Neurodevelopmental disorder

Rett syndrome is a rare genetic disorder that mostly affects girls and stems from mutations in the MECP2 gene. This trial tests GCB-002, an experimental gene therapy given as a single injection into the spinal fluid, in 6 to 9 girls aged 2 to 10 who have a confirmed MECP2 mutatio…

Researchers are testing a new enzyme replacement therapy called GNR-055 in people with Hunter syndrome (MPS II), a rare inherited disorder that causes harmful sugar molecules to build up in the body. The trial enrolls about 32 patients, including those who have never had enzyme t…

This study tests whether the drug LP352 can safely reduce seizures in children and adults with Dravet syndrome, a severe form of epilepsy. About 160 participants will receive either LP352 or a placebo, and researchers will track changes in seizure frequency over several months. T…

This study tests an experimental drug called LP352 to see if it can safely reduce seizures in children and adults with developmental and epileptic encephalopathies (DEE), a group of severe epilepsy syndromes. About 320 participants will receive either LP352 or a placebo, and rese…

This study tests a brain-responsive neurostimulation system in 24 people aged 12 and older with Lennox-Gastaut syndrome whose seizures are not controlled by medication. The device detects and responds to seizure activity in the brain to reduce drop seizures. The goal is to see if…

This phase 3 study is testing the long-term safety and effectiveness of LP352 (Bexicaserin) in 324 children and adults with developmental and epileptic encephalopathy (DEE), including Dravet and Lennox-Gastaut syndromes. Participants who completed earlier studies will receive LP3…

This study tests two approaches—lifestyle medicine consultations and cognitive behavioral therapy (CBT)—to reduce cardiovascular risk in autistic individuals aged 9 to 26 who have a high body mass index. Participants are randomly assigned to receive lifestyle coaching, CBT sessio…

This study tests whether a probiotic called Lactobacillus plantarum can improve social interactions, reduce inflammation, and enhance quality of life in adults with autism spectrum disorder. Twelve participants take the probiotic for 8 weeks, with assessments including blood test…

This early study tests the safety of TheraSphere PCa, tiny radioactive glass beads injected into the prostate to treat localized prostate cancer. The trial involves 36 men with favorable intermediate-risk cancer and aims to find the maximum safe radiation dose. Researchers will m…

This study follows about 200 people with Prader-Willi syndrome who are taking or starting VYKAT XR. Researchers will track side effects and how the drug affects their health over time. The goal is to gather more safety information, not to test if the drug cures the condition.

This study tests a one-time gene therapy called TSHA-102 in girls aged 2 to 4 with Rett syndrome, a severe genetic disorder. The therapy is given as a single injection into the spine. The main goal is to check if it is safe and tolerable, and to see early signs of whether it help…

This study tests whether a daily probiotic supplement can improve the balance of gut bacteria in children aged 3 to 7 who have autism and digestive issues like constipation or stomach pain. Over 6 months, 60 children will receive either the probiotic or a placebo. The main goal i…

This study tests an investigational drug called relutrigine in 160 children with a severe form of epilepsy called developmental and epileptic encephalopathy (DEE). The goal is to see if the drug can safely reduce monthly seizure frequency compared to a placebo. After the initial …

This trial is testing an experimental drug called PRAX-222 in children aged 2 to 18 who have a rare, severe form of epilepsy caused by changes in the SCN2A gene. The goal is to see if the drug is safe and can reduce how often seizures happen. Participants will receive either PRAX…

This early-stage trial tests a new gene therapy called FRF-001 for FOXG1 syndrome, a rare genetic disorder that affects brain development. The therapy is given as a single injection into the brain's fluid spaces. The study will include 12 children and adults and will check for sa…

This trial investigates the long-term safety of ecopipam, a drug that blocks dopamine D1 and D5 receptors, in children, adolescents, and adults with Tourette syndrome. Participants who completed a prior study may enroll and receive ecopipam for up to 24 weeks or longer. The main …

This study is testing the safety of an experimental drug called ML-004 in 120 adolescents and adults with autism spectrum disorder who have already completed a previous study. Participants will take ML-004 once daily, and researchers will monitor for side effects. The goal is to …

This early-stage trial is testing a new drug called IDE161, alone or with the immunotherapy pembrolizumab, in adults with advanced solid tumors (like breast, ovarian, prostate, or lung cancer). The main goals are to check safety, find the right dose, and get a first look at wheth…

This study tests a program called PIXI that coaches parents of infants diagnosed with rare neurogenetic disorders (such as Fragile X, Angelman, or Down syndrome) during the first year of life. The program combines education about the disorder, guided parent-child interaction acti…

This study tests a one-time gene therapy called TSHA-102 in females aged 6 to under 22 with classic Rett syndrome. The goal is to see if it is safe and can help them regain or gain new developmental skills like walking or talking. Participants receive the therapy through a spinal…

This phase 3 trial tests whether the drug troriluzole can slow the progression of spinocerebellar ataxia, a rare genetic disorder that affects coordination and balance. About 300 adults with different types of SCA are randomly assigned to take either troriluzole or a placebo dail…

This study compares two ways of fortifying breast milk for preterm babies in the NICU. One group gets standard fortification, while the other gets a personalized mix based on real-time milk analysis. Researchers will track growth, brain scans, and development up to age 2 to see w…

This study tests a gene therapy called NGN-401 in 33 girls with Rett syndrome, a rare genetic disorder that affects development and movement. The therapy delivers a working copy of the MECP2 gene to try to improve skills and daily function. Researchers will measure success by whe…

This phase 2 trial tests whether lamivudine, an antiviral drug, can improve symptoms in people with Rett syndrome, a rare genetic disorder that affects movement, communication, and behavior. Ten participants will take the drug by mouth for a set period, and researchers will measu…

This study looks at whether the drug troriluzole can slow the progression of spinocerebellar ataxia (SCA), a rare genetic disease that affects movement and balance. Researchers will compare 909 patients who took troriluzole for up to three years with similar patients who did not …

This early-stage study tests a gene therapy called ETX101 in 5 children with Dravet syndrome, a severe form of epilepsy. The therapy delivers a gene that helps calm overactive brain cells. Researchers will check if it safely reduces seizure frequency and improves thinking and dai…

This study planned to test the drug bumetanide in children aged 5 to 17 with autism of known cause. The goal was to see if it could improve autism symptoms. However, the trial was withdrawn before enrolling any participants, so no new information was gathered.

This study follows about 111 people with Lennox-Gastaut syndrome, Dravet syndrome, or tuberous sclerosis complex who are taking Epidyolex (a CBD oral solution) for seizures. Researchers track how many stay on the drug for a year and how many seizure-free days they have. It's an o…

This early-phase trial tests a single injection of AAV-MECP2 gene therapy into the spinal fluid of 8 girls aged 4-10 with Rett syndrome, a severe neurodevelopmental disorder. The goal is to see if the treatment is safe and can reduce disease severity. The study is not yet recruit…

This study tests whether a structured parent training program can reduce autism symptoms in children up to age 5 and high-risk infants under 2. 250 children and their caregivers will be randomly assigned to receive either the training plus routine care, or routine care alone. Aft…

This study tests a telehealth program that teaches parents of children with autism and overweight or obesity how to help their child manage weight through nutrition, activity, and behavior skills. About 140 families will take part, with one group receiving the parent-based progra…

This study tests whether a mix of treatments—including the patient's own stem cells, a strict keto diet, and a fecal transplant—can improve social skills, communication, and gut health in 50 teens and adults with autism. Participants must be stable on their current medications. T…

This Phase 3 study tests whether fenfluramine (ZX008) can safely reduce seizures in 87 children and adults with CDKL5 deficiency disorder, a rare genetic condition causing severe epilepsy. Participants receive either the drug or a placebo, followed by an open-label extension wher…

This study tests whether leucovorin calcium, a vitamin-like supplement, can improve language and other symptoms in young children with autism spectrum disorder. Researchers will enroll 80 children aged 2.5 to 5 years with language delays. The trial lasts about 26 weeks and compar…

This study offers fenfluramine to people with Dravet syndrome whose seizures have not improved with other medications. The goal is to provide access to the drug and monitor its safety. Participants must have a genetic diagnosis of Dravet syndrome and have tried all available seiz…

This pilot study tests whether deep brain stimulation (DBS) of specific brain pathways can safely reduce seizures in people with Lennox-Gastaut syndrome, a severe form of epilepsy. Five participants receive the electrical stimulation as an add-on to their usual treatment. The goa…

This study follows 25 people aged 2 to 65 with cerebral palsy or brain damage from lack of oxygen who have already received a new gene therapy given as a nose spray. The therapy delivers 15 genes meant to repair the brain, reduce inflammation, and improve movement and thinking. R…

This study tests a drug called CM-AT in children with autism who have low levels of a digestive enzyme. The goal is to see if the drug is safe and helps with behaviors like irritability and social withdrawal over a longer period. About 405 children aged 3 to 8 who completed a pre…

This phase 3 trial is testing the safety of pitolisant in 150 people with Prader-Willi syndrome who have already taken the drug in a previous study. Participants will receive pitolisant tablets and be monitored for side effects. The goal is to see if pitolisant is safe for long-t…

This phase 2 trial tests whether clemastine, a common allergy medication, can improve thinking, motor skills, and social abilities in people with Williams syndrome. The study includes 30 participants aged 6 to 30 and uses a double-blind, placebo-controlled design to measure safet…

This phase 2 trial tests the drug tiratricol (Triac) in 22 young boys (up to 30 months old) with MCT8 deficiency, also known as Allan-Herndon-Dudley syndrome. The goal is to see if the drug can improve brain development and reduce harmful thyroid effects in the body. Participants…

This early-stage trial tests whether metformin, a common diabetes drug, is safe and feasible for infants who suffered brain injury from lack of oxygen at birth. Thirty infants under 6 months old will receive metformin for 6 weeks, with close monitoring of kidney and liver functio…

This phase 2 trial tests setmelanotide (Imcivree), a daily injection, in 18 people aged 6 to 65 with Prader-Willi syndrome who have obesity. The study will check if the drug is safe and helps reduce body weight and hunger over 52 weeks.

This study looks at the long-term safety of an experimental drug called zorevunersen (STK-001) in 60 people with Dravet syndrome, a severe form of epilepsy. Participants must have completed an earlier STK-001 study. Researchers will monitor side effects, seizure frequency, and qu…

This study tests a new medicine called PRAX-562 in 77 children with rare genetic epilepsies (SCN2A or SCN8A). The goal is to see if it safely reduces motor seizures. The trial has two parts: a double-blind phase where some children get the drug and some get a placebo, followed by…

This program provides expanded access to the drug stiripentol for people aged 6 months and older with Dravet syndrome, a severe form of epilepsy. The goal is to help control seizures that do not respond to other treatments. Participants must have a confirmed diagnosis and meet sa…

This early-phase trial is testing whether glycerol phenylbutyrate (Ravicti), a drug already approved for a different condition, is safe and tolerable for children with genetic disorders like STXBP1 and SLC6A1 that cause seizures and developmental delays. The study will enroll 50 …

This study tests a gene therapy called ETX101 in 4 children with Dravet syndrome, a severe seizure disorder caused by a gene change. The therapy uses a harmless virus to deliver a working copy of the gene to brain cells. The main goals are to see if it is safe and if it reduces s…

This study tests a new gene therapy called TSHA-102 for girls aged 5 to 8 with Rett syndrome, a severe genetic disorder that affects brain development. The therapy is given as a single injection into the spinal fluid. The main goal is to check safety and side effects, while also …

This study tests a custom-made drug called an antisense oligonucleotide (ASO) designed specifically for one person with a rare genetic brain disorder (NEDBA) caused by a MAPK8IP3 mutation. The goal is to see if the drug can improve motor skills and reduce seizures over 12 to 24 m…

This program provides continued access to the seizure medication perampanel (Fycompa) for people who completed certain earlier studies and are still benefiting from the drug. It runs until the drug is commercially available in their country or no participants remain. Only those a…

This study offers voluntary screening for newborns in North Carolina to detect a wide range of rare health conditions early. Using a small blood sample already collected at birth, the program tests for dozens of disorders, including spinal muscular atrophy, cystic fibrosis, and m…

This study is testing whether artificial intelligence can analyze a person's speech to help diagnose mental health conditions like depression, anxiety, autism, and schizophrenia. Researchers will record 500 people speaking and compare the AI's predictions to standard clinical dia…

This study tests a virtual behavioral therapy (Functional Behavioral Training) for children aged 2-12 with genetic syndromes like Fragile X, Angelman, or Rett syndrome who have challenging behaviors. The therapy teaches parents how to identify what triggers problem behaviors and …

Researchers are testing whether occupational therapy that uses virtual reality can improve cognitive skills, daily performance, and motivation in children with autism. The study includes 32 children aged 6 to 12 with autism level 1. Half receive virtual reality-supported cognitiv…

This study compares cognitive behavioral therapy (CBT) with online expert coaching to usual care for children aged 6-14 with autism who struggle with anxiety, irritability, or emotional outbursts. About 100 children and 50 therapists will take part. The goal is to see if the coac…

This trial tests a structured, group-based training program designed to improve executive function skills in elementary school children aged 9 to 12 with autism spectrum disorder. Over 12 weeks, children attend weekly sessions that teach planning, organization, time management, e…

This study tests a behavioral sleep and circadian intervention for 50 school-aged children (6–12 years) with autism who have sleep problems. Parents receive 5 Zoom sessions over 12 weeks to learn strategies to improve their child's sleep. The goal is to see if the program is feas…

This early-stage study tests whether psilocybin (the active ingredient in magic mushrooms) combined with talk therapy can safely help autistic adults with depression that hasn't improved with other treatments. Twenty participants will receive two doses of psilocybin along with ab…

This study tests a version of the RUBI parent training program that has been adapted for Spanish-speaking families of autistic children aged 2 to 9. About 120 parents will join eight online group sessions to learn practical strategies for managing challenging behaviors. Researche…

This trial tests a web-based sex and relationship education program called Our Love, Our Style (OLOS) for autistic young adults in South Korea. Participants start with either web-based learning alone or web-based learning plus group coaching. Based on their progress, non-responde…

This pilot study explores whether robot-assisted walking devices can be used in schools to help children who cannot walk independently. The devices attach to a walker and power leg movements, allowing children to stand and walk upright. The study aims to identify the best ways to…

This study tests whether adding a motor-sound intervention (PROMPT) to a play-based therapy (JASPER) helps minimally verbal children with autism speak more words and combine them into phrases. Fifty-six children aged 4-7 who use fewer than 20 words will receive either JASPER alon…

This study tests whether personalized sequences of behavioral interventions can help minimally verbal preschoolers with autism improve their language skills. Researchers will compare two teaching approaches—JASPER (play-based) and DTT (structured)—to see which works best for each…

This study tests a 12-month education and health coaching program for parents of children ages 3-7 who were recently diagnosed with autism. The program focuses on lifestyle factors like diet, sleep, and environment. Researchers want to see if this approach helps reduce autism sym…

This study tests whether training early intervention providers in Reciprocal Imitation Training can help children with autism improve imitation and communication skills. The program involves 440 children and their caregivers, with providers learning simple strategies during play.…

This study tests whether listening to special audio fairy tales before sleep can improve speech, motor skills, and independence in a child with autism and central auditory processing disorder. The child stays at home with their mother, avoiding clinic stress. Early results from t…

This study tests whether DHA, a key omega-3 fatty acid found in fish oil, can improve symptoms of autism in children and adolescents aged 5 to 17. Participants receive either DHA or a placebo daily for 12 weeks. Researchers also look at genes and urine markers related to DHA to u…

This study tests an internet-based cognitive behavioral therapy (CBT) program designed to help autistic adolescents aged 11-17 manage anxiety. The program, called LUNA-Adolescent, is parent-led and delivered online, making it easier for families who cannot access in-person therap…

This trial explores whether spending time on a farm with animals and nature can help socially withdrawn young adults (ages 18–30) with autism spectrum disorder. Participants take part in supervised group activities on a farm twice a week for 12 weeks. Researchers will interview p…

This study tests whether a parent-led therapy called Pathways can improve social skills in 80 toddlers (16-30 months old) who show early signs of autism. One group of parents learns a technique to encourage mutual eye contact during play, while the other group uses the same thera…

This study tests whether wearing a virtual reality (VR) headset during dental treatment can reduce fear and stress in children with mild intellectual disabilities or autism spectrum disorder. Two hundred children aged 6 to 12 will either receive standard dental care or use a VR h…

This study tests a new virtual reality program designed to help adults with autism improve their social skills and daily functioning. 140 participants will either receive the VR training plus usual care, or usual care alone, for 3 months. Researchers will measure changes in socia…

This study tests a therapy called RETELL that helps people with severe mental illness explore and reshape their life stories. The goal is to improve personal recovery, quality of life, and identity. Twenty adults with conditions like schizophrenia, bipolar disorder, or PTSD will …

This study tests whether an autism-friendly mobile app can help children and teens (ages 8-17) with autism improve their toothbrushing skills and habits. Participants use either the special app or a basic control app for 3 months while researchers track brushing quality and habit…

This study tests whether a probiotic supplement called Neuralli® MP can help adults aged 18 to 50 who have autistic traits. Participants take two capsules daily and set their own personal goals to track progress. The trial is small and open-label, meaning everyone knows they are …

This study looks at whether playing pickleball for 12 weeks can improve thinking skills like attention and memory, as well as motor skills like balance and strength, in adolescents aged 14-18 with mild intellectual disability. Thirty-two students will either play pickleball three…

This small study tests whether adding robot-assisted walking training to regular physiotherapy can improve body composition (like muscle and fat) and daily function in children with cerebral palsy or similar conditions. Nine children will each receive both standard physiotherapy …

This study tests whether a nurse-led sailing program can improve resilience, self-esteem, and social skills in school-aged children with autism. About 184 children aged 7 to 12 will either take part in a 6-day sailing program or do simple craft activities. Researchers will compar…

This study looks at whether N-acetylcysteine (NAC), a well-tolerated nutritional supplement, can reduce restricted and repetitive behaviors in children with autism spectrum disorder. About 144 children who previously took part in related NAC trials will receive the supplement and…

This small pilot study at Yale University is testing a modified form of cognitive-behavioral therapy (CBT) for children with autism and intellectual disability who struggle with irritability, tantrums, and aggression. Up to 6 children aged with an IQ between 55 and 85 will receiv…

This study compares two 8-week exercise programs for adults with intellectual disabilities who are currently sedentary. One group does traditional strength and endurance exercises, while the other plays sports like soccer and basketball. Researchers will measure changes in physic…

This study tests whether teaching parents of children with autism special behavior support techniques can reduce challenging behaviors and lower parenting stress. About 80 families in South Punjab, Pakistan, will take part in an 8-10 week program. The goal is to see if this appro…

This small study tests whether group drumming sessions can improve emotional well-being for parents of autistic children. Ten parents will take part in drumming groups and report changes in anxiety, depression, and feelings of belonging. The goal is to see if making music togethe…

This study tests whether folinic acid (leucovorin), a drug already used to reduce side effects from chemotherapy, can improve language skills in children with autism spectrum disorder. Researchers will give the drug or a placebo to 56 children aged 5 to 17 for 12 to 24 weeks and …

This study tests a program called MSE-PEACE, where parents and children with developmental disabilities (like autism or ADHD) attend 10 sensory sessions together. The goal is to see if it improves children's engagement and emotional control, and helps parents feel more confident …

This study tests an 8-week program called SCERTS for children aged 3 to 6 with mild or moderate autism. The program focuses on improving social communication and emotional regulation while supporting parents. Researchers will measure changes in the child's social skills and the f…

This study compares a home-based, tailored sleep therapy (CBT-I) to a web-based version for 80 caregivers who have insomnia while caring for a child with autism. The goal is to see which approach is more acceptable and feasible for improving sleep and reducing caregiver burden. P…

This phase 3 study tests a device that gently stimulates the vagus nerve through the skin (tVNS) to see if it can safely reduce temper outbursts in people with Prader-Willi syndrome. About 102 participants aged 10 to 40 will use either continuous or intermittent stimulation. The …

This study tests a 16-week parent-assisted social skills program for 60 preschoolers with autism. Children learn through puppet shows, role-playing, and structured play, while parents get coaching. The goal is to improve social skills like making friends and playing with peers.

This study tests a virtual reality program where children with autism watch avatars of themselves doing social behaviors like making eye contact and then practice those behaviors. 75 children aged 6-11 will be split into three groups: one gets the VR training, one gets standard t…

This pilot study tests whether benzodiazepines (midazolam and clonazepam) and, if needed, electroconvulsive therapy (ECT) can safely reduce catatonic symptoms in 30 adolescents aged 10–19 with profound autism. Participants first receive a midazolam challenge; those who respond th…

This study looks at whether a program involving horseback riding and horse care can help school-age children with intellectual disabilities. Researchers will measure stress (using heart rate, oxygen levels, and skin responses), balance, and daily living skills in 60 children. The…

This study tests whether children with autism will do daily 5-10 minute exercises if they see themselves, other kids, or kids in rabbit suits in a mobile app. Twenty children and their parents will use the app for 8 weeks. Researchers will measure how long kids stick with the exe…

This study tests a group therapy program called Regulating Together for children with autism who struggle with intense emotions. The program teaches children and their caregivers skills to manage emotional reactions using techniques like cognitive behavioral therapy and mindfulne…

This study tests whether ChatGPT can act as a job coach for adults with autism spectrum disorder (ASD). Thirty motivated adults aged 18-45 will use ChatGPT to build resumes, practice interviews, and discuss workplace social skills over one week. Researchers will measure changes i…

This study tests a 6-8 week caregiver-child therapy called THRIVE, delivered by school counselors via video or in school, to improve emotional and behavioral health in children aged 3-7 who are near the clinical threshold for mental health problems. THRIVE is compared to an onlin…

This study tests whether an internet-based job interview training program (Virtual Interview Training) helps autistic young adults improve their interview skills and find jobs. About 200 participants will either use the training or continue with their usual services. Researchers …

This study tests whether virtual reality games can improve executive functions—like memory, focus, and planning—in 50 youth aged 8 to 18 with autism and ADHD. Participants will either play VR cognitive games or receive standard care. Researchers will compare thinking skills befor…

This study tests whether personalized computer games can help people with Rett syndrome use their arms more purposefully and reduce repetitive hand movements. Fourteen participants will play games that reward them for separating their hands and reaching for targets. The training …

This study tests whether a type of brain stimulation called rTMS can reduce depression in people with autism. Twenty-four participants aged 13 to 26 will receive either real or sham (fake) stimulation to see if it helps. The goal is to find a new way to treat depression that hasn…

This study tests a classroom program called the Kids' Empowerment Program (KEP) to help children ages 6 to 12 reduce symptoms of anxiety and depression. About 120 children will take part, with some starting the program right away and others later. The goal is to give kids practic…

This study compares two suicide prevention programs designed specifically for autistic youth aged 12-24. One program is a safety planning intervention (SPI-A), and the other adds follow-up contacts (SPI-A+). Researchers will track changes in suicidal thoughts and behaviors over a…

This study tests whether a parent training program called Project IMPACT can improve social communication and reduce autism symptoms in children aged 1.5 to 6.5 years. Eighty children will be randomly assigned to either the IMPACT group (parents do at least 10 hours of therapy pe…

This study tests a program called FACE-Rare, designed to support family caregivers of children with rare, life-limiting diseases. The program includes three sessions to help families prepare for future medical decisions and improve their quality of life. Researchers will compare …

This study looks at whether a specially designed group resistance training program (called RESID) is safe, enjoyable, and helpful for adults with Down syndrome aged 20 to 45. The program includes exercises like lifting weights or using resistance bands. Researchers want to see if…

This study tests whether a probiotic called Saccharomyces boulardii can improve digestive health, immune function, nutrition, and behavior in children aged 5 to 12 with autism and a history of gut problems. Sixty-six children in Ecuador will take the probiotic for 4 months and th…

This study tests a 14-day digital writing program designed to help parents of autistic individuals manage stress and improve mental health. Participants use a WeChat mini-program to write about their feelings and practice self-kindness for about 5 minutes each day. The trial incl…

This study tests a gamified app called MITA designed to improve language and thinking skills in children with autism aged 2 to 5. Sixty children will be randomly assigned to either use the app alongside their usual therapy or continue with standard care alone. The goal is to see …

This pilot study tests whether a home-based computer training program can improve executive function (like shifting attention and controlling impulses) in autistic children aged 8 to 17. Fifteen participants will play two social-themed games for 30 minutes a day, five days a week…

This study tests a computer-based training program designed to improve executive function (thinking, self-control, and memory) in 40 autistic children aged 5-7 in South Korea. Children will play games on a tablet and do activities with a coach over several sessions. Researchers w…

This study looks at whether a set of thinking and memory tests can detect changes caused by a stimulant medication in children aged 6 to 17 who have both intellectual disability and ADHD. About 68 participants will receive either the medication or a placebo, then switch after a b…

This study tests whether a special type of family therapy, called Narrative Family Therapy, can improve how families with an autistic child communicate and function. Sixty-three families will either receive eight therapy sessions or play family games. Researchers will also use br…

This study tests a family-centered program called Transitioning Together / Juntos en la Transición, designed to help autistic youth (ages 14-21) and their parents navigate the move to adulthood. The program includes one individual family session and four group sessions covering t…

This phase 2 trial tests whether sulforaphane, a compound found in broccoli, can improve core symptoms of autism. 48 males aged 13-30 with autism will receive either sulforaphane or a placebo for several weeks. Researchers will measure changes in behavior and social responsivenes…

This study looks at whether telehealth tools can help families concerned about autism in their infants. Researchers are testing an online assessment tool and a set of video modules that teach parents strategies to support their child's communication. 80 families will be randomly …

This study tests a drug called pitolisant in 62 boys and teens aged 6 to 17 with autism. The goal is to see if it improves social skills and behavior. Participants are randomly assigned to receive the drug or a placebo, and neither they nor the researchers know who gets which. Th…

This study tests whether cannabidiol (CBD) can reduce severe behavior problems like aggression, self-harm, and repetitive actions in children with autism. About 42 boys aged 7-14 with severe autism and daily behavioral issues will receive both CBD and a placebo at different times…

This study tests a 6-week virtual group program that teaches parents strategies to encourage their toddler's vocalizations and shared smiling. The program is for children aged 12-42 months who have early signs or a diagnosis of autism or related social communication challenges. R…

This study tests a wearable robotic exoskeleton called EXPLORER in 15 children with movement problems from conditions like cerebral palsy. The goal is to see if it helps them walk better in their everyday environments, such as home and the community. The study focuses on safety, …

This study tests whether a non-invasive brain stimulation technique called cTBS can improve how children with autism (ages 6-12) combine what they see and hear. Forty children will receive either real or sham stimulation over 10 sessions. Researchers will use brain scans, genetic…

This study tests whether a non-invasive brain stimulation technique called iTBS can improve attention and self-control in children aged 6 to 12 with ADHD. Over two weeks, 40 children will receive either real or sham stimulation, and researchers will measure changes in attention u…

This pilot study explores whether laughter yoga can reduce depression and anxiety in children with autism spectrum disorder (ages 8–12) and lower parenting stress for their parents. 66 families will either take part in laughter yoga sessions or be placed on a waiting list. The st…

This Phase 3 trial tests a nasal spray called carbetocin to reduce the intense, constant hunger (hyperphagia) in people with Prader-Willi syndrome. The study involves 170 participants aged 5 to 30 and lasts 12 weeks. Caregivers will rate changes in hunger-related behaviors using …

This study tests a brain training method that combines EEG and fNIRS to help autistic children aged 8 to 12 improve attention, emotion control, and social skills. Thirty children will receive neurofeedback sessions and complete thinking and behavior assessments. The goal is to se…

This study planned to test whether sulforaphane, a compound found in broccoli, could improve social skills and behavior in people with autism spectrum disorder (ASD). Researchers aimed to enroll 50 people with ASD aged 12–34 and 30 healthy volunteers, giving the ASD group sulfora…

This study tests a robotic feeding device called Obi for people who cannot use their arms to eat on their own. About 50 participants, including patients, caregivers, and healthcare providers, will use the device at home or in care settings for one week. Afterward, they will fill …

Researchers are sequencing the gut microbiomes of people with a wide range of conditions, from autism and Alzheimer's to Crohn's disease and multiple sclerosis, to see whether bacterial patterns differ by disease. The study also enrolls healthy participants for comparison. By mat…

Researchers at the University of Management and Technology Sialkot are studying how motor coordination relates to postural stability in children with autism spectrum disorder. The study includes 30 children aged 5 to 12 who can stand and walk without help. Parents answer a questi…

This study looks at whether young adults (ages 18-27) with mild to moderate intellectual or developmental disabilities feel less lonely and more connected when they learn travel skills from a peer who also has a disability, compared to learning from regular staff. About 325 parti…

This study tests whether wearable biosensors can accurately track symptoms like heart rate, breathing, sleep, and movement in people with Rett syndrome. Up to 30 participants will use the devices at home for 9 weeks and also undergo one overnight sleep study in the clinic. The go…

This study follows 154 people taking Epidiolex (cannabidiol) for seizures caused by Lennox-Gastaut syndrome, Dravet syndrome, or tuberous sclerosis complex. Researchers will monitor for signs of chronic liver injury and fibrosis over time using blood tests and liver scans. The go…

This study tests a program called Individual Placement and Support for Autism (IPS-AUT) to help autistic adults ages 18-40 find and keep competitive jobs. Researchers will work with employment agencies to train staff and track job outcomes, satisfaction, and thinking skills. The …

This study investigates the link between autism and physical symptoms like gastrointestinal issues, autoimmune diseases, and heart problems. Researchers will compare autistic individuals, people with anxiety or depression, and healthy controls to understand how these symptoms are…

This observational study aims to measure how many people in Portugal have Dravet syndrome (DS) or Lennox-Gastaut syndrome (LGS), two rare and severe forms of epilepsy. Researchers will review existing medical records from about three public hospitals to estimate the percentage of…

This study looks at how parents of autistic young people (ages 12-18) with anorexia nervosa make sense of a group intervention designed for them. Researchers want to understand how parents interpret the concepts from the group and apply them to daily family life, and how they see…

This study follows mother-infant pairs from birth to 24 months to see how early nutrition, environmental exposures, and family factors relate to growth, development, and allergies in preterm and term infants. Researchers will use questionnaires and biological samples to examine h…

This study follows people with Phelan-McDermid syndrome, a rare genetic condition linked to autism and intellectual disability, to map how the syndrome unfolds over two years. Researchers will measure changes in thinking, behavior, language, and motor skills, and use brain imagin…

This study looks at whether specific brain wave patterns on an EEG can predict how well the drug fenfluramine works for people with Lennox-Gastaut Syndrome (LGS), a severe form of epilepsy. Researchers will follow 20 children and adults aged 2 to 35 who are already taking fenflur…

This study looks at whether air pollution exposure before birth is linked to ADHD risk in Puerto Rican children. Researchers will use brain scans (MRI) in 182 kids aged 6-11 to examine brain areas tied to attention. They also want to see if inflammation during pregnancy or sleep …

This study follows individuals with autism who carry a PTEN gene mutation, along with comparison groups, to track changes in thinking, language, and behavior over time. Researchers will also collect blood samples and medical data to build a large database for future studies. The …

This study investigates whether advanced brain imaging and electrical recordings can predict the presence and severity of autism spectrum disorder (ASD) and intellectual disability (ID) in people with tuberous sclerosis complex (TSC). Researchers will follow participants over tim…

This study looks at auditory brainstem evoked potentials (BAEPs) — electrical signals from the brain in response to sound — in children with autism spectrum disorder (ASD). By comparing these signals to those of children with other neuropsychiatric conditions, researchers hope to…

This study follows children aged 6 to 21 who have a CNKSR2 gene mutation, which can cause epilepsy, language difficulties, and intellectual disability. Researchers will use brain scans, EEGs, and developmental tests to track how the condition changes over time. The goal is to cre…

This study compares social cognitive abilities—like recognizing emotions and understanding others' thoughts—in children with autism versus typically developing children aged 2.5 to 14 years. Researchers will use behavioral tasks and eye-tracking to map developmental patterns. The…

This study compares two programs for parents of teens and young adults (ages 11-27) with intellectual or developmental disabilities. One program teaches parents how to advocate for their child's health, while the other connects them with other parents for shared learning. Researc…

This study aims to uncover the genetic and environmental factors behind autism spectrum disorders (ASD), including autism, Asperger's syndrome, and PDD. Researchers will analyze gene expression in 240 participants aged 18 months and older who have an ASD diagnosis. The goal is to…

This study uses brain scans and behavioral data from 267 adults (80 with autism, 79 with schizophrenia, and 108 healthy controls) to explore shared brain activity patterns related to social thinking. No new treatments are given; researchers analyze existing data to understand how…

This study works with children aged 7 to 16 who have borderline intellectual disorder, their families, and experts to improve a tablet-based AI program that helps with thinking and language skills. Researchers are collecting feedback through interviews and workshops to make the p…

This study is not a treatment trial. It aims to translate and test a new assessment tool (FARAS-IT) for adults with autism who are involved in the Italian criminal justice system. Researchers will enroll 100 adults with autism, including those in forensic psychiatric care, those …

This study tests a toolkit called PEACE, designed to help early intervention providers coach parents of young children with autism. Researchers will enroll 200 providers and 400 parent-child dyads to see if the toolkit improves how providers support families. The goal is to make …

This study tests a therapy where parents of children with autism (ages 1.5 to 3) get video feedback to help them better understand and respond to their child's signals. The goal is to improve joint attention, a key skill for social communication. Twenty-seven families will be ran…

This study is observing up to 198 people with rapid-onset dystonia-parkinsonism (RDP) or related genetic mutations. Researchers aim to identify affected individuals, document how common the disease is, and track its progression over time. Participants undergo assessments of sympt…

This study aimed to see if a 16-week perceptual-vision training program could improve thinking skills (like focus) and physical fitness (balance, strength, agility) in adults aged 40-70 with mild intellectual disability. Participants were to be split into training, training-then-…

This study looks at why children with autism spectrum disorder (ASD) have trouble imitating others. Researchers will have 201 children aged 8 to 12 perform imitation tasks while recording brain activity with EEG. The goal is to pinpoint where the breakdown happens, which could im…

This study creates a registry for up to 500 people with CDKL5 Deficiency Disorder (CDD), a rare genetic condition that causes seizures and developmental delays. Patients and their caregivers will provide information about symptoms, treatments, and quality of life over several yea…

This study looks at whether melatonin, a common sleep aid, can improve sleep spindles—special brain waves that help with sleep and attention—in 30 children with autism aged 12-18. Researchers want to understand why sleep and sensory issues happen in autism and test if melatonin c…

This study looked at whether a real-time pose recognition system could help 30 teenagers with mild to moderate intellectual disabilities learn tennis skills more accurately and quickly than traditional coaching. Participants were split into two groups: one used the technology dur…

This study follows about 12 children and young adults (ages 4 to 21) with autism who are already using intranasal insulin as part of their regular care. Researchers want to see if this treatment is safe and if it helps with social skills, communication, and behavior over 6 months…

This observational study follows 36 people with neurologic conditions like traumatic brain injury, cerebral palsy, multiple sclerosis, or Alzheimer's who are already receiving intranasal MuSE stem cell or exosome therapy as part of their routine care. Researchers track safety and…

This study looks at whether treating obstructive sleep apnea (OSA) with a breathing device (CPAP) lowers blood pressure more in people who are very sleepy compared to those who are not. Researchers will analyze data from about 10,000 adults with OSA from previous studies. The goa…

This pilot study explores a new language intervention called Milieu Teaching-AV for infant siblings of autistic children, who are at higher risk for autism or language delays. The intervention teaches caregivers to encourage babies to look at the mouth during play, aiming to impr…

This study tests whether a home visiting program for mothers facing stress or past trauma can improve their mental health and their child's emotional and behavioral development. Researchers will follow 352 mother-child pairs from pregnancy until the child is 18 months old. They w…

This pilot study will test whether an online assessment battery is practical for children with autism who are receiving complementary and integrative healthcare. Twenty children aged 3-12 will complete online questionnaires and check-ins with their practitioners. The main goal is…

This study looks at how the gut microbiome (the bacteria in our intestines) is linked to stress in children with autism or those who have experienced trauma. Researchers will give 75 children, ages 4 to 7, a probiotic powder for 4 weeks and measure changes in their gut bacteria a…

This small study will give 5 teens with autism a single dose of escitalopram (Lexapro) or a placebo, then scan their brains to see how the drug affects networks linked to rigid thinking and repetitive behaviors. The goal is to understand whether this medication might help with in…

This study looks at whether a group therapy program called Unstuck & On Target can improve flexibility and learning in 64 teens with autism aged 14-18. Researchers will use brain scans (MRI) to see if the therapy changes how the brain processes new information. The goal is to und…

This study planned to test vagus nerve stimulation (VNS) in children and adults with autism and developmental disabilities to see if it could improve behavior, thinking, and body rhythms. Participants would have chosen 1 to 3 months of daily VNS and completed monthly check-ins. T…

This study tests a program called the Community Mobility Planning Process (CMPP) for young adults aged 16-30 with mild to moderate intellectual disabilities. Over 8 weeks, participants work with support staff on activities to plan how to get around their community more independen…

This study looks at how eye movements, specifically eye vergence (how eyes work together for depth perception), relate to attention in people with ADHD and autism. Researchers will use computer-based eye-tracking tasks with 200 children and adults to see if these eye patterns can…

This study planned to use high-frequency brain signals to locate important brain areas and understand conditions like epilepsy, migraine, and ADHD. It aimed to create a large database of brain activity in people aged 1 day to 69 years. However, the study was withdrawn before any …

This study tests whether a non-invasive brain stimulation technique called repetitive Transcranial Magnetic Stimulation (rTMS) can change how adults with autism process social information. Researchers will measure brain responses with EEG and eye-tracking before and after stimula…

This study looks at whether a tiny device placed under the scalp can safely and comfortably track seizures in adults with mild to moderate intellectual disability and epilepsy. Nine participants will wear the monitor for up to 6 months, and researchers will compare the device's r…

This study tests whether wearable sensors and video recordings can help detect motor development problems in infants from birth to 24 months. Researchers will compare data from these devices with standard clinical tests. The goal is to create a more precise and automated way to i…

Researchers at Arkansas Children's Hospital are studying how mitochondria work in children with autism spectrum disorder. They aim to identify distinct patterns of mitochondrial dysfunction that may relate to developmental delays. The study involves up to 5 visits with blood draw…

This study looks at how repeated seizures affect breathing and heart function in people with Dravet syndrome, a severe form of epilepsy. Researchers will monitor 92 children and adults for 24-48 hours using video, brain wave, heart, and breathing recordings. The goal is to find c…

This study uses brain scans and magnetic stimulation to compare brain activity in 160 adults with autism, depression, both, or neither. Researchers want to understand how brain inflammation and the balance of excitation and inhibition relate to social and emotional symptoms. No t…

This study looks at whether taking common painkillers like paracetamol or ibuprofen during pregnancy can affect a child's brain development, behavior, and language skills. Researchers will follow 685 children aged 2 to 4 years whose mothers were part of an earlier study on painki…

This study is collecting genetic information and observable traits from 1500 people with autism and their families. The goal is to better understand how genes relate to autism. Participants must be at least 18 months old and have an autism diagnosis or a related genetic finding.

This study looks at whether special reading lessons can help young children (ages 4-8) with intellectual disabilities who use pictures or devices to communicate. Researchers will compare different timing and combinations of reading programs in kindergarten and first grade. The go…

This study looks at whether a special training program called TEAMS helps therapists and teachers use proven autism therapies more effectively. Researchers will work with about 1206 people, including program leaders, providers, and parents of children with autism. The goal is to …

This study works with 110 adults who have intellectual or developmental disabilities and receive care from a Norwegian municipality. The goal is to create better routines and digital tools to make sure they get the right medicines safely. Researchers will review each person's med…

This study is testing a new online sex education program called 'Our Love, Our Style' for autistic young adults in South Korea. The program covers relationships, consent, and sexual health over 10 weekly sessions. Researchers want to see if it improves knowledge and relationship …

This small pilot study aims to see if digital biomarkers—like heart rate and movement tracked by a watch—can help measure changes in autism symptoms during treatment with sulforaphane, a compound found in vegetables. Ten males aged 13-30 with autism who previously took part in a …

This pilot trial tests a culturally adapted parent training program for Navajo families of children with autism or developmental disabilities. About 29 parents will be randomly assigned to join the program right away or after a wait. The program is led by trained Navajo Family Ad…

This study looks at spinal posture, balance, and ankle muscle tightness in children with autism compared to typically developing children. Researchers will measure these physical traits using tools like the Spinal Mouse and balance tests. The goal is to understand if children wit…