Autism Spectrum Disorder Clinical Trials

The goals of this project are to (a) incorporate empirical findings on imitation training and learning in autistic children into a comprehensive protocol for Applied Behavior Analysis practitioners designed to rapidly facilitate generalized imitation called Emergent Multi-Class Imitation Training (EMIT), and (b) collect pilot data on the efficacy of EMIT with a small sample of autistic children. EMIT will incorporate several features that are grounded in prior research including: (a) evidence-based procedures for establishing trained matching relations (a pre-requisite to generalized imitation), (b) concurrent training of different response types (e.g., motor imitation, object imitation, vocal imitation) to address restricted generalization, (c) multiple manipulative object imitation training, (d) evidence-based procedures for remediating slow acquisition, and (e) frequent tests for the emergence of generalized imitation. EMIT will be the first protocol designed for clinical use that reflects research findings on imitation learning spanning almost five decades. Read Less

The goal of this observational study is to learn about the impact of the diabetes drug glibenclamide (glyburide) on neurodevelopment in individuals with iDEND (developmental delay, epilepsy and neonatal diabetes) due to the V59M mutation in the KCNJ11 gene. The main question it aims to answer is whether initiating sulphonylurea (SU) therapy in the first year of life results in better neurodevelopmental outcomes in affected individuals, in comparison to starting therapy later than 12 months of age. Participants will undergo a neurodevelopmental assessment comprising parental and teacher completion of standardised questionnaires, and where possible face to face neuropsychological testing. Researchers will compare the outcomes of these standardised tests in the individuals who started SU therapy \<12 months of age in comparison to those who started \>12 months of age. 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

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 is a randomized interventional study designed to evaluate the effects of repetitive Transcranial Magnetic Stimulation (rTMS) on neural and behavioral facets of social cognition in adults with autism spectrum disorder (ASD). Read Less

Autism Spectrum Disorder (ASD) is one of the most frequently occurring childhood-onset neurodevelopmental disorders affecting 1 in every 54 children. Most children with ASD experience challenges participating in daily activities (e.g.: eating, sleeping, bathing, grooming, playing, etc.) and receive occupational therapy intervention to address these. The COVID-19 pandemic has restricted in-person therapy for many of these children and there is an urgent need for evidence-based, validated telehealth intervention. This project will adapt an evidence-based occupational therapy intervention, termed OT4ASD to a telehealth delivery model. The aims of the project are to: 1) adapt the existing intervention protocol to a telehealth delivery model, 2) train therapists and evaluate the therapist's ability to conduct OT4ASD, 3) determine if OT4ASD delivered via the telehealth is acceptable and feasible to parents and interventionists; and 4) whether children improve in the daily living skills. OT4ASD follows a systematic protocol and uses active, individually-tailored sensory motor activities that are specifically designed to address the child's needs. The investigators believe this will be the first telehealth manualized protocol to address the sensory motor symptoms of ASD and measure outcomes at the daily life activity and participation levels. Read Less

FOXP1, also known as Forkhead-box Protein P1, is a transcription factor protein belonging to the FOX gene family. Disruptions in the FOXP1 gene cause a phenotype characterized by global developmental delay, speech deficits, mild dysmorphic features, and traits of autism spectrum disorder. This study seeks to characterize FOXP1-related neurodevelopmental disorders using a number of genetic, medical and neuropsychological measures. Read Less

This proposal will evaluate a series of peer-mediated interventions (PMIs) for preschool children (3 to 6 years) with ASD and limited or no spoken language, using an innovative Sequential Multiple Assignment Randomized Trial (SMART) design. Available evidence supports the beneficial effects of PMIs for improving social communication in children with ASD. Peer-related social competence is vital to a wide range of child outcomes, such as improved communication and fewer behavioral problems. Unfortunately, approximately 30% of children with ASD remain minimally-verbal in kindergarten, restricting participation in inclusive activities. Recent studies report improved communication after a speech-generating device (SGD) is included in treatment. Effective interventions that can be modified is necessary to ensure optimal communication outcomes when children do not make anticipated progress. A strength of the study is that these interventions can be adopted by community-based, early service providers. All participants will receive an adapted Stay-Play-Talk (SPT) peer-mediated intervention that varies in active ingredients. With SMART designs, it is possible to test and identify alternative combinations of PMI approaches, such as the addition of a SGD. In this study, 132 preschoolers with ASD (and N=264 peers without disabilities) will be initially randomized to SPT and SGD with spoken peer input only (SPT Basic; peers taught to model language) or SPT and SGD with augmented peer input (SPT Plus; peers taught to use verbal language models concurrently with the SGD). Each child's response to treatment after 5 weeks will determine that child's next phase in the SMART design. Children showing a positive response will continue in their originally assigned group; slow responders will be randomly assigned to receive added treatment components to improve communication (either SPT Plus or SPT Advanced). SPT Advanced adds direct instruction strategies (i.e., adult prompts, reinforcers, and teaching trials) to increase child vocalizations in SGD interventions. The use of a SMART design extends our prior work by testing the systematic addition of selected peer-mediated strategies in combination with an SGD that allows for flexible application of interventions based on child response. The investigators have assembled an outstanding team of highly qualified investigators with complementary skills in preschool assessment, language intervention, clinical trials, and statistics. Read Less

This is a multicenter, open-label study to evaluate the efficacy and safety of a daily single oral dose of tasimelteon in treating sleep disturbances in pediatric and adult participants with ASD. Read Less

Children with Autism Spectrum Disorder (ASD) and insomnia, and their parent(s) will undergo 4 sessions of behavioral therapy for sleep problems followed by 4 bimonthly booster sessions. Children and their families will be randomly assigned to one of three conditions: cognitive behavioral therapy (in-person), cognitive behavioral therapy (remote), or behavioral therapy (remote). Arousal will be measured through heart-rate variability. Sleep and secondary outcomes (child daytime behavior, parent sleep) will be collected at baseline (weeks 1-2 before starting the treatment), post-treatment (weeks 6-8 from baseline), 6-month follow-up, and 12-month follow-up. Read Less