Clinical Research Directory

A Phase 2A Trial of DT402 for Autism Spectrum Disorder

A Phase 2A Trial of DT402 Open-Label Study in Adults with Autism Spectrum Disorder

Theta Burst Stimulation for Refractory Depression in Autism Spectrum Disorder

Evaluate the efficacy of accelerated theta burst stimulation (aTBS) in reducing depressive symptoms in autism spectrum disorder (ASD)

Differences in N-CAD Concentration and Brain Function Between Children With ASD and TD

The incidence of autism spectrum disorders is increasing, about 1 / 36. Neural cadherin (NCAD) is closely related to synaptic structure and function. The cdh2 gene encoding NCAD has been shown to be associated with a variety of neurodevelopmental diseases and is one of the six risk genes for ASD. Functional near-infrared spectroscopy (fNIRS) is a neuroimaging technology that uses the principle of near-infrared spectroscopy to detect the functional activation of human cortex. The application of fNIRS to explore the neural mechanism of children with ASD has developed rapidly. It has the advantages of non-invasive, portable, relatively low cost and unlimited physical activity. And for ASD children, their command compliance and self-control are relatively low, so fNIRS is more widely used in ASD children. According to dsm-5 standard, we recruited ASD children and TD children, collected their peripheral blood, and detected the plasma n-cad concentration. At the same time, the resting state of fNIRS of the two groups of children was collected, and the ultra scan monitoring was carried out simultaneously during Gesell assessment. The purpose of this study was to explore the difference of peripheral blood NCAD concentration between preschool ASD children and TD children and the difference of brain function in resting state and social interaction state. And the relationship between the characteristics of brain function and the level of cognitive development in children with ASD.

Comprehensive Toileting Program

The current study aims to monitor fecal continence after autistic youth complete enuresis treatment and for individuals who continue to experience encopresis after acquiring urine continence, evaluate a caregiver-mediated version of a Multidisciplinary Intervention for Encopresis (CM-MIE) delivered via telehealth to determine efficacy in a randomized clinical trial.

Adia MED of Winter Park LLC Autism Spectrum Disorder Research Study

This 24-month study is testing whether adding AdiaVita, an umbilical cord blood-derived stem cell and exosome product, to glutathione therapy helps improve autism symptoms in children ages 3-12 more than glutathione alone. Children will be randomly placed into one of two groups for the first three months: one group receives glutathione only, and the other receives glutathione plus monthly intravenous AdiaVita infusions. Both groups also use topical glutathione cream twice daily at home. Autism symptoms will be tracked over two years using the Autism Treatment Evaluation Checklist (ATEC) filled out by parents and by therapists or teachers. Safety, side effects, quality of life, and overall well-being will be closely monitored through regular clinic visits, physical exams, blood tests, and adverse event reporting. After the initial three-month phase, children who received glutathione alone may cross over to receive AdiaVita infusions at no additional cost if safety checks at month 6 are satisfactory. Approximately 100 children with a confirmed autism diagnosis from the Central Florida area will take part. Participation is completely voluntary, and families may withdraw at any time.

Omega-3, Autism Spectrum Disorder (ASD)

This is a 12 week study to investigate the effects of omega-3 polyunsaturated fatty acids (PUFAs) in youth with autism spectrum disorder (ASD).

Trial of Center-Based vs. In-Home Pivotal Response Treatment (PRT) in Autism

The aim of this clinical trial is to compare the efficacy of a 16-week center-based Pivotal Response Treatment (PRT-C) versus home-based Pivotal Response Treatment (PRT-H) in targeting social communication deficits in young children with autism spectrum disorder (ASD) with significant language delay. The two groups will also be compared to a control group that consists of children who are receiving treatment as usual (TAU).

Mental Imagery Therapy for Autism (MITA) - an Early Intervention Computerized Language Training Program for Children With Autism

Mental Imagery Therapy for Autism (MITA) is a highly innovative adaptive language therapy application for children with autism. MITA exercises are limitless in variations, therefore avoiding routinization. Each activity is dynamic, quickly adjusting to the child's exact ability level. All activities are disguised as games that engage children. A 3-year observational clinical study of 6,454 children with ASD demonstrated that children who engaged with MITA showed 2.2-fold greater language improvement than children with similar initial evaluations (p\<0.0001). This study explores MITA intervention in a randomized controlled trial of 60 children with ASD. Two- to five-year-old ASD children will be randomly assigned to one of two groups. The MITA group will supplement their conventional language therapy with MITA exercises. The control group will receive treatment-as-usual. The hypothesis is that the MITA group will show greater improvement in developmental milestones.

GENES AND AUTISM - IPSC

Autism spectrum disorder (ASD) is a neurodevelopmental disorder characterized by deficits in social interaction and verbal and non-verbal communication (DSM-5, 2013), affecting approximately 2% of the general population. In 5 to 40% of cases, genetic factors are identified as the cause of these disorders, with prevalence depending on the technique used (exomes and/or SNP arrays) and the associated intellectual deficit. In the majority of cases, the aetiology remains unknown. Studies of microdeletions/microduplications (copy number variants) or Whole Exome Sequencing and Whole Genome Sequencing (Single Nucleotide Variants) show the involvement of numerous genes in the predisposition to autism. ASD remains a genetically heterogeneous disorder, as more than 250 genes have been associated with ASD to date. The main objective of the project is to continue identifying genetic factors, and also to understand the biological mechanisms involved in the emergence of autistic symptoms. Identifying biological pathways is an essential step in developing new therapeutic strategies. In addition, one of the major challenges of this study is to better understand the phenotype/genotype relationships in ASD. This requires in-depth knowledge of the phenotypic characteristics of ASD participants and their families, as well as neurotypical populations. This study combines the scientific expertise of researchers specializing in molecular biology, phenotypic exploration (clinical, cognitive, MRI, EEG, biochemistry, immunology), and the use of pre-therapeutic cellular models (iPSCs, neural precursors, organoids). The objectif of this work is the identification of numerous genes associated with ASD and involved in synaptic formation and regulation: NLGN3-4, SHANK1 and SHANK3, CNTN-6, and CNTNAP4. This work was combined with in-depth phenotypic explorations of ASD participants and their relatives. It has made it possible to clarify the neuroanatomical characteristics of ASD participants and their genetic substrate, as well as the underlying cognitive processes. All of this work opens up new prospects for identifying new therapeutic targets using preclinical cell models (IPS, neural progenitors, organoids) developed in particular by I-Stem and Human Technopole.

Testing Scalable Caregiver Interventions for Autism

Autism spectrum disorder affects 1-2% of children worldwide, yet access to quality care remains limited, especially in underserved communities. Families face systemic barriers such as workforce shortages, high caregiver stress, and a lack of culturally appropriate services. To address these gaps, researchers developed a group-based caregiver training program to improve caregiver well-being and child communication and behavior. Successfully piloted in rural U.S. communities and western Kenya through the AMPATH Program, the intervention showed promising results in reducing caregiver stress and autism severity. Building on this success, a new study will evaluate two delivery models-professionally-led and peer-led-using a rigorous effectiveness-implementation trial. The project applies a reciprocal innovation approach, using insights from Kenya to inform U.S. strategies for scaling community-based autism support. The long-term goal is to reduce disparities in autism care by creating scalable, low-cost, caregiver-driven models. A Community Advisory Panel will guide the research to ensure relevance and impact. This initiative represents a transformative step toward equitable autism services across global and U.S. settings.

Effects of Neuralli® MP on Self-Defined Outcomes in Adults With Autistic Traits

To evaluate the efficacy of Neuralli® MP in improving participant-defined outcomes in adults with autistic traits.

MRI-Eye Tracking Pairing, a Tool for Assessing Social Cognition in Children With ASD

Most studies use static visual percepts that are less representative of joint attention versus an ecological environment. This has the consequence of decreasing the perception of an interaction with a social partner, which is an essential step in achieving joint attention. The originality of this study is to improve the design of visual percepts (in the form of video) in order to mimic an ecological environment as much as possible by using MRI-ET coupling. The second originality of this study is the longitudinal exploration of the neurodevelopment of social cognition in autistic children. Studies by the Redcay and Oberwelland teams observe different activations at different ages. The hypothesis is that the perception of joint attention varies over time in people with ASD. To date, there are no studies to determine the influence of childhood neurodevelopment in autistic people on the perception of joint attention. It would be unprecedented to use the MRI-ET pairing as a tool for assessing social cognition as a function of the development of children with ASD.

Electrocardiographic and Electrophysiologic Changes After Percutaneous Closure of Atrial Septal Defect

Primary outcomes : Determining the incidence of SAN and AVN dysfunction before and after percutaneous ASD closure Comparing ECG and EP parameters of SAN and AVN before and after percutaneous ASD closure Secondary outcomes : Assessing clinical, echocardiographic and procedural risk factors affecting the AVN function after ASD closure device implantation Determining the incidence of supraventricular arrhythmia inducibility before and after percutaneous ASD closure

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

The purpose of this study is to determine cross-sectional and longitudinal medical, behavioral, and cognitive differences between PTEN ASD and other groups, as well as to identify cognitive, neural systems, and molecular biomarkers specific to PTEN ASD. In addition, this study will be creating and maintaining a biorepository and linked phenotypic database for PTEN ASD.

An Intervention Program to Improve Emotion Regulation Among Parents of Children with ASD

To examine the effectiveness of a short-term therapeutic workshop in improving reflective functioning, the belief that emotions can change, the reported use of effective emotional regulation strategies, and perceived parental self-efficacy and child behavior.

Lower Urinary Tract Symptoms and School Functioning in Children

Children in primary school often suffer from lower urinary tract symptoms (LUTS), which may negatively impact their overall well-being. Co-occurring neurodevelopmental disorders (NDDs) can adversely affect children as well and can cause restrictions in their daily life, especially in their school-environment. The goal of this observational study is to identify the prevalence of LUTS in Flemish primary school children.The main questions it aims to answer are: * How prevalent are LUTS in regular primary education? * Is there a relation with well-being in school environment? * Is there an influence of co-occuring NDDs? Children, parents and teachers will be asked to fill in questionnaires related to this research question.