Complex neurodevelopmental disorder

This study tests whether clemizole HCl (EPX-100) can safely reduce seizures when added to current treatments for Lennox-Gastaut syndrome, a severe form of epilepsy. About 260 children and adults aged 2 to 55 will receive either the drug or a placebo. The main goal is to measure c…

This Phase 3 study tests a drug called zorevunersen in 170 children aged 2 to 17 with Dravet syndrome, a severe form of epilepsy. The drug is given as an injection into the spine and compared to a sham procedure. The goal is to see if it reduces seizures and improves daily living…

This early-stage trial tests a single dose of TSHA-105, a gene therapy injected into the spinal fluid, in 8 people aged 2 to 20 with SLC13A5 citrate transporter disorder. The goal is to see if it is safe and whether it can improve motor and thinking skills. Because the trial is v…

This study tests an experimental drug called EPX-100 (clemizole) in 150 children and adults with Dravet syndrome, a severe form of epilepsy. The goal is to see if adding EPX-100 to current treatments can safely reduce the number of motor seizures. Participants will be randomly as…

This early-stage trial tests a gene therapy called RC001 in children aged 2 to 18 with Dravet syndrome, a severe form of epilepsy caused by a genetic mutation. The therapy is given as a single injection into the spinal fluid. The main goal is to check safety and how the body proc…

This early study tests whether a smart brain implant can help people with spinocerebellar ataxia type 6, a condition that causes trouble with balance and coordination. Five adults will get the implant, which records brain signals and adjusts stimulation automatically. The goal is…

This study tests a gene therapy called ETX101 for children with Dravet syndrome, a severe form of epilepsy caused by a genetic change. The treatment uses a harmless virus to deliver a working copy of the gene to help control seizures. The trial includes infants and children up to…

This study tests a drug called radiprodil in people with GRIN-related neurodevelopmental disorder, a rare genetic condition that often causes seizures and developmental delays. The trial includes two groups: one with frequent seizures and one with fewer or no seizures. Participan…

This study tests a new medicine called ION337 in children aged 2 to 12 with Dravet syndrome, a severe form of epilepsy. The main goal is to see if the drug is safe and tolerable when given as a spinal injection. Researchers will also measure how the drug moves through the body an…

This study tests a treatment called FBET to reduce elopement (wandering away) in children aged 4–12 with autism. About 50 children will receive either FBET or usual care at community clinics. Caregivers will help track progress and practice skills at home.

This study tests an experimental drug called ABS-1230 in children and young adults (ages 1 month to under 22 years) with a rare epilepsy caused by changes in the KCNT1 gene. The goal is to see if the drug is safe and can reduce the number of seizures compared to a placebo. Partic…

This study tests whether a probiotic called Clostridium butyricum GKB7 can help children with autism. About 90 kids aged 4 to 15 will take either the probiotic or a placebo for a period. Researchers will also look for biomarkers in the gut and blood to better understand the gut-b…

This 24-month study tests whether adding umbilical cord stem cells (AdiaVita) to glutathione therapy helps improve autism symptoms in children ages 3-12. About 100 kids will be randomly assigned to get either glutathione alone or glutathione plus stem cell infusions. Parents and …

This study tests a telehealth program called tele-ImPACT for infants aged 6-24 months who have an older sibling with autism, putting them at higher risk for social communication delays or language disorders. The program teaches parents strategies to support their child's communic…

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 whether a combination of umbilical cord stem cells and glutathione can safely improve autism symptoms in 100 people aged 3 and older. Participants receive either the stem cell product plus glutathione, or a placebo plus glutathione, with the option to switch grou…

This study tests a new medicine (S230815) for children aged 2-12 with a rare, severe epilepsy caused by changes in the KCNT1 gene. The goal is to see if the drug is safe and can help control seizures. About 20 children will take part, receiving the drug through a spinal injection…

This study tests a medicine called elsunersen in 40 children with a rare, severe form of epilepsy caused by changes in the SCN2A gene. The goal is to see if the drug can safely reduce how often seizures happen over 24 weeks. Children must have started having seizures before 3 mon…

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 spironolactone, a common diuretic, can improve learning and behavior in children aged 3–10 with NCOR gene mutations, which are linked to autism spectrum disorder. Only 2 children will be enrolled, and researchers will measure changes in intelligence and a…

This early-stage trial tests an experimental drug called CSTI-500 in 12 people aged 13 to 50 with Prader-Willi syndrome, a genetic condition that causes constant hunger and behavioral issues. The study aims to see if the drug is safe and can reduce excessive eating behaviors. Par…

This study is testing the safety of fenfluramine, a drug already approved for children 2 and older with Dravet syndrome, in younger children aged 12 to 23 months. Dravet syndrome is a severe genetic epilepsy that starts in the first year of life. The study will enroll 5 children …

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 early-stage trial is testing the drug vorinostat in 15 girls and young women with Rett syndrome, a rare genetic disorder that affects brain development. Each participant will receive placebo and two different doses of vorinostat, acting as their own control. The study aims t…

This study tests a digital program that teaches parents strategies to help their young children (14-48 months) with autism traits improve communication and daily living skills. The program is completed online without a coach, making it easier for families who face long waitlists …

This study tests the drug fenfluramine in 10 children aged 12 to 36 months whose infantile spasms did not get better with standard treatments. The children take fenfluramine for 21 days, and doctors check brain waves and seizure diaries to see if the spasms stop. If it helps, the…

This early-stage trial is testing whether a nasal spray made from stem cell exosomes can safely improve core symptoms in 40 children aged 3-7 with autism. Half the children get the spray every other day for 10 doses, while the other half receive no treatment. Researchers will tra…

This study tests whether deep brain stimulation (DBS) can safely reduce seizures in children aged 5-14 with Lennox-Gastaut syndrome, a severe form of epilepsy. The trial uses a special device called Picostim to send electrical pulses to the brain. Researchers will track seizure f…

This study tests whether propranolol, a common blood pressure medication, can reduce aggression, self-injury, and severe disruptive behaviors in autistic adolescents and adults. Sixty participants will receive either propranolol or a placebo for 12 weeks, with the option to take …

This trial tests whether deep brain stimulation (DBS) of the nucleus accumbens can reduce severe, repetitive self-injurious behaviors in 25 children aged 5-17 with autism. Half will get active stimulation, half will have the device off, and parents will report behavior changes. T…

This study tests whether probiotics and prebiotics can safely help control appetite and weight gain in 60 children with Prader-Willi syndrome, a rare genetic condition that causes severe obesity. Researchers will measure changes in body mass index, body fat, and gut bacteria over…

This study tests a new drug called ION440 in 48 people with MECP2 duplication syndrome, a rare genetic condition that causes intellectual disability and seizures. The drug is given via a spinal injection to see if it is safe and how the body processes it. Some participants will r…

This study tests whether fecal microbiota transplantation (FMT) can help children and young adults with autism who also have stomach issues. FMT involves transferring healthy donor stool into the patient's gut via colonoscopy. Researchers will track changes in gut and autism symp…

This early study tests whether a deep brain stimulator placed in the cerebellum can safely improve movement and balance in 5 adults with spinocerebellar ataxia type 6. The device automatically adjusts its stimulation based on the person's brain signals. The goal is to see if this…

This study is testing whether a nasal spray containing oxytocin, a natural hormone, can improve bone health in children with autism spectrum disorder (ASD) aged 6 to 18. Participants will receive either oxytocin or a placebo spray twice daily for 12 months, followed by 6 months w…

This study tests a 16-week inclusive exercise program called PACE for adults with intellectual disability. Participants will attend fitness classes, meet with coaches, and use a web dashboard to set goals. The trial includes 376 people and will measure daily steps and moderate-to…

This phase II trial is testing whether mirtazapine, an antidepressant, can improve mood, sleep, and motor symptoms in people with Rett syndrome. The study will enroll 54 females aged 5 to 40 with a confirmed MECP2 mutation. Participants will receive increasing doses of mirtazapin…

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 whether L-serine, a natural amino acid taken as a supplement, can improve overall functioning in children and young adults (ages 2-30) with GRIN-related neurodevelopmental disorders caused by certain gene variants. Each participant will receive both L-serine and …

This study aims to find the best way to sequence two parent-led therapies for young children with autism: one focused on social communication and another on disruptive behavior. Researchers will enroll 184 children aged 18-48 months and their caregivers to compare different thera…

This study tests an experimental drug called ASO-GNAO1 (Tianasen) in 5 children aged 1 to 14 with a specific GNAO1 gene mutation that causes severe epilepsy and movement problems. The drug is given via spinal injections over 12 months to see if it can reduce seizures and improve …

This study tests whether a child's own bone marrow stem cells can safely improve movement and muscle control in cerebral palsy caused by lack of oxygen at birth. Fifty-eight children aged 1 to 10 will be split into two groups: one gets stem cell infusions right away, the other wa…

This study looks at how well a special intensive therapy called ACQUIRE Therapy works for children with motor problems caused by conditions like cerebral palsy, autism, or brain injury. The therapy involves many hours of play-based exercises each day, often with a cast or splint …

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 study tests a weekly injection called RM-718 in healthy people with obesity and in patients with obesity caused by problems in the MC4R pathway, including hypothalamic obesity and Prader-Willi syndrome. The main goal is to check the drug's safety and how the body processes i…

This study tests whether the Early Start Denver Model (ESDM), a play-based behavioral therapy, helps young autistic children improve language and daily living skills when used in community programs. Researchers will enroll 600 participants, including 300 children aged 1-5, across…

This study will follow about 4,000 people who have received a fecal microbiota transplant (FMT) — a procedure that transfers gut bacteria from a healthy donor to restore balance in the gut. It aims to see how well FMT works over 3 months, 1 year, and 5 years for conditions like r…

This study tests a 10-week swim and water safety program called AquOTic for children with autism ages 5 to 9. The program uses one-on-one coaching in small groups to teach swimming and safety skills. Researchers want to see if the program works and which teaching method is best—u…

This study compares two common stimulant classes (amphetamine and methylphenidate) and alpha-2 agonists to treat ADHD in 500 children aged 4-17 with autism. The goal is to find which medication works best and reduce the trial-and-error process. Parents and doctors will track symp…

This study tests tirzepatide, a weight loss medication, in 36 young adults aged 18-26 with obesity from Prader-Willi syndrome, hypothalamic obesity, or general causes. Participants take the drug for 48 weeks to see how much weight they lose and how their health changes. The goal …

This early-phase trial tests whether transplanting healthy donor gut bacteria (washed microbiota transplantation) can improve autism and ADHD symptoms in 80 children aged 3-17. The approach targets the 'gut-brain axis,' since many children with autism have gut microbiome imbalanc…

This study is testing whether a portable eye-tracking device can accurately screen 9-month-old infants for autism spectrum disorder (ASD). Over 2,000 babies will watch short videos while cameras track their eye movements. Researchers will then compare these results with standard …

This study is testing whether a simple, non-invasive eye-tracking test can help diagnose autism in young children (ages 24-72 months) born to mothers with HIV in Kenya. Researchers will compare eye-tracking results with standard clinical autism evaluations in 850 children. If the…

This study is testing whether a smartphone app called BlinkLab Dx1 can help doctors diagnose autism in children ages 2 to 11. The app shows videos and sounds, then measures how children react. Parents also answer questions about their child's development. The goal is to see if th…

This study is testing a digital app called QbMobile that measures attention, impulsivity, and activity levels through two 10-minute tests. Researchers want to see if the app can help distinguish ADHD from conditions like bipolar disorder, autism, depression, and anxiety. About 30…

Researchers at Indiana University are studying whether a short, non-invasive eye-tracking test can help detect autism risk in children aged 12 to 48 months. The test uses a camera to measure eye movements and pupil size while children watch pictures and videos. Results will be co…

This study is testing a new test called the Five Digits Test (FDT) to see if it can measure a person's ability to stop automatic responses—a skill often affected in ADHD and autism. The FDT does not require reading, making it useful for people with different language backgrounds.…

This study is testing a device called SenseToKnow that uses video and a caregiver survey to help detect autism in children aged 16 to 36 months. The goal is to see how accurate the device is compared to a doctor's diagnosis. About 350 children from Duke Health will take part.

This study is testing whether a portable sleep monitor can accurately detect obstructive sleep apnea in children with autism spectrum disorder. Twenty children aged 6 to 18 will wear the portable device at the same time as their standard in-hospital sleep test. Researchers will c…

This study is testing whether a simple 8-item screening tool called the Autism Mental Status Exam (AMSE) can help non-specialist doctors and psychologists identify autism in adults. Currently, adults in France wait an average of 19 months for a diagnosis at expert centers, and ma…

This early-phase study tests whether a ketogenic (high-fat) diet can prevent epileptic spasms in infants with genetic epilepsy. Ten babies under 9 months old will receive the diet and be monitored with regular EEGs. The goal is to see if this approach is safe and feasible, potent…

This study looks at how sensory sensitivity affects sleep and emotions in autistic children ages 6-10. Researchers will test a simple bedtime routine called Power Down, where caregivers give their child a gentle massage to help them relax before sleep. The goal is to see if this …

This study tests whether a version of Dialectical Behavior Therapy (DBT) skills training, adapted for autistic adults, is feasible and helpful for those with emotion regulation difficulties. The program uses simplified language, more visuals, autism-relevant examples, and hands-o…

This study is testing a 12-week early intervention program for preschoolers (ages 2 to 5) with developmental disorders like autism or intellectual disability. The program provides 12 hours of therapy each week, either at a center or at home, aiming to improve social communication…

This study tests whether a 16-week behavioral therapy called Pivotal Response Treatment (PRT) works better when done at a center or at home for young children with autism who have language delays. Researchers will compare both groups to children receiving usual care. The goal is …

This study tests a 12-week behavioral therapy program delivered via video calls to help autistic youth (ages 4-17) cope with insistence on sameness—difficulty tolerating changes in routine. Parents are trained to support their child during weekly sessions. The goal is to see if t…

This pilot study tests whether esomeprazole (Nexium), a common stomach acid medication, can improve social communication in 25 children with autism aged 2 to 6. Over 12 weeks, kids take the drug daily, and researchers measure changes in social behavior using standard rating scale…

This study tests whether a non-invasive brain stimulation technique called tDCS can improve social communication and reduce repetitive behaviors in adolescents with autism. Researchers will use machine learning to analyze brain activity (EEG) and clinical data to predict who will…

This study tests whether an online Spanish course can teach parents of young children with autism to use Pivotal Response Treatment (PRT) to boost communication and social skills. Forty Spanish-speaking families with children aged 2 to 5 will take 12 weeks of online lessons, atte…

This study tests whether folinic acid (a form of vitamin B9) can reduce irritability and improve behavior in 150 children aged 3 to 6 with autism. Half will get folinic acid, half a placebo, for 9 weeks. Researchers will also check if it helps with communication, sleep, and gut s…

This study tests whether shining a low-level infrared light on the forehead (called photobiomodulation) can improve autism symptoms like inattention, impulsivity, and social difficulties. Researchers will compare the real light treatment to a sham (inactive) device in 280 people …

This study tests whether the drug arbaclofen can improve social function, anxiety, and quality of life in children and adolescents with autism. Participants first receive a single dose of arbaclofen or placebo to see if brain-wave changes can predict who will benefit. Then everyo…

This study tests whether a parent-led online program can improve sleep in autistic children aged 3 to 7 years. About 130 children will be randomly assigned to one of two behavioral sleep programs for 10 weeks. The goal is to see if the structured program reduces insomnia symptoms…

This study tests whether a web-based program called autimia, which uses cognitive behavioral therapy techniques, can help adults with autism spectrum disorder manage stress and improve their well-being. About 236 participants will either use autimia plus their usual care, or just…

This study tests whether immersive virtual reality (VR) can train children with autism, aged 8 to 16, to better process multiple senses at once. Fifty participants will either receive VR training plus usual care or usual care alone. The goal is to see if VR practice helps them fe…

This study tests a new parent group program for teens aged 12-17 with autism who also have anxiety or depression. The program teaches parents skills to support their teen's mental health. Researchers will see if the program is practical and helpful for families.

This study tests whether transferring healthy gut bacteria (fecal transplant) through a tube or colonoscopy can reduce autism symptoms in 75 children aged 3-16 with moderate-to-severe autism. Kids continue their usual behavioral therapy. The main goal is to see if autism severity…

This study tests whether a safe, non-invasive brain stimulation technique called tDCS can help reduce certain autism symptoms, like repetitive behaviors and hyperactivity, in children and young adults aged 4 to 17. Researchers will apply a mild electrical current to the cerebellu…

This study tests whether folinic acid, a form of vitamin B9, can improve autism symptoms in children aged 3 to 10. About 184 children will receive either folinic acid or a placebo for 12 weeks, then all can try the vitamin. The goal is to see if it reduces behavioral and communic…

This study tests a specially adapted early intervention for young Hispanic autistic children (18-42 months) from low-income families. The program, called CLR-Pathways, is delivered by bilingual coaches in the home and focuses on improving social communication and daily skills. Re…

This study adapts a group therapy program called Regulating Together for children aged 8-12 with autism and intellectual disability who struggle with emotions like irritability and aggression. The therapy uses relaxation, mindfulness, and parent training to help kids regulate the…

This study tests a new, non-invasive brain stimulation technique called tTIS in 16 children aged 4-10 with autism. The main goal is to see if it is safe and tolerable. Some children will receive real stimulation and others a sham (fake) version for 5 days. Researchers will track …

This study tests a group program to help teens (ages 13-17.5) with Prader-Willi Syndrome and their families learn practical ways to handle irritability, meltdowns, and anxiety. Ten participants will attend weekly 90-minute sessions. The goal is to see if the program improves emot…

This study tests a 12-week exercise program called FMS-CrossFit in 184 preschoolers with autism, ages 3-6. The program combines motor skill drills with circuit training to improve school readiness, social skills, and self-regulation. Researchers will compare these kids to those r…

This study tests a brief online program called Project Independence for caregivers of autistic youth aged 10 to 14. The program gives practical tools to help kids build daily living skills like dressing or chores. About 70 caregivers will be randomly assigned to the program or an…

This study tests whether bright light therapy can help reduce excessive daytime sleepiness in people with Prader-Willi syndrome. About 50 participants will receive either bright light or a dim placebo light. The goal is to see if the therapy improves alertness, mood, and behavior…

This pilot study tests whether a trauma-focused therapy (TF-CBT) adapted for autistic youth is practical and well-received in community mental health centers. About 24 autistic children and their therapists will participate. The goal is to see if the therapy is acceptable and fea…

This study tests whether N-acetylcysteine (NAC), a well-tolerated nutritional supplement, can reduce restricted and repetitive behaviors in children with autism. About 144 children aged 3 to 12 will receive either NAC or a placebo for 12 weeks. Researchers will measure changes in…

This study tests two different exercise programs—SPARK (a structured sports and play program) and game-based circuit training—in 22 children with autism aged 5 to 12. Over 12 weeks, researchers will measure changes in motor skills and social interaction. The goal is to find out w…

This study tests whether a non-invasive brain stimulation technique called theta burst stimulation (TBS) can improve core symptoms of autism, such as social difficulties and repetitive behaviors. Researchers will apply TBS to a specific brain area (right inferior frontal gyrus) i…

This study looks at whether playing with kinetic sand can help children with autism improve their balance and hand-eye coordination. Seventeen children aged 6 to 12 will take part in 30-minute play sessions, three times a week for six weeks. The activities include molding shapes …

This study tests whether Ayre's Sensory-Motor Integration Exercises, a type of play therapy, can help children with autism spectrum disorder (ASD) improve their sensory processing and motor skills. The trial will involve 28 children aged 9-11, with half receiving 12 sessions of t…

This study tests a new therapy for young children (ages 2-5) with autism who use few or no words. The therapy involves parents or therapists responding to the child's sounds and gently encouraging more vocalizations. Researchers will see if this approach helps children make more …

This study tests whether a therapist-guided online cognitive-behavior therapy program can help reduce obsessive-compulsive disorder (OCD) symptoms in children and teens with autism. About 220 participants will receive the online treatment or a comparison condition. The goal is to…

This study tests whether simple behavioral strategies—like social stories, videos, and positive reinforcement—can help autistic children (ages 3 and up) stay calm and cooperative during needle procedures at Boston Medical Center. Researchers will work with 10 children who have a …

This study tests whether playful physiotherapy can improve emotional self-regulation and quality of life in children aged 7-12 with ADHD or autism. Twenty-five children will take part in movement- and play-based group sessions. Parents will report changes in behavior and well-bei…

This study is testing whether a liquid form of leucovorin (a type of folate) can improve social communication and language in young children with autism. Researchers will enroll 80 children ages 2.5 to 5 years who have a specific antibody linked to folate transport. The study las…

This study tests whether guided breathing exercises can help autistic young adults (ages 16-25) manage stress and improve heart rate variability, a measure of how well the body handles stress. Participants will practice two types of slow breathing exercises and report how useful …

This study tests a new telehealth program called DINOSAUR to help young children (ages 4 to 6) with autism who also have anxiety. The program teaches children and their parents strategies to reduce worry and handle uncertainty. About 70 children will take part in this 14-week onl…

This study is testing a behavioral therapy called 'Being Brave' to help reduce anxiety in autistic children aged 3 to 6. The therapy involves 16 weekly sessions with parents and children, using visual aids and practice exercises. Researchers will also look at biological markers t…

This Phase 2 study tests whether TB006, an antibody that targets a protein called Galectin-3, can improve core symptoms of autism in adults aged 18 to 35. Forty-five participants will be randomly assigned to receive either TB006 or a placebo for 14 weeks, with an open-label exten…

This study tests if giving a sedative nose spray (dexmedetomidine or esketamine) before anesthesia can reduce negative behavior changes like sleep problems or anxiety in children with autism after surgery. About 234 children aged 2-12 will be randomly assigned to receive one of t…

This pilot study tests a digital coaching program for autistic young adults aged 18 to 35. The program uses two conversational agents to deliver personalized goal-setting and support based on a proven model called COMPASS. Over 10 weeks, 38 participants will use the app to work o…

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 compares two therapy programs designed to help autistic people manage their emotions. One program (EASE) was created specifically for autistic individuals, while the other (Unified Protocol) is a general therapy. Researchers want to see which one works better in real-w…

This study tests a telehealth program that coaches parents of children with autism to improve their child's communication and behavior while reducing parental stress. Researchers will train community providers to deliver the coaching to 90 families, including those in remote area…

This study tests two approaches in public schools: a teacher-led program (RUBIES) to reduce challenging behaviors in autistic children, and a leadership strategy (HELM) to help schools keep using effective methods. About 373 teachers and students in grades K-5 will take part. The…

This study tests a drug called DT402 (a form of MDMA) in 20 adults with autism spectrum disorder. The goal is to see if it can reduce the severity of social and communication problems. Participants will take the drug and report their experiences using simple rating scales. It is …

This study tests a Spanish-language program called Unstuck and On Target for children ages 8-11 with autism and/or ADHD. The program helps kids improve skills like planning and flexibility, and supports their caregivers. About 40 families will either join the program right away o…

This study tests a special speech therapy for minimally verbal children who have both autism and childhood apraxia of speech (a motor planning disorder). The therapy uses motor learning principles in a natural play-like setting. Researchers will enroll 20 children to see if this …

This study tests whether a mindfulness program delivered over Zoom can reduce anxiety and depression in autistic teenagers. About 160 teens and their caregivers will either join weekly group mindfulness sessions or use a meditation app on their own. The goal is to see if the grou…

This study tests whether a drug called pimavanserin can reduce rigid, repetitive behaviors in people with autism. About 30 adolescents and adults aged 12 to 40 will receive either the drug or a placebo, then switch. The goal is to see if it improves flexibility and reduces compul…

This study tests whether combining two types of exercises—sensory motor training and special stretches—can reduce toe walking and improve foot posture in children with mild autism. Researchers will measure changes in foot position and sensory responses in 36 children. The goal is…

This study tests if a marijuana-based drug (mostly CBD with a little THC) can reduce anxiety in adults with autism. About 108 people aged 18 to 45 will take the drug or a placebo daily for 8 weeks. Researchers will track anxiety, social skills, and repetitive behaviors through we…

This study tests an oral medicine called bionetide in 210 girls and women with Rett syndrome, a rare genetic disorder. The goal is to see if bionetide improves behavior and communication compared to a placebo over 12 weeks. Participants must be 5 to 20 years old and have a confir…

This study tests whether diphenhydramine (Benadryl) can help children and teens with autism fall asleep faster and sleep more soundly. Twenty-six participants aged 8 to 17 will receive either Benadryl or a placebo in a crossover design, with sleep measured by brain waves and acti…

This study tests whether suvorexant (Belsomra) can improve sleep in teenagers with autism spectrum disorder. About 26 teens aged 13-17 will receive either the drug or a placebo in a crossover design over 8 weeks. Researchers will measure sleep quality using brain wave recordings …

This study tests whether zolpidem, a sleep medication, can improve sleep in children and teens with autism. About 26 participants aged 8-17 will receive either the drug or a placebo for 8 weeks, then switch. The goal is to see if the drug helps them fall asleep faster and sleep m…

This study compares online parent coaching to in-person coaching for improving social communication in toddlers with autism or social communication delays. About 188 children aged 18-33 months will take part. Parents learn techniques to engage their child during play, aiming to i…

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 is testing whether a non-invasive brain stimulation technique called repetitive transcranial magnetic stimulation (rTMS) applied to the cerebellum can improve cognitive deficits and mood in people with schizophrenia, autism, bipolar disorder, depression, or Parkinson's…

This Phase 3 trial tests whether pitolisant can reduce excessive daytime sleepiness in people aged 6 and older with Prader-Willi syndrome. About 134 participants will receive either pitolisant or a placebo, and researchers will also look at effects on behavior and appetite. The s…

This study tests whether a non-invasive brain stimulation technique called rTMS can safely and effectively reduce depression in autistic youth aged 16-24. Eighty participants will receive either real or sham rTMS over 30 sessions. The goal is to see if this approach, already used…

This study looks at whether adding a therapy dog to a group behavioral program helps children with autism manage their emotions better. About 240 children with autism who have trouble controlling emotions will take part. The program teaches emotion regulation skills, and some chi…

This study compares two parenting programs for parents of children aged 2-6 with autism spectrum disorder. One is a structured 13-week group program (Incredible Years), the other is a shorter workshop series. The goal is to see if the longer program reduces parent stress and impr…

This study is testing two group programs for autistic middle-schoolers who are fully included in regular classes. One program focuses on organization, planning, and study skills; the other on social skills. 224 teens and their parents will be randomly assigned to one of the two p…

This study tests a telehealth program called STRW-T that teaches daily living skills like laundry, cooking, and money management to autistic adolescents in their last two years of high school. The program involves 15 weekly group and one-on-one sessions via Zoom. Researchers will…

This study tests whether a modified sports program (Sports Stars Brazil) combined with a context-focused intervention (PREP) can help adolescents with autism spectrum disorder (ASD) become more physically active and engaged. The trial will include 52 teens with ASD and measure ch…

This study tests a new therapy called TUNE In for adults with autism who struggle with social situations. The therapy helps with social motivation, anxiety, and skills. About 40 adults will take part to see if it improves their social functioning.

This study tests whether high-dose vitamin B6 supplements can reduce anxiety and sensory over-reactivity in autistic adults. Researchers will compare a group taking vitamin B6 to a group taking a placebo. The trial involves 40 participants aged 18 to 60 with an autism diagnosis.

This study tests whether a postbiotic dietary supplement called PostbiotiX Comfort® can improve gut and behavioral symptoms in children with autism spectrum disorder (ASD). Researchers will give the supplement daily for one month to 90 children aged 3-8 years. They will measure c…

This study looks at whether Adderall XR can reduce ADHD symptoms in children and teens (ages 8-18) who also have autism. Participants will take the medication or a placebo for 4 weeks and complete brain scans to measure changes in attention, impulsivity, and memory. The goal is t…

This study tests whether a team of a psychologist, speech therapist, and motor therapist can help children aged 2-7 with neurodevelopmental disorders eat more foods and have fewer mealtime struggles. 24 children will be split into two groups: one gets the team therapy, the other …

This study tests whether immersive virtual reality (VR) therapy can improve cognitive, motor, and social skills in children aged 8 to 18 with ADHD, autism, or cerebral palsy. Participants will use the CAREN VR system, which provides interactive exercises with visual, sound, and t…

This study looks at whether playing pickleball twice a week for 8 weeks can improve movement, sleep, mood, and core autism traits in adults aged 18-45 with autism spectrum disorder. Participants are randomly assigned to start pickleball right away or after a delay. The goal is to…

This study tests whether a safe, low-level brain stimulation technique called tDCS can help children with autism who are not making enough progress in their learning programs. The study will involve 24 children aged 5 to 12 who are already in behavioral therapy. They will receive…

This study tests a technique called Wilbarger Deep Pressure and Proprioceptive Technique (DPPT) in 40 autistic children aged 6-10 who have trouble with motor skills and sleep. The therapy involves gentle brushing, joint compressions, and calming activities for 30-45 minutes, thre…

This study tests whether sensory exercises can improve repetitive behaviors, communication, social interaction, and motor skills in children with autism. About 26 children aged 5 to 11 will take part. Researchers will measure changes using standard questionnaires before and after…

This pilot study tests a 16-week telehealth program that coaches parents to help their autistic children who struggle with insistence on sameness—like needing things to stay the same or getting upset over changes. Researchers will see if parents can learn the techniques and if ch…

This study tests whether a smartphone app, with or without personal coaching by phone, can improve sleep in children aged 6–12 with autism and insomnia. Parents use the app to learn behavioral sleep strategies. The trial will enroll 195 families and also measure effects on childr…

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 study tests whether a noninvasive brain stimulation technique called rTMS can improve social abilities in adults with autism. Researchers will measure brain responses and eye gaze patterns before and after treatment. The goal is to see if this approach can ease core social c…

This study tests whether adding a special armband that turns heart rate into game rewards can help children with autism (ages 6-10) better control their emotions and improve social skills during play therapy. Sixty-eight children will be randomly assigned to either standard play …

This study tests whether a 6-week virtual magic trick training program can improve social and emotional skills in adolescents with autism. Sixty teens aged 11-18 will learn tricks from a magician and practice presenting them. Researchers will measure changes in social knowledge, …

This pilot study tests whether a combination of two natural compounds, L-theanine and paraxanthine, can temporarily improve attention and self-control in adults with ADHD or autism. About 24 participants will complete thinking tests and brain scans after taking the supplement or …

This study tests whether a non-invasive brain stimulation technique called tDCS, combined with computer-based cognitive training, can improve executive function (like planning and focus) in children with autism spectrum disorder. 90 children aged 8-12 will be randomly assigned to…

This study tests whether a short program of aerobic exercise, combined with parent-led sleep training, can improve sleep and behavior in children with autism who have sleep problems. About 120 children aged 3-11 will take part. The exercise sessions are twice a week for 30 minute…

This study tests whether a simple, non-drug method called Social Stories can help children with autism feel less anxious and cooperate better when getting anesthesia for surgery. Forty children aged 3 to 18 will either read a special story at home for 1-2 weeks before surgery or …

This study tests whether virtual reality (VR) can help children with autism learn to recognize and understand emotions. Thirty children aged 7 to 10 will either use an Oculus VR headset or traditional methods to practice identifying feelings like joy, anger, and shame. The goal i…

This study tests whether omega-3 supplements can reduce behavioral problems in children and teens with autism spectrum disorder (ASD). Over 12 weeks, 50 participants aged 6-17 will take omega-3 or a placebo. Researchers will measure changes in behavior, attention, and blood marke…

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 …

This study tests whether a gentle brain stimulation technique called tDCS, given while doing social learning tasks on a computer, can reduce anxiety and improve social skills in adults with autism. Twenty high-functioning adults will receive both real and fake (sham) stimulation …

This study tests whether a common probiotic (Lactobacillus rhamnosus GG) can reduce stomach issues like constipation and diarrhea in children with autism spectrum disorder. About 50 children aged 4 to 12 with both autism and ongoing stomach problems will receive the probiotic or …

This study tests whether training caregivers of young autistic children (18–72 months) in South Africa can improve the child's communication and daily living skills. Non-specialist coaches teach caregivers strategies they can use during everyday activities. The goal is to support…

This study is testing whether a daily probiotic supplement can reduce autism symptoms, improve quality of life, and ease stomach problems in children with Autism Spectrum Disorder. About 110 children aged 7 to 15 will take the probiotic or a placebo for 3 months. Researchers will…

This study tests a non-invasive device that gently stimulates the vagus nerve through the ear to see if it can help ease symptoms of autism, such as anxiety and social difficulties. The trial will include 20 people aged 7 to 26 with an autism diagnosis. The main goal is to see if…

This study tests a new approach called ENGAGE, where children with disabilities (like autism or cerebral palsy) help set their own therapy goals. About 96 kids aged 5-12 and their therapists will be split into two groups: one using ENGAGE and the other using usual therapy. Resear…

This phase 2 trial tests whether suramin (KZ101) can improve social skills in 45 boys aged 5–14 with autism. Participants receive either suramin or a placebo by IV, then switch after a period. The main goal is to see changes in social behavior using a standard test. The study bui…

This study tests a mobile team of pain specialists who visit children and young adults (up to age 25) with moderate to severe intellectual disabilities, autism, or multiple disabilities. The team works with families and caregivers to assess and manage pain in the patient's usual …

This study tests a chatbot that delivers positive psychology exercises to reduce stress and improve well-being in parents of children with autism. About 130 caregivers will be randomly assigned to use the chatbot or receive standard information. The goal is to see if this easy-to…

This study tests whether a noninvasive technique called transcranial magnetic stimulation (TMS) can change brain activity related to hunger and fullness in people with Prader-Willi syndrome. Researchers will stimulate a part of the brain called the cerebellum and measure changes …

This study tests whether repetitive transcranial magnetic stimulation (rTMS) can safely and effectively treat depression in adults with autism spectrum disorder. Twenty-five participants will receive 20 sessions of rTMS over five days. The main goal is to see if mood improves aft…

This study tests a gentle brain stimulation technique called tDCS in 150 teens with autism. The goal is to see if it can improve social skills, thinking, and behavior, and whether extra 'booster' sessions help the effects last longer. Participants will receive multiple sessions o…

This study tests whether a speech therapy that involves parents can improve early social and communication skills in children aged 6 months to 5 years with neurodevelopmental disabilities. One group gets the parent-focused therapy, while the other gets standard speech therapy. Re…

This study tests whether a non-invasive brain stimulation technique called tDCS can improve thinking and social skills in 90 adolescents with autism. Participants will receive multiple sessions of tDCS, and some will get booster sessions to see if benefits last longer. The goal i…

This study tests a digital therapy called NDTx-02, designed to help children aged 5 to 12 with ADHD or autism improve their executive function—skills like planning, focus, and self-control. The study will involve 48 participants and compare the digital therapy to standard care. T…

This study tests whether a cannabis oil containing CBD, terpenes, and a small amount of THC can reduce challenging behaviors and social difficulties in children with autism. About 78 children aged 4 to 12 will take either the cannabis oil or a placebo for 2 months, then all may r…

This study tests whether cannabidiol (CBD) pills can reduce anxiety, mood problems, sleep issues, and agitation in adults with autism spectrum disorder (ASD). 40 adults with ASD will receive CBD for 6 weeks and a placebo for 6 weeks, with a 2-week break in between. Researchers wi…

This study tests a 10-week group program called SDARI that uses acting games and activities to help autistic young adults (ages 18-30) feel more connected and improve their social relationships. Half the participants will do SDARI, while the other half does other structured activ…

This Phase 2 trial tests whether cannabidivarin (CBDV), a compound from cannabis, can reduce irritability and repetitive behaviors in children with autism spectrum disorder. About 100 children aged 5 to 18 will receive either CBDV or a placebo daily for several weeks. Researchers…

This study tests whether a special light therapy device (transcranial photobiomodulation) can reduce autism symptoms in 80 children aged 2 to 8. Half of the children will receive the real treatment, and half will receive a sham (fake) treatment. Researchers will measure changes i…

This study tests a training program for caregivers of adolescents (ages 10-17) with profound autism. The training teaches caregivers how to use picture-based activity schedules to help their child complete tasks on their own. Researchers will see if the training is practical and …

This study tests a device worn on the forehead during sleep to see if it can reduce ADHD symptoms in children with autism. About 60 children will use the device nightly for 8-12 weeks. Half will start with a real device and half with a fake one; later, those who got the fake one …

This study tests a smartphone app called GAINS that guides caregivers step-by-step through teaching life skills to children with autism, similar to how a GPS gives driving directions. The app breaks down complex tasks into simple steps and provides audio and visual cues. Research…

This study tests three types of talk therapy to help autistic teens and young adults (ages 16-25) feel more motivated to complete daily living skills, like chores or self-care. Participants choose a goal and bring a family member or friend to support them. The goal is to see if f…

This study in Norway is testing whether nature-based group activities—like outdoor education and therapy—can improve quality of life for 240 children and youth with ADHD, autism, or Tourette's. Participants will take part in 10- to 12-week programs in schools or healthcare settin…

This study tests a 10-session group therapy designed to help teens aged 15-18 with autism who also have anxiety or depression. The therapy focuses on changing how they think about their own thoughts. Researchers want to see if the therapy is practical, acceptable, and shows promi…

This study compares two well-known therapies—cognitive-behavioral therapy (CBT) and mindfulness-based therapy (MBT)—to see which works better for autistic adults with depression and anxiety. About 300 participants from North Carolina and Virginia will receive therapy via teleheal…

This study tests whether a 'social story' — a short, step-by-step picture story — can help autistic preschool children feel less anxious and more cooperative during dental checkups. 106 children in Hong Kong will be split into two groups: one gets the dental story, the other gets…

This study tests a remote therapy model called STAR for toddlers with autism. 110 children will be split into two groups: one receiving STAR therapy plus community care, and the other receiving only community care. The goal is to see if STAR improves social and communication skil…

This study tests a 12-week group therapy program called Launching! to Adulthood for young adults with autism. The program includes weekly group sessions and individual coaching to improve daily living skills, social abilities, and readiness for adulthood. Researchers will compare…

This study follows 450 babies born with serious heart defects to find early signs of developmental delays by 6 months of age. Researchers want to understand why over half of these children face learning, thinking, or behavior challenges later in life. The goal is to identify risk…

This study creates a large registry of people with Rett syndrome, a rare genetic disorder that causes severe intellectual and physical disabilities. Researchers will collect information from doctors and caregivers to understand how the disease progresses over time. The goal is to…

This study screens up to 5,000 healthy volunteers and people at risk for psychiatric or neurodevelopmental disorders like autism. Participants undergo interviews, cognitive tests, and other evaluations to see if they qualify for future research studies at NIH. The goal is to buil…

This observational study aims to understand why some people are more impulsive or compulsive than others. Researchers will use brain scans, genetic tests, and behavioral surveys in 1,100 participants aged 6 to 80, including those with ADHD, OCD, autism, and conduct disorder. The …

This study aims to learn more about childhood-onset behavioral, psychiatric, and developmental disorders by observing people of all ages who have these conditions, along with their family members. Researchers will collect medical histories, perform psychiatric assessments, and ma…

This study follows 1,200 families who already have a child with autism (high-risk group) and 500 families from the general population (low-risk group) starting from early pregnancy. Researchers collect biological samples and survey data to explore how genetics, environment, diet,…

This study tests whether eye-tracking tasks can detect changes in attention and learning in people with Rett syndrome who are starting a new medication called trofinetide. Researchers will compare eye movements and heart rate before and after 4 weeks of treatment in 20 participan…

This study looks at how using single words or short phrases (instead of full sentences) affects language processing and word learning in young children with autism, ages 1 to 4. Researchers will show children screen-based tasks and track their eye movements to see which type of l…

This study looks at how the brain responds to sensory inputs like sight and sound in 200 children and adults, some with neurodevelopmental disorders (NDD) and some without. Participants will have EEG recordings during sensory tasks, complete behavioral tests, and practice percept…

This study explores whether autistic adults have too much 'plasticity' (flexibility) in the part of the brain that controls movement, and if a non-invasive brain stimulation technique called rTMS can reduce that over-plasticity to improve motor function. Researchers will compare …

This pilot study will screen 150 homeless adults in French shelters for autism, ADHD, and intellectual disability using interviews and questionnaires. The goal is to find out how many have these conditions, which may have contributed to their homelessness. Results could help tail…

This study is testing a new, quicker way to diagnose autism in toddlers aged 16-30 months who are already flagged as high risk. Researchers will track how many families are referred, agree to join, and complete the process, including a telehealth option. The goal is to see if thi…

This observational study follows up to 2,000 people with Williams syndrome or related chromosome changes, along with their family members. Researchers will track health, development, behavior, and quality of life from childhood through adulthood. They are also building a biobank …

This study compares two strategies for teaching new words to autistic children aged 4 to 10: repeating words (re-study) versus quizzing after labeling (repeated quizzing). Researchers want to see which method helps children learn nouns and adjectives better and whether they can u…

This study looks at facial features of males aged 2 to 40 with creatine transporter deficiency (CTD), a genetic disorder that causes intellectual disability, seizures, and behavioral issues. Researchers will examine photos of participants to see if they share common facial traits…

This study aims to learn how sleep changes as children's brains grow, by collecting sleep and development data from healthy children and those with conditions like autism or ADHD. About 244 children ages 6 months to 8 years will have overnight sleep studies, developmental tests, …

This study follows 50 children and adults with Baker Gordon Syndrome, a rare genetic disorder, to track how the condition changes over time. Participants will undergo brain scans, EEGs, and genetic testing, and complete developmental assessments. The goal is to create better tool…

This study looks at people who have changes in a gene called FOXP1, which can cause developmental delays, speech problems, and autism-like traits. Researchers will use interviews, play-based assessments, and genetic tests to better understand these conditions. The goal is to lear…

This study tests whether an online training course can teach parents of children with autism (ages 2-5) a therapy technique called Pivotal Response Treatment. Researchers will check if parents learn the technique and if children's communication improves. 44 families will particip…

This study aims to find the best way to train early intervention providers to deliver high-quality services to children under 3 with communication delays or autism. Researchers will compare two training models for a program called Project ImPACT, measuring how well therapists lea…

This study looks at whether sensitivity to rhythm can predict how well autistic toddlers respond to a social-communication therapy. Forty toddlers aged 18-36 months will receive either standard imitation training or a music-enhanced version. Researchers will track eye gaze to see…

This study tests a new training program called EMIT that helps young autistic children learn to imitate others. Imitation is a key skill for learning and social interaction. The program uses play-based exercises to teach children how to copy actions, sounds, and object use. The s…

This study aims to improve how healthcare professionals recognize autism in older adults and to test a psychoeducation program for those diagnosed. Around 120 participants aged 55 and older with an autism diagnosis will take part. The goal is to see if better training for doctors…

This study looks at how a single dose of N-acetylcysteine (NAC), a common supplement, changes brain chemistry in children with autism who have repetitive behaviors. Researchers will use brain scans and EEG to measure these changes. The goal is to understand why NAC might help red…

This study tests a smartphone app called Footsteps that helps school aides track behaviors of children aged 4-17 with conditions like autism, ADHD, or oppositional defiant disorder. The goal is to see if the app improves how consistently and accurately aides record data, and whet…

This study uses PET and MRI scans to look at serotonin pathways in the brainstem of adults with Dravet syndrome, a severe form of epilepsy. Researchers want to see if these pathways are different in Dravet patients compared to those with other drug-resistant epilepsy and healthy …

This observational study uses a noninvasive EEG test to measure how the brains of people with Rett syndrome respond to simple sounds. Researchers will also ask parents or caregivers about symptoms and daily functioning. The goal is to better understand brain activity patterns in …

This study looks at how autistic people who also have anorexia nervosa differ in brain structure and behavior from autistic people without anorexia. Researchers want to understand if 'masking' (acting non-autistic to fit in) is linked to eating disorder symptoms and brain changes…

This observational study is looking at 1,500 people with autism to find biological and clinical markers that define specific subtypes. The goal is to identify which patients might respond best to certain medications. Participants must have a formal autism diagnosis and detailed e…

This study tests a remote training program for autistic teens and young adults (ages 13-21) and their primary caregiver. The program teaches strategies to improve thinking and daily life skills. The goal is to see if the program is practical and helpful for participants.

This pilot study is testing a program called CRUSH, which is a sexual health curriculum designed specifically for autistic adults aged 18 to 30. The program includes group sessions and one-on-one coaching to teach about relationships and sexual health. The goal is to see if the p…

This study aims to find biological markers in the blood that may help explain brain development disorders like autism, epilepsy, and brain tumors. Researchers will compare children with and without these conditions to identify unique patterns. Up to 500 children will participate …

This study watches how stress affects children and young people with autism who have trouble communicating. Researchers use a wrist device to track heart rate and sweat, plus saliva samples to measure stress hormones. They compare these signals to behavior in real-life settings l…

This study looks at whether training mental health therapists in a program called Unstuck and On Target helps children with autism improve their executive functioning (skills like planning and flexibility) and reduce behavior problems. About 672 children, their caregivers, and th…

This study looks at medical records of 1000 children with conditions like autism, epilepsy, and Down syndrome to find common biological patterns. Researchers want to understand what causes these disorders and how children respond to treatments. The goal is to improve diagnosis an…

This study aims to better understand how children aged 8 to 16 with neurodevelopmental disorders (like autism or ADHD) process social information. Researchers will use a new set of tests to measure skills like recognizing emotions and understanding others' thoughts. The goal is t…

This study tests a two-week online program designed to help kindergarten and elementary school teachers better support autistic children and those with developmental delays. About 70 public school teachers will either take the program or receive standard materials. Researchers wi…

This study looks at how a brain chemical called GABA may affect the way people with autism process sound. Researchers will use brain scans and magnetic pulses to measure brain activity in children and young adults. The goal is to better understand the brain, not to provide treatm…

This study tests if wearable sensors can be used to monitor movement and sleep in children with autism. Researchers will ask 80 children aged 2 to 18 to wear a wrist sensor for 7 days and do a one-night sleep test at home. The goal is to see if the sensors are comfortable and pro…

This study tests an online training program called WE PLAY for Parents, designed to help parents of young children with autism (ages 3-5) encourage more physical activity. About 114 parents will be randomly assigned to take the training right away or wait 3 months. Researchers wi…

This study explores whether playing specially designed sounds through headphones can normalize brain activity in children aged 5-10 with Fragile X Syndrome or autism. The goal is to reduce sensory sensitivity and improve learning. Participants will listen to sounds while their br…

This study looks at when to start a parent coaching program called JASPER Babble for infants who have an older sibling with autism, putting them at higher risk. About 140 babies will join at 6-8 months old and be randomly assigned to start coaching at 9, 12, or 15 months. Researc…

This study looks at videos and sounds from babies to find early clues about conditions like cerebral palsy and autism. About 46 infants who were in the NICU will have their movements and cries recorded at home. Researchers hope to identify patterns that could lead to earlier dete…

This study looks at whether a non-invasive brain stimulation technique called TMS can change brain activity related to understanding others' thoughts (mentalizing) in young adults with autism. Researchers will compare brain scans before and after stimulation in 80 participants, i…

This study looks at how common central sensitization (a condition where the nervous system is overly sensitive to pain) is in adults with autism who do not have intellectual disabilities. Researchers will ask 100 participants to fill out a questionnaire about pain and sensitivity…

This study looks at how a 2-week, personalized therapy program helps children aged 4-12 with conditions like cerebral palsy, autism, or genetic disorders. Kids get about 2.5 hours of tailored therapy each day, focusing on skills like movement, attention, and communication. The go…

This study looks at sleep disorders in preschool-aged children (18-60 months) with autism. Researchers will collect data from routine care and measure blood levels of vitamin D and iron to see if they relate to sleep issues. The goal is to better understand sleep problems in this…

This study uses magnetoencephalography (MEG), a non-invasive brain scan, to compare brain signals in 1,500 children with neurodevelopmental disorders (like epilepsy, autism, ADHD) and healthy children. The goal is to improve diagnosis and understanding of these conditions. No tre…

This study looks at ways to help families show up for their scheduled appointments at clinics for autism and other neurodevelopmental disorders. Researchers will test different types of reminder messages and see if having a patient navigator call high-risk families makes a differ…

This study looks at how the brain processes speech when there is background noise, in people with autism, Fragile X syndrome, and those without either condition. About 60 participants will complete hearing tests, questionnaires, and brain-recording tasks while listening to sounds…

This study looks at how children with autism spectrum disorder (ASD) move, including their fine motor skills, walking, and eye movements. Researchers will compare 55 children with ASD to 55 children without ASD, all aged 6 to 11. The goal is to identify specific motor patterns th…

This study watches how children and adults with autism behave and feel during a routine blood test compared to people without autism. Researchers will record facial expressions, body movements, heart rate, and stress levels. The goal is to learn what makes blood tests harder for …

This study is collecting genetic information from 2,000 infants in China who had seizures before their first birthday. Researchers aim to find gene changes linked to seizures and see if personalized treatments can help control them better. The goal is to understand the genetic ca…

This study follows 100 infants and toddlers with either a high or low family risk of autism spectrum disorder (ASD). Researchers will track how prosocial behaviors—like helping and understanding others—develop, along with brain network changes measured by EEG and MRI. The goal is…

This study aims to build a database of genetic information from 1,000 people with intellectual disabilities or autism spectrum disorders of known genetic cause. Families will answer online questionnaires to share clinical details, helping researchers understand how different gene…

This study aims to identify sensory patterns in autistic individuals from childhood to adulthood. Researchers will use questionnaires and brain imaging (fMRI and EEG) to explore how sensory experiences relate to social challenges like avoiding eye contact or touch. The goal is to…

This study follows 1,000 children and teens with rare epilepsies (like West and Dravet syndromes) to see how different treatments affect their seizures and thinking skills. Researchers will compare care across hospitals to find what works best. The goal is to give families cleare…

This study collects blood samples from 100 children aged 3-15 with autism to create personalized cell models in the lab. Researchers will analyze these cells to understand biological differences that may contribute to autism. The goal is to gather knowledge that could one day lea…

This study tests new tools to better understand the needs and strengths of children with autism, aged 0-11, in everyday healthcare settings. Researchers want to see if these tools help doctors and families make better care decisions together. The goal is to improve quality of lif…

This study tracks children and adults with genetic developmental and epileptic encephalopathy (DEE) over time. It does not test any treatment but collects information on development, seizures, and quality of life through in-person visits, virtual visits, or online surveys. The go…

This study is testing a program that helps teachers and parents work together to support preschoolers who have trouble with social communication. The program is run by preschool staff and uses proven teaching methods. Researchers want to see if the program is easy to use and if f…

This registry collects information from caregivers of people with Rett syndrome worldwide. Participants can track symptoms and treatments online, and researchers can use the de-identified data to better understand the condition and plan future clinical trials. The goal is to spee…

This observational study will follow 2000 children and adults with imprinting disorders—rare genetic conditions like Silver-Russell and Prader-Willi syndromes. Researchers aim to describe the natural history of these diseases and identify common metabolic profiles, risks for obes…

This study looks at balance problems in children with autism aged 6 to 10. Using a virtual reality system, researchers will measure how kids stand still and see if balance is linked to thinking skills. The goal is to better understand these challenges, not to test a treatment.

This study compares children with autism spectrum disorder (ASD) to typically developing children, aged 3 to 5 years. Researchers measure a substance called N-CAD in the blood and use a non-invasive brain imaging technique (fNIRS) to look at brain activity during rest and social …

This study follows 120 people with STXBP1-related disorders, a rare genetic condition causing severe developmental delays and seizures. Researchers aim to track how the disease changes over time and identify the best ways to measure improvement. This work will help design future …

This study looks at how movement and sensory differences in children with autism might serve as early signs of the condition. Researchers will follow 300 participants, including autistic children, their parents, and infants at higher risk, over time. The goal is to link these phy…

This study follows 50 children and young adults with Dravet syndrome caused by SCN1A gene mutations over four years. Researchers will use standard tests to measure changes in thinking, movement, and daily skills. They will also look for chemical markers in the blood that might re…

This study looks at whether trouble processing sounds (called central auditory processing disorder, or CAPD) causes poor sleep in children and teens with autism. Researchers will compare two groups of 100 kids with autism—those with CAPD and those without—using parent surveys and…

This study explores the brain basis of social difficulties in 30 teens with autism and 30 with schizophrenia. Participants take part in a 16-week social skills program called PEERS, and undergo brain scans before and after to see how the intervention affects brain networks. A con…

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 tests an online program called OPT-In-Early for parents of 120 toddlers (16-48 months old) on waitlists for an autism diagnosis. The program provides training and support to help parents address their child's developmental needs before a formal diagnosis. Researchers w…

This study is creating a large database to collect information on people with rare diseases like amyloidosis, sarcoidosis, and Gaucher disease. Researchers will track patients' health over time, including their symptoms, treatments, and outcomes. The goal is to improve diagnosis …

This study looks at how autism affects motor functions like strength, balance, and coordination in adults aged 18 to 45. Researchers will also test whether a 6-week virtual reality training program can help improve these skills. The goal is to better understand the link between m…

This study observes 100 people with rare genetic neurodevelopmental disorders like Baker Gordon Syndrome and Syt-1 disorder. Researchers will track symptoms, collect genetic and imaging data, and create cell models to better understand these conditions. The goal is to improve dia…

Researchers at Boston Children's Hospital are recruiting 5,000 people with epilepsy to study their genes. By comparing DNA from patients and their parents, they hope to find genetic changes linked to epilepsy. This study does not test a new drug or treatment, but aims to better u…

This study pairs autistic adults (ages 18-36) with a non-autistic learning partner, like a parent. The autistic adult takes emotional intelligence training, while the partner learns how to better accommodate autism. Both then rate how well the trainings worked for themselves and …

This study is looking at the DNA of 3,800 people with autism to find genetic changes that might be linked to the condition. Researchers hope to better understand which genes are involved and how they affect brain function. The goal is to lay the groundwork for future treatments, …

This study aims to understand brain differences in people with autism spectrum disorder. Researchers will use MRI scans and eye-tracking to see how the brain works and how people with autism process social information. The study involves 160 participants, including children and y…

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 is looking at how people with conditions like schizophrenia, bipolar disorder, depression, and neurological diseases (such as Alzheimer's or stroke) perform on computer-based thinking tests. The goal is to create a detailed map of cognitive strengths and weaknesses acr…

This study looks at whether using a preferred communication method (like a touch talker or picture cards) helps people with intellectual or developmental disabilities maintain positive behavior changes and avoid relapse. Researchers will compare two groups: one starting with a pr…

This study tests a dual executive function training program for 288 primary school children aged 6-12 with ADHD, dyslexia, or autism. The program includes weekly group sessions to improve thinking, emotional control, and behavior. Researchers will measure changes in memory, atten…

This study is creating a registry for children with PTEN Hamartoma Tumor Syndrome (PHTS), a rare genetic condition that increases cancer risk. Researchers will follow 100 participants for 3 years to link genetic changes with health outcomes. The goal is to develop better monitori…

This early-stage trial is testing a new drug called ABS-1230 in 74 healthy adults to see if it is safe and how the body processes it. The drug is being developed for a rare type of epilepsy caused by changes in the KCNT1 gene. Participants will receive either the drug or a placeb…

This study is creating a registry of 200 patients with a rare, severe epilepsy that starts in the first month of life, often linked to a mutation in the KCNQ2 gene. Researchers will collect data on seizures, brain activity, development, and long-term outcomes through questionnair…

This study looks at whether brain scans can predict which young children with autism (ages 2 to 4) will improve their language skills after 16 weeks of Pivotal Response Treatment (PRT), a behavioral therapy. About 36 children will be randomly assigned to receive PRT right away or…

This study is observing 600 people of any age with STXBP1 or SYNGAP1 gene mutations to better understand how these disorders affect development, seizures, and quality of life. No treatment is given; instead, researchers will collect data from routine clinical assessments over tim…

This study follows 1,000 people who have certain genetic changes (hnRNP and others) that may cause developmental delays, autism, seizures, or muscle problems. Researchers collect medical records, school reports, and questionnaires to see how these gene variants affect behavior, t…

This study tests whether virtual coaching, called Family Navigation, helps families in rural North Carolina start autism therapy faster for toddlers under 4. Some families get 4 telehealth sessions with a trained navigator, while others receive standard educational materials. The…

Researchers at UT Dallas are studying whether 'parentese' — the sing-song, high-pitched speech parents often use with babies — helps autistic children learn new words. The study will involve 100 children aged 18 to 59 months, with and without an autism diagnosis. Children will wa…

This study looks for brain activity patterns (biomarkers) in girls with Rett syndrome. Researchers will use EEG to measure brain waves and see if these patterns change with symptoms or stay stable over time. About 200 girls with Rett syndrome and typically developing girls will t…

This study looks at how a brain area called the cerebellum influences social and thinking abilities in young adults with and without autism. Researchers will use a gentle brain stimulation technique and brain scans to see how it changes these skills. The goal is to better underst…

This study tests whether a gentle, non-invasive brain stimulation technique called tDCS can improve cognitive functions like reaction time, memory, and multitasking in 90 adolescents with autism (ages 12–22). Each participant will receive three types of stimulation (including a s…

Researchers at Semmelweis University are studying 600 children aged 1.5 to 6 years in Hungary to see how common autistic traits and feeding difficulties are, and whether they are linked. The study includes children with and without autism. The goal is to gather information, not t…

This study tests whether a child-focused goal-setting method called ENGAGE helps children with neurodevelopmental disabilities do better in therapy. About 96 kids aged 5-12 and their therapists will be split into two groups: one using ENGAGE and one using usual care. Researchers …

This study is testing a redesigned approach to help elementary school teachers use behavioral skills interventions for autistic children. Researchers will work with 160 participants to see if the new strategy is usable and effective. The goal is to improve access to evidence-base…

This study aims to learn more about rare disorders that affect how the body processes chemicals called pyrimidines and purines. These disorders can cause problems in the brain, blood, kidneys, and immune system, ranging from mild to life-threatening. Researchers will compare test…

This study follows 100 children under 18 who are already on a ketogenic diet for conditions like drug-resistant epilepsy, autism, chronic migraine, or brain tumors. Researchers want to see if the diet supports healthy growth, improves symptoms, and boosts quality of life. The die…