Clinical Research Directory

Parent-implemented Social Communication Treatment in Preschool Children With Autism Spectrum Disorder

An accumulation of research evidence has pointed to parent-implemented communication treatment as effective in reducing the severity of social communication deficits in preschool children with ASD. Despite even high-quality evidence, real-world translation to clinical practice remains challenging, especially for children from lower-income families, for two reasons. First, the treatment outcome is highly variable despite study-level efficacy data, most likely due to unique child and parent factors that make treatment response uneven across individual children. Second, the cost of intervention with the largest effect sizes remains high due to its one-on-one format. With the overarching goal to reduce cost and to increase treatment effectiveness at the individual-child level, this project will conduct a randomized controlled trial (RCT) to compare the effectiveness of two options for intervention to address two specific objectives. The investigators will first ascertain whether parent-implemented communication treatment taught by a speech therapist in an Individual (one-on-one) format is more effective than treatment taught in a Group format (up to 8 families learning together) at the study level. The Individual format is at least 4 times more expensive than the Group format; its relative treatment effect must be empirically ascertained to justify its cost. The investigators will then evaluate what combinations of parent and child behavioral and neural factors determine which format of intervention is likely to be more effective at the individual-child level. It is likely that not all families require the more costly Individual format of intervention. Machine-learning analytics with cross-validation will be used in constructing predictive models of treatment response, which will increase the likelihood of these models being generalizable to new patients. This study will be among the first examples of fulfilling the promise of Precision Medicine in providing guidance to patients and families with developmental disorders not about whether to receive intervention but which option for intervention to receive in the context of multiple options. This predict-to-prescribe approach of ASD intervention will likely lead to a paradigm shift in clinical practice and ultimately result in lowering the overall cost and increasing the effectiveness of intervention for children with ASD as individuals.

Safety and Tolerability Trial of Lumateperone in Pediatric Patients With Schizophrenia, Bipolar Disorder or Autism Spectrum Disorder

This is a multicenter, global, 26-week, open-label study to assess the safety and tolerability of lumateperone in pediatric patients with schizophrenia, bipolar disorder or autism spectrum disorder.

Parent-implemented Social Communication Treatment in Autism

An accumulation of research evidence has pointed to parent-implemented communication intervention as effective in reducing the severity of social communication deficits in autistic preschoolers. Despite even high-quality evidence, real-world translation to clinical practice remains challenging, especially for children from lower-income families, for two reasons. First, the intervention outcome is highly variable despite study-level efficacy data, most likely due to unique child and parent factors that make intervention response uneven across individual children. Second, the cost of intervention with the largest effect sizes remains high due to its one-on-one format. With the overarching goal to reduce cost and to increase intervention effectiveness at the individual-child level, this project will conduct a randomized controlled trial (RCT) to compare the effectiveness of two options for intervention to address two specific objectives. The investigators will first ascertain whether parent-implemented communication intervention taught by a speech therapist in a Group format (up to 8 families learning together) is more effective than intervention learnt by the parents themselves (learning the same materials without the guidance of a therapist) at the study level. The investigators will then evaluate what combinations of parent and child behavioral factors determine which format of intervention is likely to be more effective at the individual-child level. It is likely that not all families require the more costly Group format of intervention. Machine learning analytics with cross-validation will be used in constructing predictive models of intervention response, which will increase the likelihood of these models being generalizable to new patients. This study will be among the first examples of fulfilling the promise of Precision Medicine in providing guidance to patients and families with developmental disorders not about whether to receive intervention but which option for intervention to receive in the context of multiple options. This predict-to prescribe approach of autism intervention will likely lead to a paradigm shift in clinical practice and ultimately result in lowering the overall cost and increasing the effectiveness of intervention for autistic children as individuals.

SIT and Deep Pressure Therapy Effects on Motor Coordination in Children With ASD

To evaluate the combined effect of Sensory Integration Therapy and Deep Pressure Therapy on motor coordination and stereotypical behaviors in children with Autism Spectrum Disorder

Transcranial Magnetic Stimulation (TMS) for Autism Spectrum Disorder

Autism spectrum disorder (ASD) is a neurodevelopmental disorder characterized by persistent difficulties in social communication and interaction, as well as restricted and repetitive patterns of behavior. Emerging evidence suggests that the cerebellum plays an important role in social cognition and behavioral regulation, and abnormalities in cerebellar structure and function may contribute to the pathophysiology of ASD.Repetitive transcranial magnetic stimulation (rTMS) is a non-invasive neuromodulation technique that may modulate neural activity and functional connectivity within brain networks associated with ASD. However, the therapeutic effects of individualized cerebellar-targeted rTMS in children and adolescents with ASD remain unclear. This study aims to investigate the efficacy and safety of individualized cerebellar-targeted rTMS guided by magnetic resonance imaging (MRI) in children and adolescents with ASD. Additionally, the study will explore the effects of rTMS on brain structure and functional connectivity. This is a multicenter, single-arm, exploratory interventional study involving 120 participants aged 6 to 18 years who meet the DSM-5 diagnostic criteria for ASD. Participants will receive individualized cerebellar-targeted intermittent theta-burst stimulation (iTBS) guided by MRI-based neuronavigation for two consecutive weeks.Clinical assessments and MRI examinations will be conducted at baseline, week 2, and week 12. Clinical outcomes will be evaluated using the Social Responsiveness Scale (SRS), Repetitive Behavior Scale-Revised (RBS-R), and Strengths and Difficulties Questionnaire (SDQ). Adverse events associated with rTMS will also be monitored. Peripheral blood samples will be collected once at baseline for genetic testing and genotyping to characterize genetic variation among participants. The findings of this study may provide preliminary evidence regarding the feasibility, safety, and potential therapeutic effects of individualized cerebellar-targeted rTMS in children and adolescents with ASD.

Cerebellar Transcranial Direct Current Stimulation (tDCS) in Children With Autism Spectrum Disorder: Raynor Cerebellum Project

The purpose of this research study is to investigate the effects of transcranial direct current stimulation (tDCS) on some of the challenges faced by children with Autism Spectrum Disorder (ASD).

Impact of Equine-Assisted Therapy on the Quality of Life of Children With Autism Spectrum Disorders (ASD)

The main objective of this research is to measure the influence of equine-assisted therapy sessions on the quality of life of children with ASD, using the PedsQL™ 4.0 questionnaire as an evaluation tool. The secondary objectives of this study are to measure the influence of these equine therapy sessions on the quality of life of parents of children with ASD, using the PAR-DD-QOL questionnaire, and to qualitatively analyze the progress of children in terms of communication, motor skills, social connections, etc., during equine therapy sessions, through tracking sheets. This interventional, prospective, and monocentric study aims to evaluate the effect of equine therapy on the quality of life of children with ASD and their families. As a pilot study, it aims to assess the feasibility of a larger-scale study with a rigorous methodology, with the aim of including a control group and conducting a randomized, multicentric, single-blind trial in future studies. It will take place at the La Musse Hospital - La Renaissance Sanitaire, specifically within the equine therapy center of the La Musse facilities. Recruiting 20 children (8-12 years old) with ASD is required for this study. To do this, the inclusion criteria are as follows: participants must be children aged 8 to 12, diagnosed with ASD by a doctor. They must be able to participate in equine-assisted therapy sessions and understand instructions during these sessions. In addition, they must be affiliated with the social security system. The primary and secondary assessment criteria respectively include the average of the two scores obtained from the PedsQL™ 4.0 child and parent report questionnaires; the overall score of the PAR-DD-QOL questionnaire, as well as the qualitative analysis of the equine therapists' tracking sheets. Measures are taken to minimize selection, recruitment, and confusion biases, and specific eligibility criteria are defined. Each participant will benefit from 15 equine therapy sessions, twice a week, over 7 to 8 weeks. Each session will last 60 minutes. The intervention of this study begins with the participant selection process, including an initial contact by phone or during a medical consultation, followed by an oral explanation and the provision of written information to parents and the child. After a reflection period, and if they agree, the parents and the child sign a consent form. The children then undergo 15 equine therapy sessions, including various activities such as emotional expression, contact with the horse, grooming, activities on foot or on horseback, and a debriefing time. Quality-of-life evaluations will be conducted five times during the study using the PedsQL™ 4.0 child, PedsQL™ 4.0 parent report, and PAR-DD-QOL questionnaires (before the first session, after the first session, at the 5th session, at the 10th session, and at the 15th session). Additionally, a tracking sheet will be completed by equine therapists during the sessions. The criteria for interrupting or modifying interventions are established, allowing participants to withdraw at any time for various reasons. Strategies are implemented to improve adherence to rehabilitation protocols, such as regular reminders and the distribution of a schedule of appointments. The individuals concerned are fully informed of the study and their rights. The expected outcomes include an improvement in the average quality-of-life scores of children with ASD, an improvement in the quality-of-life score of their parents, and behavioral progress in the children. The sample size is set at 20 children with ASD, with a detailed statistical analysis plan using Excel and SPSS.

Effect of Social Stories on Anesthesia Compliance in Children With Autism

Children with Autism Spectrum Disorder (ASD) frequently experience heightened anxiety and reduced cooperation during the perioperative period, which can complicate anesthesia induction and negatively affect procedural safety and efficiency. Non-pharmacological behavioral preparation methods that enhance predictability and understanding of medical procedures may improve anesthesia-related cooperation in this vulnerable population. The Social Stories Technique is a structured, visual-based behavioral intervention designed to support children with ASD by explaining upcoming experiences in a clear and developmentally appropriate manner. This prospective controlled study aims to evaluate the effect of the Social Stories Technique on anesthesia compliance and preoperative anxiety in children with Autism Spectrum Disorder undergoing elective surgery. Children in the intervention group will receive Social Stories materials to be reviewed with their families prior to surgery, while the control group will receive standard preoperative information only. Primary and secondary outcomes will include anesthesia induction compliance, preoperative anxiety levels. The findings of this study are expected to contribute evidence regarding the effectiveness of a low-risk, non-pharmacological behavioral intervention to improve perioperative care in children with ASD.

Transdiagnostic Sleep and Circadian Treatment for Autistic Adolescents

Sleep and circadian problems are associated with deleterious social, emotional, and cognitive outcomes, yet are modifiable. This prospective study will adapt and optimize an empirically supported behavioral intervention that addresses common sleep and circadian problems of autistic adolescents, who are at increased risk for mental health disorders, using a transdiagnostic approach. The knowledge gained from this study will help to address a critical need for accessible transdiagnostic sleep interventions for autistic adolescents, who experience a broad range of sleep and circadian problems at high rates and often lack access to specialty care treatment.

Effectiveness of the Autism Pupil Screen (APS) Detect Pupillometer for Identifying Autism Spectrum Disorder (ASD) in Routine Healthcare

Autism spectrum disorder (ASD) is a developmental condition that can affect communication, social interaction, and behavior. Identifying ASD early helps children and families access support services sooner. Pupillometry is a way of measuring the nervous system and brain function by recording the pupil response to light. It is a non-invasive technology that may support screening practices by providing health care providers with an objective physical measure to bolster behavioral screening tools used to identify children with ASD . This study is evaluating how well the APS Detect™ handheld pupillometer works to help screen for ASD in children 2 to 4 years of age during routine pediatric healthcare visits. This investigational device measures the child's pupil response to light and uses these measurements to estimate the likelihood of ASD. Researchers will compare screening results using the APS Detect with the results of reference standard clinical assessments used to determine whether a child has autism. The study will assess how accurately the device identifies children with autism compared to children with typical development based on the Autism Pupil Screen (APS) score. The study will also evaluate whether the device provides the same results when used by different healthcare professionals. Information collected during this study will be used to evaluate device performance, safety, and suitability for use in pediatric healthcare settings and may support future review by the U.S. Food and Drug Administration (FDA).

Impact of an E-Learning Coaching for Parents of Young Children With Autism

The goal of this study is to evaluate the impact of a parental coaching based on E-Learning (EL), intended for parents of children with autism spectrum disorder (ASD). Population: parents of young children (2 to 4 years old) with an ASD diagnosis. The main questions that our study will answer are: * Has parental coaching via E-Learning a significant impact compared to no coaching? * If so, is that impact comparable to standard parental coaching such as PACT? The impact will be evaluated in three domains: * Child development: behavior, sleep, eating behavior * Parental competences: stress, coping, feeling of parental competencies * Parent-child interaction: communication, visual attention The investigators will compare 3 groups: 1. ASD children whose parents will follow a parental coaching via E-learning, 2. ASD children whose parents will follow a standard parental coaching (PACT) 3. ASD children whose parents will not follow any specific coaching program

COMPASS Across Settings (CAST) for Improving Transition Outcomes for Students With ASD

Purpose: The purpose of this project is to develop and test the COMPASS \[Collaborative Model for Competence and Success\] Across Settings (CAST) intervention to enhance the goal setting and attainment skills of autistic youth. Despite federal education law mandating transition services as part of the Individualized Education Program (IEP) for ensuring good outcomes for students with disabilities, current educational practices have been unable to demonstrate that autistic students experience positive postsecondary outcomes. There are existing, evidence-based interventions aimed at supporting positive outcomes for these students. However, these interventions have not systematically provided coaching support to the caregivers, students, and employment specialists. To address these issues, CAST will integrate three evidence-based interventions for supporting student transitions while providing this critical coaching support. By doing so, CAST aims to align the priorities and goals of interventions across home, school, and community settings to better support positive postsecondary outcomes for autistic students.

Word Learning From Parentese in Autistic Children

The overall objective of this research is to determine whether parentese delivered in the video format (Aim 1) and in live interaction (Aim 2) facilitates novel word learning in autistic children and to investigate if there are factors that influence the effect of parentese on word learning (Aim 3).

Evaluation of a Novel Quantum-Inspired Intervention (QII) for Children With Autism Spectrum Disorder: A Global Confirmatory Study

The purpose of this global multicenter confirmatory study is to evaluate the clinical efficacy and safety of Quantum-Inspired Intervention (QII), a novel non-invasive neurodevelopmental protocol, in children diagnosed with Autism Spectrum Disorder (ASD). Autism Spectrum Disorder involves complex neurodevelopmental dynamics characterized by challenges in emotional regulation and adaptive functioning. Existing clinical paradigms primarily rely on pharmaceuticals for secondary symptom control or behavioral methods aimed at external behavioral modification, while interventions targeting core functional shifts remain limited. Grounded in a quantum-inspired conceptual framework aimed at addressing foundational adaptive processing, QII integrates AI-enabled, fine-grained characterization of each child's needs with a proprietary quantum-inspired intervention protocol delivered by trained practitioners. While fully elucidating the fundamental mechanisms will require extended scientific timelines, the urgent needs of individuals and families make confirming tangible, real-world functional efficacy the paramount priority. Preliminary case reports of the QII protocol observed encouraging signals in emotional stabilization and spontaneous communication. Building on these initial findings, this study systematically evaluates the efficacy of QII at scale in an expanded, multicenter cohort. Eligible participants will be randomized to receive either the standardized QII protocol over a defined period or a waitlist control, with the waitlist control arm offered QII following completion of the primary analysis. The primary endpoint evaluates improvement in the autism-related symptom or functional difficulty identified as the primary intervention target at baseline, as measured by the Clinical Global Impression-Improvement (CGI-I) scale; changes in irritability and behavioral dysregulation, as measured by the Aberrant Behavior Checklist-Irritability Subscale (ABC-I), are evaluated as a secondary endpoint.All efficacy endpoints are assessed by independent clinicians blinded to group allocation. Caregivers may report any adverse event or safety concern at any time and may request to stop the intervention at any time; adverse events are also recorded at scheduled study assessments.

Effects of Mounted vs Unmounted Human-Equine Interaction on Autistic Youth

The goal of this clinical trial is to learn how interacting with horses affects autistic children and youth. The main questions it aims to answer are: * How does interacting with horses affect cortisol, heart-rate variability, and electrodermal activity in autistic children and youth? * How does interacting with horses affect parent-reported behaviors in autistic children and youth? Researchers will compare riding a horse, brushing a horse, and brushing a pretend stuffed horse to see if there are different effects on autistic children's physiology and behavior. Participants will * Attend three different horsemanship lessons on three consecutive weeks. One horsemanship lesson will be mounted (riding), one unmounted (brushing), and one with a pretend horse (brushing). * Wear monitors on their chest and wrist * Provide saliva samples

Smith Magenis Syndrome and Autism Spectrum Disorders

Autism Spectrum Disorders (ASD) are a neurodevelopmental disorder. Their prevalence is estimated at around 0.4% of the general population worldwide. Their early onset and chronic nature make them a disabling disorder, all the more so as there is a high prevalence of sleep disorders in these populations, estimated at between 50 and 80%, with many complaints of insomnia in particular. These sleep disorders may result from biological, psychological, social, environmental and family factors. Smith Magenis Syndrome (SMS) is a complex disorder characterized by severe neurological, psychological and behavioral disorders including sleep-wake rhythm disorders. It is a rare disease with a prevalence of 1/25 000. These sleep disorders observed could be the consequence of a general dysregulation of the circadian system, since SMS patients show an inversion of the melatonin secretion profile (with a totally abnormal diurnal peak) and in patients with autism spectrum disorders, an overall reduction in melatonin secretion. These sleep-wake disturbances cycle could play a significant role in learning deficits and in the frequency and severity of behavioral abnormalities observed in SMS and ASD. In this project, investigators propose to study the mechanisms involved in the sleep-wake cycle disorders observed in Smith Magenis and Autism Spectrum children, in particular by evaluating the quality of the pupillary reflex using a pupillometer. The pupillary reflex is a simple and non-invasive method to test light sensitivity and the photobiological mechanisms involved. In this way, investigators want to evaluate the diurnal profile of the pupillary reflex in children with Smith Magenis syndrome and with Autism Spectrum Disorders in relation to the diurnal melatonin profile. Investigators will complete this study by determining the chronobiological profile of these patients by measuring different variables: * Diurnal cortisol and amylase profile * 24h body temperature and heart rate profile * Urinary cortisol and 6-sulfatoxymelatonin (major metabolite of melatonin) profiles * Daytime sleepiness profile measured subjectively by questionnaire and objectively via a waking EEG recording. * Actimetry at home * Polysomnography * A neurocognitive and behavioural assessment

Psilocybin for Treatment-Resistant Depression in Autism

We propose a first-of-its-kind open-label clinical trial to investigate the feasibility, tolerability, and safety of administering psilocybin in autistic adults with treatment-resistant depression (TRD). In this study, 20 participants (intellectually able and fluent-speech adults) with autism and co-occurring TRD will receive manualized psychotherapy that has previously been used with psilocybin (Agin-Liebes et al., 2020). They will also receive psilocybin at 2 different time points, firstly a safety dose of 10mg, followed by a treatment dose of 25mg, separated by a week. This study design is in accordance with previous studies investigating the use of psilocybin with psilocybin-assisted therapy (PAT) to treat TRD (Carhart-Harris et al., 2016, 2018)

Strategies for Toddler ASD With Remote-therapy (STAR) Model for ASD

The project aims to develop and implement the Strategies for Toddler Autism spectrum disorder(ASD) with Remote-therapy (STAR) model for early toddlers with ASD in local community sites in Shanghai. It's a randomized control trial lasting 3 years. The children will be divided into 2 groups, one for STAR intervention group and the other for community intervention group. The STAR intervention group will be performed STAR other than community intervention.

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)

Evaluation of Effectiveness of Child-oriented Goal-setting in Paediatric Rehabilitation (the ENGAGE Approach)

Children with disabilities often access rehabilitation services to improve their abilities to participate in everyday activities. Goal-directed therapy is considered an important therapeutic strategy to achieve outcomes that are meaningful to families. Not a lot is known about the effects of goal setting on rehabilitation outcomes. Strategies to help children participate in the goal-setting process are rarely used in clinical practice. The aim of this project is to test the effects of a child-focussed goal setting approach, Enhancing Child Engagement in Goal Setting (ENGAGE), on therapy outcomes. Service use and the cost vs. benefits of the ENGAGE approach compared to usual practice will also be examined. Children with neurodevelopmental disabilities aged 5-12 years old (n=96) who access paediatric rehabilitation services at six rehabilitation sites will participate. Therapists (n=24) at participating sites in Alberta, Canada will be randomized into 1) the ENGAGE intervention group or 2) the usual therapy practice control group. Children will participate in the ENGAGE approach to goal setting or usual practice based on the allocation of their therapist. This study will determine if the ENGAGE approach to goal setting affects child goal performance, satisfaction with goal performance, functional abilities, participation, and parent and child quality of life. The investigators will also evaluate differences in parent and child quality of life in relation to parent costs (e.g., absenteeism, presenteeism, travel costs) and compare amount of therapy time between the two groups to see which approach is more cost-effective and efficient. After the study, children, parents and therapists will be asked to discuss aspects that influenced effective implementation of the ENGAGE approach. This study could provide evidence to improve meaningful child and family outcomes in paediatric rehabilitation and improve efficiency of paediatric rehabilitation services.

AWARE: Management of ADHD in Autism Spectrum Disorder

This study is a pragmatic clinical trial examining the comparative effectiveness of two stimulant medications (methylphenidate and amphetamine) in the treatment of ADHD in children and adolescents with autism. Using a sequential, multiple assignment randomization trial (SMART) design the study will not only assess these two medications but also the role of an increasingly popular class of ADHD medication, the alpha-2 agonists. Findings from this study will help improve clinicians' approach to medication selection and reduce the repeated trials of multiple medications that are current standard care.

DOD Regulating Together Intervention

Our long-term goal is to validate Regulating Together (emotion regulation intervention) and improve psychosocial outcomes for youth with autism spectrum disorder and emotion dysregulation.

Pai.ACT: AI-Driven ACT Chatbot for Mental Health Triage and Service Evaluation

Parents of children with special needs in Hong Kong often face limited psychological support, which can negatively impact the child rehabilitation process and the well-being of parent-child relationships. To address this gap, we have developed Pai.ACT, the first deep learning-based mental health advisory system for parents. Pai.ACT features an AI chatbot that integrates the counselling principles of Acceptance and Commitment Therapy (ACT) through natural language processing, providing parents with a human-like voice-to-text experience. Using data from chatbot interactions, the Pai.ACT platform offers assessments regarding the individual's psychological inflexibility status and delivers stratified mental health interventions by: * Low-risk: Users access self-help ACT digital modules tailored to their specific psychological inflexibility processes. * Moderate-risk: In addition to the self-help modules, users receive 4-6 sessions of video-conferencing-based ACT interventions (45-60 minutes per session) conducted by our trained counseling team. * High-risk: Users are directed to specialized mental health services provided by collaborating units. The study includes a regional randomised controlled trial (RCT) in Hong Kong's Sha Tin District, in collaboration with the Shatin District Office. The goal of this regional study is to evaluate the feasibility, acceptability, and potential efficacy of combining AI-driven mental health support across all of Hong Kong. Focus group interviews will also explore parents' perceptions of Pai.ACT and help identify the most effective service model for scaling its use. Pai.ACT provides accessible and comprehensive mental health services to Chinese-speaking parents, helping to alleviate the psychological burden of caregiving. By integrating mental health support with child rehabilitation services and non-governmental organisations, Pai.ACT has the potential to enhance family caregivers' well-being, reduce stigma associated with special needs children, and promote more significant mental health awareness in Chinese-speaking communities.

Building Resilience and Appropriate Independence in Young Adults With Autism

This study evaluates the effects of a 12-week life-skills resilience curriculum for young adults with autism. The project utilizes a cohort single arm design.