Autism Spectrum Disorder (ASD). Read Less

The purpose of this study is to identify whether researched and commonly used face-to-face interventions can be effectively implemented through artificial intelligence (AI) using an application on the phone or computer. The investigators plan to recruit verbal individuals diagnosed with autism spectrum disorder who demonstrate challenges with socialization. Modules focusing on various difficulties experienced by autistic individuals will provide practice and feedback using voice recognition and feedback. If effective, this intervention can be scaled up to provide cost-effective accessible assistance to individuals, particularly those who do not have access to care or prefer to secure services in the comfort of their own homes. Read Less

The goal of this collaborative R01 is to demonstrate the therapeutic value and community-wide implementability of an early intervention (EI) platform for toddlers with autism spectrum disorder (ASD) that is completely virtual, from recruitment through intervention. This platform-Early Social Interaction Mobile Coaching (ESI-MC) deploys individual telehealth sessions with coaching and feedback to help families embed intervention in everyday activities. Specifically, the investigators will conduct an effectiveness trial of ESI-MC to address the important question of whether starting evidence-based intervention earlier leads to better outcomes than starting later. The investigators will address this question by using a modified stepped wedge design and blended implementation research to analyze data obtained with ESI-MC start at 18, 24, or 30 months. The investigators will diagnostically ascertain 240 children from a pool of 360 18-month-olds with early signs of autism, 30 in each of 8 US regions (Central and SW Florida; Atlanta, GA; suburbs of Philadelphia, PA; New York City, NY; Cincinnati, OH; Chicago, IL; Seattle, WA; and Los Angeles, CA). Research participants will be recruited using a new virtual platform-My Baby Navigator-linking a new surveillance and screening tool, an app to upload video-recorded home observations and telehealth intervention sessions, and a package of educational resources. The 240 children will be randomly assigned to one of three ESI-MC timing groups. ESI-MC will be delivered by community-based early intervention providers (EIPs) currently working within the the early intervention system in the recruitment regions. The investigators will measure child active engagement and social communication change every 6 months as the primary outcome variables. Outcome measures of developmental level, autism symptoms, and adaptive behavior will be examined to measure differential treatment effects. Maximizing the use of mobile technology, ESI-MC offers the prospect of a community-viable, scalable and sustainable treatment to improve EI services for toddlers with ASD, particularly among minority and low-resource communities. Read Less

Background: Some patients with unusual genetic conditions are referred to the National Institutes of Health (NIH). They may not be eligible to join current research studies. Testing such patients is a good way to improve the skills of research staff. The findings could lead to new processes and research. Objectives: To recruit a diverse group of pediatric subjects with genetic disorders. To give clinic staff hands-on experience working with these patients. Eligibility: Children any age with a known or suspected genetic disorder. Design: Participants will be screened with medical history and physical exam. They may have lab and other tests. Family members may give DNA samples. Participants will have: Medical history Physical exam Height, weight, and other measurements taken. A clinical evaluation of their disorder. They may have: Blood, urine, and saliva samples taken Imaging tests. These may include x-rays, scans, ultrasound, or skeletal survey. A sleep study A visit with other specialists at NIH A genetic test from a commercial lab Medical photographs taken Other tests Participants may have follow-up visits. They may get medical or surgical treatment. ... 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

The goal of this observational study is to test a reciprocal relationship between statistical learning and the development of language and literacy in first-graders with autism and their non-autistic peers. The main questions it aims to answer are: whether children's statistical learning abilities can predict their long-term improvement of language and literacy skills in school; how children's brains automatically learn patterns from speech and prints; whether children's learning in the lab reflects the language patterns they have learned over the years from their native language. First-grade students will participate in the study twice across three months. During Time 1, children will complete a battery of language, reading, and cognitive assessments a series of computer-based statistical learning games both inside and outside of functional MRI scanner. During Time 2, children will complete a battery of language and reading assessments to detect the growth in three months. Researchers will compare the autistic and the non-autistic groups to see if statistical learning plays a similar or different role in predicting children's language and literacy growth. 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

DDX3X syndrome is a genetic cause of intellectual disability and other neurologic features including, in some cases, autism. Variants in the DDX3X gene are thought to account for 1-3% of unexplained intellectual disability in females, making it one of the more common causes of intellectual disability.This study seeks to characterize DDX3X-related neurodevelopmental disorders using a number of genetic, medical and neuropsychological measures. Read Less

This protocol is a blinded randomized controlled study of the effects of BB-12 with LGG at different doses in 70 healthy children with autism spectrum disorders at lower and higher doses over an 56-day period and a 28- day observation period. The study is being conducted in order to assess safety and tolerability of the probiotic (BB-12 with LGG) at 2 different doses of BB-12 with LGG. Identifying effects on behaviors in healthy children with ASD using SRS-2 and ABC, GI symptoms using GI symptom severity index, and relevant biomarkers of inflammation, microbiota, and metabolites. Primary testing and procedures will be conducted at the University of Texas Health Science Center at Houston and Memorial Hermann. Biomarker identification includes Integrative analysis of plasma metabolome and stool microbiota will be conducted with the collaboration of Dr. Ruth Ann Luna and Dr. Jim Versalovic at Alkek Center for Metagenomics and Microbiome Research, Department of Molecular Virology & Microbiology of Baylor College of Medicine. Read Less

This study will inform understanding of how to support emerging adults with autism spectrum disorder (ASD). The investigators will use a theoretically informed and research-based transition support program developed for people with ASD and test how effective it is when delivered by community agencies. Results will provide information on the effectiveness of the program as well as on mechanisms of change and contextual factors that promote or impede implementation. Read Less

The AIRB research team will compare the use and effectiveness of each intervention (Mind the Gap, Remaking Recess and Self Determination Learning Model of Instruction) with and without the addition of an implementation strategy, UNITED. In all groups, the research team will train community practitioners using remote delivery of professional development modules specific to the intervention, and active coaching for up to 12 sessions as dictated by the intervention procedures during a time span of 6 months. The research team will pair UNITED with three interventions that cover the ages of early childhood, childhood, and adolescence. These include Mind the Gap (MTG), a family navigation intervention for children newly diagnosed under age 8, Remaking Recess (RR), a school-based social/peer engagement intervention for children ages 5-12, and Self-Determination Learning Model of Instruction (SDMLI), a self-advocacy intervention for adolescents (13-22 years; 22 is the upper age limit of high school for individuals with disabilities). For MTG, peer navigators (staff working in an organization that already works with parents) and parents (with children with ASD) will meet via phone or video conference for up to 12 sessions within a 6 month span (an hour each time). They will go over sessions that were created by the research group that help parents understand the system of acquiring services. The total time commitment for peer navigators is about 18 hours and for parents with children with ASD is about 8.5 hours. Identified families connected to the community organization will be matched with one peer navigator who will then guide and support the caregiver through completion of the MTG modules with active coaching of the family. Family needs and preferences will guide topic selection. Active coaching will occur via zoom, or over the phone, based on family preference and some recorded sessions will be shared with the research group for analyses. Mind the Gap will be available in English, Spanish, and Korean. Read Less