Clinical Research Directory

Smart Inhaler and Remote Asthma Management in Adolescents With Intellectual and Developmental Disabilities

Correct use of daily medications containing inhaled corticosteroids is key for asthma control, yet children with intellectual and developmental disabilities (IDD) face additional barriers to proper inhaler use. Smart inhalers, a novel technology that provides guidance and immediate feedback on inhaler use techniques, have been shown to enhance correct medication administration in the typically developing pediatric population, but their effectiveness has not been evaluated on the pediatric IDD population for remote home use. This study aims to investigate whether daily application of smart inhalers (1) is feasible, acceptable and usable in the IDD population for remote asthma management, (2) improves the rate of medication use and correct medication administration, and (3) results in improvement in lung function. This effort aims to promote better asthma management in the IDD population for remote home use.

Pepea Pamoja: Cluster-Randomized Trial for Caregivers of Children With Autism in Kenya

Pepea Pamoja is a ten-session, group-based caregiver intervention designed to improve caregiver well-being and strengthen caregivers' ability to support communication and positive behavior in children with autism. This study will enroll approximately 240 caregivers of children ages 2-8 years in western Kenya. Caregiver groups will be randomized to one of three conditions: standard-of-care control, professional-led Pepea Pamoja, or peer-led Pepea Pamoja. The study will determine whether each Pepea Pamoja delivery model improves caregiver and child outcomes compared with control and will directly compare peer-led and professional-led delivery. Outcomes will include caregiver stress and well-being, child communication and behavior, and blinded observations of child social communication and caregiver-child interaction. An embedded mixed-methods evaluation will examine implementation, sustainability, program and societal costs, and cost-effectiveness.

Pediatric Patients With Metabolic or Other Genetic Disorders

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.

Probiotics for Autism Spectrum Disorders: a Randomized Controlled Trial (The PASD Study)

Autism spectrum disorders (ASD) are a group of severe neurodevelopmental conditions characterized by impaired communication and social interaction, as well as repetitive/stereotyped behaviors deriving from a combination of genetic and environmental factors. The ASD diagnosis rates increased dramatically over the past number of decades. The Diagnostic and Statistical Manual of Mental Disorders (DSM-5) describes a worldwide prevalence of approximately 1%. The prevalence of ASD is 1 in 59 individuals in the US reported by the Centers for Disease Control and Prevention. According to the latest data from the Italian National Observatory for ASD, the actual prevalence in Italy is about 1/77 for children aged between 7 and 9 years, with a 4.4 times higher prevalence in male. The origin of ASD is still largely undefined. It has been hypothesized a possible role for the influence of early life alteration of gut microbiome (GM). We demonstrated an imbalance in Bacteroidetes and Firmicutes phyla with a decrease in Bacteroidetes/Firmicutes ratio in the GM of pediatric patients affected by ASD. Similar data have been observed by others. Data from ASD animal model confirm the presence of GM dysbiosis with significant correlation with behavioral, gastrointestinal and immunologic alterations. Altogether these data support the hypothesis that GM dysbiosis could be involved in the ASD pathogenesis. The ASD children present an increased prevalence of functional gastrointestinal disorders (FGIDs), mainly chronic constipation, functional diarrhea, and irritable bowel syndrome (IBS). A role for GM has been suggested also for these conditions. The presence of these disorders negatively influence the disease severity and the parental quality of life of ASD children. Starting from all these considerations GM is becoming a possible target of intervention for pediatric ASD. Probiotics are one of the most investigated strategy for a beneficial modulation of GM. Probiotics are commonly defined as live microorganisms which when ingested in adequate amounts confer a beneficial effect on the host. The most used probiotics in the pediatric age are Saccharomyces and Lactobacillus strains including Lactobacillus rhamnosus GG (LGG). Data report a beneficial influence elicited by LGG on GM structure and function. This probiotic resulted also effective in treating FGIDs patients. Preliminary evidence suggest the potential efficacy of probiotics for FGIDs treatment in ASD children. Altogether these evidence strongly support the hypothesis that LGG could exert a beneficial action in ASD children. The purpose of this study is to evaluate the therapeutic efficacy of LGG on FGIDs in ASD children.

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.

Social Information Processing in Children: an ocUlo-pupillometric Tool for Standard Evaluation

The goal of this observational study is to identify behavioral and physiological markers of social alterations in Autism Spectrum Disorders compared to Typically Developping peers. This single-centre study plan to use eye tracking to evaluate social disorders in Autism Spectrum Disorders (ASD). The investigators will estimate oculometric and pupillometric indices as potential ASD biomarkers. The study will last 4 years and will be organized in two phases.

Structured Sensory-Based Occupational Therapy in Children With Autism

This controlled clinical study will investigate the effects of a 12-week structured sensory-based occupational therapy intervention on occupational performance, sensory processing, gross motor development, and eating behaviors in children with autism spectrum disorder. Forty children aged 3-9 years will be assigned to either an intervention group receiving face-to-face occupational therapy twice weekly or a control group receiving an individualized home program.

Evaluation and Refinement of KF-STRIDE Into the Interview!

Examine the acceptability, usability, feasibility, and initial efficacy of KF-STRIDE 3.0, the data from which will serve as a basis for a future fully powered RCT

Exercise Interventions for Children With Autism Spectrum Disorder

The purpose of this clinical trial is to find out whether exergame and aerobic exercise can improve sleep, movement skills, thinking abilities, and brain activity in children with autism spectrum disorder (ASD). This study aims to answer the following questions: 1. Can exergame and aerobic exercise improve sleep, movement skills, and thinking abilities in children with ASD? 2. Can exergame and aerobic exercise change brain activity in children with ASD? Researchers will compare three different intervention programs: 1. Exergame combined with motor and behavioral intervention. 2. Aerobic exercise combined with motor and behavioral intervention. 3. Motor and behavioral intervention only. By comparing these three groups, researchers hope to determine whether adding exergames or aerobic exercise provides additional benefits for children with ASD. Participants will: 1. Attend one intervention session each week for 8 weeks. Depending on the group they are assigned to, they will receive either exergames combined with motor and behavioral intervention, aerobic exercise combined with motor and behavioral intervention, or motor and behavioral intervention only. 2. Complete a home exercise program twice a week for 12 weeks. 3. Visit the laboratory three times for assessments: before the intervention begins, at the end of Week 8, and at the end of Week 12.

Dialectic Behaviour Therapy (DBT) for People With Autism: A Multiple Baseline Single Case Design

The goal of this experimental study is to test the effectiveness of Dialectic Behavioural Therapy in patients with Autism Spectrum Disorder. The aim questions are: 1. What is the effect of DBT on maladaptive emotion regulation strategies and emotional well-being in patients with ASD? 2. Is the use of DBT skills associated with a reduction in maladaptive emotion regulation strategies and an increase of emotional well-being over time? 3. What are the dynamics over time between maladaptive emotion regulation strategies, emotional well-being and sensory overload? Participants will: * Follow DBT of 10 months * Keep a diary on their urge of maladaptive emotion regulation strategies and well-being

Effects of Lactobacillus Plantarum PS128 in Children With ASD

Autism spectrum disorder (ASD) is a neurodevelopmental disorder that can cause significant social, communication and behavioral deficits. Probiotics are regarded as active microorganisms. With sufficient amounts, probiotics can regulate intestinal flora, intestinal permeability, inflammation and antioxidant reactions in the body. These reactions may further promote health, regulate metabolic disease progression and prevent complications. Lactobacillus plantarum PS128 (PS128) was reported to be a psychobiotic in several animal and human studies which modulated the levels of neurotransmitters in different brain areas. This study is to evaluate whether the consumption of PS128 can improve the symptoms of patients with ASD. The current randomized, placebo-controlled trial was conducted to investigate the psychophysiological effects of PS128 in school children with ASD.

Telecoaching Intervention for Children With Autism and Their Parents in Palestine

Families of children with autism in Palestine may have difficulty accessing early intervention and rehabilitation services because specialized services are limited and families may face geographical, financial, and other barriers. Providing support to parents remotely may help families access professional guidance without needing to travel for every session. This pilot study examined whether an eight-week parent telecoaching program called Occupational Performance Coaching (OPC) could be delivered successfully and was acceptable to parents of young children with autism in Palestine. The study also explored changes in children's participation in everyday activities and autism-related characteristics, as well as parents' confidence in supporting their child and their quality of life. Because this was a small pilot study, these outcomes were explored to help plan future research rather than to determine whether the intervention was effective. Parents were assigned to one of two groups. Parents in the telecoaching group received eight individual online coaching sessions over eight weeks, with each session lasting approximately 40-60 minutes. Coaching helped parents identify activities that were important to their child and family, understand difficulties affecting the child's participation, develop possible strategies, try these strategies during everyday routines, and review progress with the coach. Parents in the waitlist group had access to general Arabic-language information and videos about autism through the Sanad Alhayaa digital platform during the eight-week comparison period but did not receive individual coaching. After completing the post-intervention assessment, they were offered the telecoaching program. The study also included interviews with parents who received telecoaching to better understand their experiences, perceived benefits, and barriers to using this form of support.

Movement-Based Digital Game Intervention for Children With Autism Spectrum Disorder

The goal of this study is to learn if movement-based digital games can help improve motor skills in children aged 6-10 years with Autism Spectrum Disorder (ASD). The main question this study aims to answer is: \* Do children who take part in movement-based digital games show greater improvement in motor skills than children who continue their usual education and rehabilitation program? Researchers will compare 10 children who take part in the movement-based digital game program with 10 children who continue their usual education and rehabilitation program. Participants in the movement-based digital game group will: * Play movement-based digital games twice a week for 10 weeks. * Take part in game sessions lasting about 60 minutes. * Complete motor skill assessments before, during, and after the program. Participants in the control group will continue their usual education and rehabilitation program and complete the same motor skill assessments at the same time points.

Addressing Disparities in Autism Spectrum Disorder Diagnosis Using a Direct-to-home Telemedicine Tool

The investigators propose to evaluate the use of a telemedicine tool, the TELE-ASD-PEDS (TAP), that is designed to assess for autism spectrum disorder (ASD) symptoms in toddlers. The TAP was developed at VUMC by a team of clinical psychologists with expertise in the early identification of ASD. The TAP has been studied in controlled laboratory settings, with high levels of family and clinician satisfaction, as well as excellent agreement with blinded comprehensive ASD evaluation. The TAP has also been used to complete direct-to-home telemedicine assessments during the COVID-19 pandemic. However, the investigators have not yet compared direct-to-home assessments using the TAP with gold standard, in-person ASD assessments. It has also not yet been studied in a diverse sample of families or with providers outside of VUMC. This study will allow the investigators to address those gaps.

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.

Social Robots and Board Games for Children With Autism Spectrum Disorder

This pilot randomized controlled trial evaluates the effectiveness of combining social robot-assisted intervention with physical board games to improve socio-emotional skills in children with Autism Spectrum Disorder (ASD). Children with ASD often face challenges in emotion recognition, emotional regulation, and social interaction. This study aims to investigate whether integrating an interactive social robot (Kebbi) into board game activities can enhance children's social engagement, emotional comprehension, and cooperative behaviors compared to conventional occupational therapy. Participants will be randomly assigned to either the robot-assisted board game group or the conventional occupational therapy group.

Developing Brain, Impulsivity and Compulsivity

Background: Impulsivity is acting 'without thinking.' Compulsivity is being overly inflexible. People vary in how impulsive or compulsive they are. Extreme versions of these behaviors play a role in mental disorders. Researchers want to study changes in the brain to learn more about these behaviors. Differences in genes may also play a role. Objective: To learn about genetic \& brain features that explain why levels of impulsivity and compulsivity vary across people. Eligibility: People ages 6 - 80 Design: Participants will be screened with a medical history and medical record review. Participants will talk about their mental and behavioral development. They may discuss topics like drug use and sexual activity. They will complete surveys about their compulsivity and impulsivity. Parents of child participants may also complete these surveys. Participants may take memory, attention, and thinking tests. They may give blood or saliva samples for gene studies and they may give blood to make induced pluripotent stem cells. Participants may have their face and irises photographs taken. Participants may have a magnetic resonance imaging scan. It will take pictures of their brain. The scanner is shaped like a cylinder. Participants will lie on a table that slides in and out of the scanner. A coil will be placed over their head. They will lie still, watch a movie, and play a game. Participants may ask family members to join the study. Researchers are particularly interested in recruiting twin pairs to the study. Participants under age 25 may repeat these tests every 1-2 years until they turn 25 or until the study ends. For those over age 25, participation will last less than 1 month.

COMpassion for Psychiatric Disorders And Self-Stigma

People with mental disorders face frequent stigmatizing attitudes and behaviors from others . In response to this, they tend to isolate themselves, with the risk of impeding care and the process of recovery and integration into society . Stigmatization can also be assimilated by patients themselves - i.e. self-stigma. Self-stigma is involved in diminished coping skills that lead to social avoidance and difficulties in adhering to care . Reducing self-stigma and its emotional corollary, shame, is thus crucial to attenuate the disability associated with mental illness. Shame is inherent to self-stigma and leads to difficulties in adhering to care as well as greater severity of clinical presentations . Compassion Focused Therapy (CFT) is a third wave cognitive behavioral therapy that targets shame reduction and hostile self-to-self relationship and allows for symptom improvement while increasing self-compassion, a major resilience factor . Although shame is a prominent part of the concept of self-stigma, the efficacy of CFT has never been evaluated in individuals with high levels of self-stigma. In this study, the investigators will evaluate the efficacy and acceptability of a group based CFT program on decreasing self-stigma, compared to treatment as usual (TAU) and a psychoeducation program whose efficacy has been assessed in a previous trial.

Prednisone in Adults With an Immune-Mediated Subtype of Autism Spectrum Disorder

The goal of this clinical trial is to learn how prednisone affects adults with autism spectrum disorder (ASD). It will also learn about the safety of prednisone. The main questions it aims to answer are: * How does prednisone affect the core features and associated target symptoms of ASD in adults with an immune-mediated subtype of ASD? * Is prednisone safe for autistic adults without causing too many side effects? * Does this study warrant larger trials studying anti-inflammatory drugs in this subject population? Researchers will compare the drug prednisone to a placebo (a look-alike substance that contains no drug) to see how prednisone affects autistic adult males. Participants will: * Visit the clinic 2 times for a screening and baseline visit. * Take prednisone or a placebo every day for 16 weeks. * Visit the clinic 2 times for checkups, tests, questionnaires, and dose changes, and 1 time for a follow-up visit 4 weeks after stopping the study drug. * Provide blood and urine samples for testing up to 4 times. * Complete 8 remote calls every 1-2 weeks for checkups and dose changes. * Keep a diary of the dose and times they take the study drug every day and any symptoms or side effects they experience.

Physiological Mechanisms of Therapeutic Horseback Riding Intervention Effects in a Psychiatric Population of ASD Youth

This randomized control trial (RCT) seeks to assess the mechanisms underlying Therapeutic Horseback Riding's (THR) previously observed significant positive effects on ASD youth, particularly those with co-occurring psychiatric disorders, and to refine information on the durability, dose and sub-population effects of the intervention.

Pediatric Massage for Children With Autism Spectrum Disorder

This study was designed as a randomized controlled clinical trial of pediatric massage for children with Autism Spectrum Disorder (ASD). 72 ASD children will be recruited and randomly divided into two groups: the treatment group (pediatric massage + usual care, n=36) and the control group (usual care/waitlist group, n=36).30 healty controls will be recruited. Children in the treatment group will received 12 weeks pediatric massage by doctors and their own parents. The Autism Behavior Checklist (ABC), Childhood Autism Rating Scale (CARS), Autism Treatment Evaluation Checklist (ATEC), Social Responsiveness Scale-Second Edition (SRS-2), Children's Sleep Habits Questionnaire (CSHQ) and Eysenck Personality Questionnaire (EPQ) will be used to assess the clinical behavioral changes of all participants, and to analyze the clinical effectiveness and safety of pediatric massage for children with ASD. Electroencephalography (EEG) and brain functional near-infrared spectroscopy (fNIRS) will be recorded before and after treatment, to observe the potential brain target of pediatric massage for children with ASD.

Using Apollo Neuro in Autistic Children With Self-Injurious Behavior

This study will evaluate the feasibility and acceptability of the Apollo Neuro, a wearable vibrotactile sensory device, in autistic children who engage in self-injurious behavior (SIB). Participants will wear the device for at least 3 hours per day over a 30-35 day period with caregiver support. Outcomes will include adherence to device use, caregiver-reported feasibility and acceptability, and descriptive characterization of caregiver-reported self- injurious and repetitive behaviors during the study period. This preliminary, single-group study is not designed to evaluate efficacy and will inform the design of future controlled trials.

Testing an Evidence-Based Supported Employment Model in Autistic Young Adults

This study aims to enhance employment outcomes for young adults with autism and intellectual and developmental disabilities (IDD) through the implementation of an evidence-based supported employment model known as Individual Placement and Support for Autism (IPS-AUT). The study will evaluate the feasibility, acceptability, and effectiveness of IPS-AUT in promoting Competitive Integrated Employment (CIE). The trial will involve partnerships with supported employment agencies, training providers in IPS-AUT, and assessing employment outcomes and implementation factors. The ultimate goal is to create a scalable, evidence-based employment support model for individuals with autism.

The Effect of Exergame-Supported Training on Reaction Time and Agility

This study looks at whether a type of exercise video game, called an exergame, can help adolescent boys with autism spectrum disorder (ASD) who play table tennis react faster and move more quickly (agility) during play. Twenty-four adolescent male table tennis players with mild ASD are taking part. Half of them add 20 minutes of exergame training, using a light-based reaction device, to their usual table tennis practice twice a week for 8 weeks. The other half continue their usual table tennis practice only, without the exergame. Reaction time is measured using a light-based reaction device, and agility is measured using a standard running drill (the T-test), before and after the 8-week period. This study aims to find out whether exergame training added to regular practice can help young table tennis players with ASD react and move faster during matches.