Neurodevelopmental disorder

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 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 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 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 trial tests whether a structured sensory-based occupational therapy program can improve daily functioning, sensory processing, gross motor skills, and eating behaviors in children with autism spectrum disorder. Forty children aged 3 to 9 years will receive either face-to-fac…

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 pilot study tests whether an antioxidant cocktail called Rett-T can improve motor function in children and adolescents with Rett syndrome, a rare genetic disorder that affects movement and coordination. Participants, aged 2 to 21 and at least partially able to walk, will rec…

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 pilot trial tests whether a digital inhaler cap, which gives real-time guidance on inhaler technique, can help adolescents with intellectual and developmental disabilities (IDD) manage their asthma at home. The study will compare active use of the smart inhaler to passive us…

This trial is testing the safety of a medication called lumateperone in children and teenagers who have schizophrenia, bipolar disorder, or autism spectrum disorder. Participants take the drug once daily for 26 weeks, and researchers monitor for side effects. The goal is to see i…

This study is checking the long-term safety of a drug called KarXT in teenagers (13-17) with schizophrenia, and a combination of KarXT and KarX-EC in children (5-17) with irritability linked to autism. About 400 participants who completed earlier studies will take the drug for up…

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 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 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 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 pilot study tests whether combining a social robot with board games can improve socio-emotional skills in children with autism spectrum disorder (ASD). Children aged 4 to 10 will be randomly assigned to either a robot-assisted board game group or conventional occupational th…

This trial tests whether deep brain stimulation—implanting electrodes in specific brain areas—can help people with severe, treatment-resistant mental disorders like OCD, schizophrenia, depression, anorexia, gambling addiction, and autism. The study involves 18 participants and me…

This early-phase trial is testing a new drug, FOG-001, in about 595 people with advanced or metastatic solid tumors, including colorectal, prostate, and liver cancers. The drug is given alone or with other cancer treatments to see if it is safe and shrinks tumors. The study is cu…

This trial compares two ways of feeding very preterm infants (born before 32 weeks and under 1500 grams). One group receives a short period of small 'trophic' feeds for one day before advancing to full feeds, while the other receives trophic feeds for three days. The goal is to s…

This study tests whether adding hands-on cooking classes to a standard weight loss program helps young adults (18-35) with mild-to-moderate intellectual disabilities lose more weight and keep it off. Over 24 months, 114 participants will either follow a healthy eating plan with c…

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 trial tests whether methylphenidate, a standard ADHD medication, can reduce attention and hyperactivity problems in children and adolescents with KBG syndrome, a rare genetic condition often accompanied by ADHD-like symptoms. Participants receive alternating blocks of the dr…

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 looks at two ways to treat Lennox-Gastaut syndrome (LGS), a severe form of childhood epilepsy: adding more anti-seizure medications or undergoing palliative surgery (like a vagus nerve stimulator or corpus callosotomy). Researchers will track thousands of children acro…

This study compares two types of fat infusions (mixed oil vs. soybean oil) given to extremely preterm infants to improve nutrition and reduce the risk of bronchopulmonary dysplasia (a chronic lung disease) and neurodevelopmental impairment. About 230 babies born before 28 weeks o…

This Phase 3 study tests whether the drug mavorixafor can reduce serious infections and increase neutrophil levels in people with chronic neutropenia—a condition where the body doesn't make enough infection-fighting white blood cells. About 176 participants will receive either ma…

This study tests whether playing digital brain games on the MentalUP app can improve visual memory and cognitive skills in young adults (18-35) with mild to moderate intellectual disabilities. Sixty participants will be split into two groups: one plays the games, the other contin…

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 pilot study tests a 10-session program called the Cognitive Orientation to Daily Occupational Performance (CO-OP) approach in primary school-aged children with autism. The program teaches children to use cognitive strategies like 'Goal-Plan-Do-Check' to improve performance o…

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 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 parent-led, home-based play program designed to improve social cognitive skills—like recognizing emotions and understanding others' perspectives—in young children with autism. Parents are trained to deliver daily 30-minute cooperative games for six months, with…

This study tests whether the drug Fintepla (fenfluramine) can improve how the brain and lungs respond to carbon dioxide in people with Dravet syndrome, a severe epilepsy. Poor responses to CO2 may increase the risk of sudden unexpected death in epilepsy (SUDEP). Twenty-five parti…

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 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 program offers continued access to rufinamide for children with Lennox-Gastaut syndrome, a severe form of epilepsy, who completed a previous study. The goal is to keep providing the medication until it becomes commercially available in Poland. Only children who were already …

This trial tests a single injection of MELPIDA, a gene therapy that delivers a working copy of the AP4M1 gene directly into the spinal fluid of children with SPG50. SPG50 is a rare genetic condition that causes muscle stiffness, intellectual disability, and developmental delays. …

This study tests a drug called deutetrabenazine for tardive dyskinesia (uncontrollable movements) in 25 adults with intellectual/developmental disabilities and psychiatric or behavioral disorders. Participants will take the medication for 24 weeks to see if it reduces movement pr…

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 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 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 study is for adults with recurrent glioblastoma, an aggressive brain cancer. It tests whether adding a precise type of radiation (stereotactic radiosurgery guided by a special PET scan) to a device that delivers electric fields to the brain (TTFields) helps people live longe…

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 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 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 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 program offers early access to tiratricol, a thyroid hormone-like drug, for people with Allan-Herndon-Dudley syndrome (AHDS), a rare genetic condition that affects brain development and movement. Patients must have a confirmed genetic diagnosis and be considered likely to be…

This study compares a special program called COPCA with standard physical therapy and parent education for infants under 12 months who are at risk of developmental disorders. About 40 families will take part, with some receiving in-person or online COPCA coaching, others getting …

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 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 metformin, a diabetes drug, can help adults with intellectual disabilities who have gained weight from antipsychotic medications. One hundred participants will receive either metformin or a placebo for 24 weeks, along with lifestyle advice. The goal is to…

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 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 early-stage trial tests a gene therapy called Urbagen in 12 children aged 2-12 with CTNNB1 neurodevelopmental syndrome, a rare genetic condition causing motor and cognitive delays. The therapy is given as a single infusion into the brain fluid, along with immunosuppressant d…

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 study looks at thinking and mental health issues in people with Velocardiofacial, Williams, and Fragile X syndromes. Researchers will test how well medications like methylphenidate, fluoxetine, and risperidone improve these symptoms. The goal is to better understand and trea…

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 phase 2 trial tests alpelisib, a drug originally developed for cancer, in 20 people aged 2 to 40 with MCAP syndrome—a rare condition causing an enlarged brain, blood vessel issues, and developmental delays. Participants receive either alpelisib or a placebo for 6 months, the…

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 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 trial tests a drug called DITPA in male infants with MCT8 deficiency, a rare genetic disorder that causes severe intellectual disability and movement problems. The drug aims to improve thyroid hormone function in the brain. Only infants whose families have a history of the c…

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 whether a new multivitamin supplement (MMS Plus) given during pregnancy and after birth can improve infant health and growth compared to standard multivitamins or iron/folic acid. About 3,000 pregnant women in Pakistan will take the supplements and be followed un…

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 …

Researchers are testing whether eye-tracking technology can identify behavioral and physiological markers of social differences in children with autism. The study will compare eye movements, gaze patterns, and pupil responses in children with autism and typically developing peers…

This study investigates whether adding RNA sequencing to standard DNA sequencing can help identify genetic causes of neurodevelopmental disorders that also involve physical birth defects. Many children with these conditions go years without a clear diagnosis. The research compare…

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 study tests whether giving a single dose of a common antibiotic soon after a breathing tube is placed can prevent lung infections and improve survival in ICU patients with severe brain injuries. Researchers will enroll 3,300 adults on ventilators due to stroke, trauma, or la…

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 Phase 3 trial tests whether fenfluramine (Fintepla) can reduce behavioral and emotional symptoms in people with Rett syndrome. About 200 participants will receive either the drug or a placebo for 14 weeks. The study measures changes in behavior, sleep, and overall condition …

This study tests whether buspirone, a medication used for anxiety, is safe and effective for autistic youth aged 7 to 17 who also have anxiety disorders. Researchers will measure changes in anxiety symptoms over 16 weeks. The goal is to see if this drug can ease anxiety in this s…

This trial is testing whether a digital therapy app called NDTx-03, used alongside usual care, can help children and adolescents aged 10 to 18 with autism spectrum disorder or social communication disorder better recognize and respond to social situations. The app combines cognit…

Researchers at Chang Gung Memorial Hospital are testing whether intermittent theta burst stimulation, a non-invasive form of brain stimulation, can help autistic people with minimal verbal ability or intellectual disability regulate their emotions and behavior. The study enrolls …

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 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 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 …

Researchers test whether an AI chatbot called Pai.ACT can help parents of young children with neurodevelopmental disorders like autism and ADHD. The chatbot delivers acceptance and commitment therapy, a type of talk therapy, and adapts its exercises to each parent's needs. The tr…

Researchers are testing whether an interdisciplinary case management approach built on the International Classification of Functioning, Disability and Health (ICF) improves rehabilitation for adults with mild to moderate intellectual disabilities. The trial enrolls 120 adults fro…

Autistic teens are three to four times more likely to experience depression than their non-autistic peers, yet almost no depression treatment research has focused on autistic youth with below average cognitive abilities. This small study tests a 12-session behavioral activation t…

This study tests a drug called N-acetyl-L-leucine for people aged 4 and older with CACNA1A gene disorders, which can cause coordination problems, dizziness, and migraines. About 60 participants will receive either the drug or a placebo, then switch, to see if it improves movement…

This pilot trial is testing whether a school-based program called the Melillo Method is practical and acceptable for children aged 8 and older who struggle with behavior and learning. The program combines sensory stimulation, rhythm exercises, and coordination activities in small…

This study tests an 8-week program called MH-POWER designed to help autistic college students manage stress, anxiety, and depression. The program combines cognitive behavioral therapy, acceptance and commitment therapy, and habit formation through weekly group sessions and daily …

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 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 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 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 trial tests a personalized program called TIHA for adults with autism who find everyday sounds painfully loud, a condition known as loudness hyperacusis. The program combines counseling, safe sound management, and gradual exposure to therapeutic sounds. Researchers will rand…

This study tests whether the drug lumateperone can safely reduce irritability in children aged 5 to 17 with autism spectrum disorder. Participants receive either a high or low dose of lumateperone or a placebo daily. The main goal is to see if the drug improves irritability score…

This study tests a behavioral program called KF-STRIDE that helps autistic adults recognize their strengths and talk about them appropriately in job interviews. Thirty participants will practice in mock interviews and be rated by experts. The goal is to see if the program is prac…

This study tests whether the drug lumateperone can safely reduce irritability in children aged 5 to 17 who have autism spectrum disorder. Participants receive either a high dose, low dose, or placebo daily for several weeks. Researchers measure changes in irritability using a sta…

This trial tests whether adding aerobic exercise or exergaming (active video games) to standard motor and behavioral therapy helps children with autism sleep better, move more skillfully, and think more clearly. Researchers will compare three groups of children aged 4 to 10: one …

This study tests whether dialectical behaviour therapy (DBT), adapted for autistic adults, can improve emotion regulation and well-being while reducing self-harm. Six participants with autism will undergo 10 months of DBT, tracking daily urges for maladaptive strategies like self…

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 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 early-phase trial tests whether the steroid prednisone can improve core autism symptoms in 32 adult males with an immune-mediated subtype of autism. Participants take prednisone or a placebo daily for 16 weeks, with regular check-ins and questionnaires. The goal is to see if…

This study tests whether a talking therapy called Compassion Focused Therapy (CFT) can help people with conditions like depression, bipolar disorder, or schizophrenia feel less shame and self-stigma. Participants will be randomly assigned to receive CFT, a standard psychoeducatio…

This study explores whether visiting a museum can support the social and emotional well-being of autistic adults. Researchers plan to enroll 90 autistic adults aged 18 to 50 with average intellectual ability. Participants choose one of three options: a solo museum visit, five gui…

This trial tests whether using a weighted blanket at night and during the day can improve sleep in children aged 5-12 with ADHD who still have sleep problems despite standard care. Half the children will receive a weighted blanket, while the other half gets a non-weighted sham bl…

This pilot study tests a bedtime routine called 'The Power Down' for autistic children aged 6 to 10 who have trouble settling down to sleep. The routine uses gentle massage and calming sounds to help children relax before bed. Caregivers will lead the routine each night for two w…

This study tests whether a short telehealth interview 3-7 days before a dental checkup helps patients with intellectual or developmental disabilities (IDD) have a smoother visit. About 50 youth with IDD and their caregivers will be randomly assigned to receive the telehealth call…

This study tests whether AI-powered modules, accessed through a phone or computer app, can help autistic individuals improve social communication. Participants practice skills like empathy, asking questions, and listening, receiving feedback via voice recognition. The goal is to …

This study tests whether a 16-week social skills program, called PEERS, can improve social functioning in adults with autism spectrum disorder (ASD). Researchers will measure changes in social symptoms and brain activity related to social processing. The goal is to see if this be…

This study tested an 8-week virtual reality program based on cognitive-behavior therapy to help adults with autism spectrum disorder handle sensory sensitivities. 90 participants practiced relaxation, ranked scary situations, and faced them gradually in VR. The goal was to reduce…

This trial tests a structured, parent-assisted social skills program called PEERS® for adolescents with autism spectrum disorder. The program uses cognitive-behavioral techniques to teach practical skills for making and maintaining friendships. Researchers will measure changes in…

This study tests a couple group therapy program designed for adults with autism spectrum disorder (ASD) and their partners. The program teaches communication skills like using "I" statements, focusing on conversations, and giving positive feedback. The goal is to improve relation…

This trial tests whether a structured social skills program, called PEERS, can help adults with autism improve their social interactions and relationships. Participants aged 18 to 45 with relational difficulties and a caregiver's support will either join the program or be in a co…

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 trial tests whether sertraline, a widely used antidepressant, can reduce anxiety in children and teens aged 8 to 17 with neurodevelopmental disorders such as autism, ADHD, Fragile X syndrome, and Tourette syndrome. Participants receive either sertraline or a placebo for seve…

This clinical trial tests whether a 15-week parent-led nutrition program called Exploradores de Sabores can help children aged 4 to 11 with autism spectrum disorder eat a more varied diet and improve their nutritional status. Caregivers attend weekly group sessions with a dietiti…

This trial tests whether a culturally adapted, nurse-led program called COPE can improve the psychological wellbeing of primary caregivers of children with autism spectrum disorder in Pakistan. The program includes four weekly sessions focused on coping skills, emotional regulati…

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 a new medicine called CTH120 in 30 adult men with Fragile X syndrome. The main goal is to see if the drug is safe and tolerable, while also checking if it helps improve symptoms. Participants will receive the drug and be monitored closely for side effects and cha…

This study tests whether a gentle electrical stimulation device worn on the ear can help improve social, communication, and behavioral symptoms in adults with high-functioning autism. Participants use the device at home for 30 minutes a day, up to 5 times a week, for 3 weeks. The…

This study explores whether a single session of transcranial magnetic stimulation (TMS) can reduce depression symptoms in adults with autism spectrum disorder. Researchers will use brain wave recordings (EEG) and eye-tracking to see how the brain responds to sad faces before and …

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 early-stage study tests a group program called HEART for autistic adolescents aged 13 to 18. The program focuses on improving how teens understand and regulate their emotions. Researchers will check if the training is practical and helpful, using parent questionnaires to mea…

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 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 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 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 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 whether L-dopa, a drug used for Parkinson's, can improve movement and communication in children with a rare genetic disorder called CTNNB1 syndrome. The condition causes developmental delays, muscle stiffness, and trouble walking. Seven children aged 1 to 15 will…

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 a new early intervention program called SAFE for high-risk infants (3-12 months old) who may have developmental delays. The program focuses on activities to improve arm, hand, and fine motor skills through play and daily routines. Researchers will compare SAFE to…

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 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 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 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 a program called BRIDGE for mothers with depression and their children aged 3-7 who have attention or behavior problems. The program aims to improve children's self-control and reduce problem behaviors by supporting mothers' mental health. About 60 families will …

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 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 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 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 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 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 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 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 using a CPAP breathing mask during radiation therapy for early-stage breast cancer can lower radiation doses to the heart and lungs. About 53 women with left-sided breast cancer will receive radiation with CPAP support. The goal is to reduce long-term hea…

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 whether a dental mouthpiece (MAD) works as well as a CPAP machine to improve thinking and memory in people with sleep apnea who also have mild memory problems. About 260 adults aged 45 and older will be randomly assigned to use either device for one year. Researc…

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 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 looks at whether using a CPAP machine at night can lower dangerously high blood pressure that happens when people with autonomic failure lie down. About 12 participants will use CPAP or a placebo pill/patch. The main goal is to see if CPAP reduces blood pressure while …

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 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 aims to identify the genetic causes of rare, undiagnosed disorders by using genome sequencing. Researchers will analyze the DNA of up to 2,000 participants, including affected individuals and their family members. The goal is to discover new disease-causing genes and i…

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 …

Researchers are building a global registry to collect patient-reported surveys, genetic test results, and medical records from people with Charcot-Marie-Tooth disease and related inherited neuropathies. The study is open to children and adults with a confirmed or suspected diagno…

Researchers in Guangzhou, China, are following 20,000 pregnant women and their children to study how the mix of bacteria in the mother's body during pregnancy and in the baby's gut early in life relates to health outcomes later on. The study tracks conditions such as obesity, ast…

This study aims to understand how the brain develops in children with Williams syndrome or 7q11.23 duplication syndrome, compared to healthy children. Researchers will use MRI scans and cognitive tests to track changes over time. The goal is to learn how these genetic variations …

This study compares brain responses to heat, cold, brushing, and mild electric shocks in people with and without intellectual disability. Researchers use brain scans and heart rate monitors to measure pain. The goal is to better understand how pain is felt in this group, especial…

Researchers in Guangzhou, China, are following 5,000 grandmothers, their daughters, and their grandchildren to understand how health traits pass across three generations. The study collects information on lifestyle, environment, and social support, along with blood and tissue sam…

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, …

Researchers want to know if breathing patterns recorded while premature babies are in the neonatal intensive care unit relate to asthma, sleep-disordered breathing, and neurodevelopmental problems at preschool age. The study follows up to 500 children born before 29 weeks of preg…

This study looks at whether a guided self-help workbook can help autistic adults feel better. Ten participants will use the workbook with a trained supporter over 4-6 sessions. They will fill out wellbeing questionnaires and share their thoughts in interviews. The goal is to see …

This study looks at whether obese children with autism spectrum disorder differ from those without autism in fitness and heart health. Researchers will review medical records of 200 children aged 6 to 18, including step test results, handgrip strength, body measurements, and bloo…

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 study looks at 48 teenagers with autism who were checked for psychotic symptoms 10 years ago. Researchers want to see how their mental health and daily functioning are now. They will compare those who had early signs of psychosis with those who did not, using interviews and …

Researchers are enrolling thousands of French college students to track how symptoms of anxiety, depression, ADHD, sleep problems, and other factors interact over time. Instead of counting how common these issues are, the study uses a network approach to see how symptoms influenc…

This study will follow 900 Spanish adults with intellectual disabilities for one year to understand why they fall. Researchers will check medical records, balance, and walking ability. The goal is to create a tool that can predict who is at risk of falling, so caregivers can take…

This study follows 819 children under 2 years old who have medium to giant congenital nevi (pigmented birthmarks). Researchers will track neurological development, melanoma risk, and quality of life over time. The goal is to create better monitoring and treatment guidelines for t…

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 study from the National Human Genome Research Institute aims to better understand Congenital Disorders of Glycosylation (CDG), a group of rare genetic conditions that affect how the body attaches sugars to proteins and fats. Researchers will examine up to 200 participants, i…

This observational study will follow 50 children aged 5 to 8 with non-progressive congenital ataxia, a rare condition that affects movement and coordination. Researchers will use detailed exams, brain scans, and genetic testing to better understand the disorder and its impact on …

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 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 40 people with Angelman syndrome over one year to see how their communication, motor skills, and behaviors change. Participants visit the study site five times for tests, brain activity monitoring (EEG), and sleep tracking. The goal is to better understand the …

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 1,000 children in Malawi, aged 4 to 8, whose mothers either chewed xylitol gum during pregnancy or did not. Researchers will compare brain development, thinking skills, and emotional behavior between the two groups. They will also test a low-cost assessment too…

This study aims to understand the genetic causes of brain development disorders, including Aicardi syndrome, polymicrogyria, and Dandy-Walker malformation. Researchers will analyze genetic data from 2000 participants with these conditions, all confirmed by MRI. The goal is to use…

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 looks at adults with spinal deformity to understand which factors—like pain, daily functioning, mental health, and frailty—most influence quality of life. It follows 320 people receiving either conservative care, first-time corrective surgery, or a reoperation, using q…

This study looks at how people with Down syndrome and Fragile X syndrome decide who to trust. Researchers will use eye-tracking and behavioral tasks to measure how participants read faces, behaviors, and detect deception. The goal is to understand the brain mechanisms behind trus…

This study follows people with several types of spinocerebellar ataxia (SCA) — rare genetic diseases that cause balance, coordination, and speech problems — to learn how these conditions change over time. Researchers will collect blood samples, perform neurological exams, and use…

This study follows pregnant women and their children from the first trimester through toddlerhood to explore how stress, early social experiences, and genetics interact. Using simple at-home activities like questionnaires, video recordings, and cheek swabs, researchers will look …

This study will follow 15 people aged 5 to 50 with 7q11.23 microduplication syndrome, a rare genetic condition linked to autism and social difficulties. Researchers will track developmental milestones, social skills, and behavior using interviews and questionnaires. The goal is t…

This study examines the prevalence of neurodevelopmental disorders like ADHD and autism among youth who have committed crimes. Researchers will analyze records from juvenile offenders to explore how these conditions relate to the types and frequency of criminal behavior. The goal…

This study will follow 60 children with SLC6A1-NDD, a rare genetic condition causing seizures, developmental delays, and autism. Researchers will track symptoms like seizure frequency and development over time to better understand the disease. The goal is to gather information th…

This international online study collects medical, behavioral, and developmental information from people with rare genetic changes that are linked to autism and other neurodevelopmental disorders. By partnering with families, researchers aim to build a detailed database to improve…

This study looks at whether adults with autism can reliably report their own personality disorder traits using a standard questionnaire. Researchers compare these self-reports with descriptions from someone who knows the participant well, such as a family member or partner. The g…

This study tests a mobile health program called FANS-EI that aims to support caregivers of young autistic children in staying engaged with early interventions. Researchers will measure caregiver comfort, participation, and sense of support. The study involves 28 caregiver-child p…

This study looks at whether a parent training program can help autistic toddlers (ages 12-36 months) learn to manage their emotions. Researchers will work with 40 families and early intervention providers to see if the program is practical and helpful. The goal is to improve supp…

This study aims to create and validate a Turkish version of a short neurological exam called the BRIEF-HINE for babies at high risk of brain problems. Researchers will test 120 infants aged 3 to 12 months who have conditions like premature birth or brain injury. The goal is to se…

This pilot study will develop and test a toolkit that helps therapists tailor mental health treatment for autistic individuals. Researchers will first hold focus groups to understand barriers, then create a resource for therapists. The toolkit will be tested with 18 autistic teen…

This study aims to better understand GEMIN5-related neurodevelopmental disorder, an ultra-rare genetic condition. Researchers will review medical records and follow participants over time to track developmental milestones, brain changes, vision, hearing, and survival. The goal is…

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 observational study follows 150 people with MEHMO syndrome or related conditions to better understand how the disease progresses. Researchers will collect medical history, imaging, and lab samples to find biological markers that could help monitor the disease. No treatment i…

This study is testing a digital platform called the Genetics Navigator to see if it can reduce distress for people undergoing genetic testing. The platform provides education, counseling, and management recommendations. Researchers will compare it to standard care in 170 adults a…

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 bladder and bowel problems in children aged 5 to 12 with intellectual disabilities and compares them to typically developing children. Researchers want to understand how these issues affect the child's daily activities and family quality of life. The goal is t…

This study looks at how to reduce the long and frustrating journey to a diagnosis for people with developmental abnormalities. Researchers will review past cases, collect new blood or skin samples, and use advanced genetic testing. The goal is to understand why some people remain…

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 is creating a central database and tissue bank for rare diseases affecting the kidneys and liver, such as ARPKD and Joubert syndrome. Researchers will collect medical information, genetic samples, and tissues from 200 participants to help doctors and scientists better …

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 looks at whether epidural pain relief given to mothers during childbirth has any effect on their child's brain development up to 24 months of age. Researchers will follow thousands of first-time mothers and their babies, comparing those who received epidural analgesia …

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 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 observational study aims to find biological markers (biomarkers) for RAI1-related disorders, including Smith-Magenis and Potocki-Lupski syndromes. Researchers will study 90 participants through clinical exams, blood tests, optional skin biopsies, and sleep studies. The goal …

This study follows 240 pregnant women and their babies to see if the mother's intake of certain nutrients (like choline, folate, and vitamin B12) during pregnancy is linked to the child's brain development up to age 2. Researchers will measure nutrients in the mother's blood and …

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 follows 180 infants who had COVID-19 around birth to see if the infection affects hormone levels and development. Researchers focus on a temporary hormone surge called minipuberty, which is important for growth. By measuring hormones and developmental milestones, they …

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 study tests whether analyzing RNA from blood and skin samples can find hidden genetic causes in people with rare diseases like intellectual disability. Researchers will compare results from both tissues to see which works best. The goal is to reduce the years of uncertainty …

This study looks at how technology, like LEGO robots, can help children with physical disabilities play. Researchers will ask parents and kids about what helps or hinders play, then let children play with robots or conventional toys. The goal is to create a complete model of play…

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 aims to find the specific gene that causes Aicardi syndrome, a rare brain disorder. Researchers will collect blood and skin samples from up to 500 patients and their parents to analyze their DNA. The goal is to identify genetic changes unique to people with Aicardi syn…

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 study looks at whether brain wave tests (EEG) done before and after heart surgery in babies under 1 year old can predict later learning or behavior problems, such as autism or ADHD. About 50 infants will be followed to age 2. The goal is to find early warning signs so that c…

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 develop and test a simple scale that caregivers can use to detect common eye disorders in adults with intellectual disability. Researchers will collect data from 1,400 participants, including socioeconomic information, daily living skills, and eye exam results.…

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 looks at whether a special version of multisystemic therapy (MST-ID) works better than standard therapy for teenagers (ages 10-19) with severe behavioral problems who come from families with intellectual disabilities. About 470 teens and their families will take part, …

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 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 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 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 will follow over 1,200 children aged 3 to 11 who were born with critical heart defects and had heart surgery in their first three months. Researchers want to find out how many of these children develop learning, attention, or behavioral problems, and why. They will use…

This study looks at how adults with intellectual developmental disorder (IDD) understand emotions, compared to people without IDD. Researchers will test 60 adults using tasks that involve recognizing facial expressions, emotions in context, and vocal tones. The goal is to find sp…

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 aims to uncover the genetic causes of hereditary spastic paraplegia (HSP), a group of rare neurological diseases that cause progressive muscle stiffness and weakness. Researchers at Boston Children's Hospital will analyze DNA from 200 people with HSP to find genetic va…

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 will follow 1,000 adults in Spain who are suspected of having obstructive sleep apnea (OSA) and daytime sleepiness. Participants will undergo standard sleep tests, blood pressure monitoring, and fill out questionnaires about their quality of life and sleepiness. Resear…

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 follows 80 youth aged 9 to 19 with bipolar disorder across four U.S. sites. Researchers will track mood symptoms, inflammation, and family conflict over 12 months to find what predicts better or worse outcomes. The goal is to create standardized tools and best-practice…

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 looks at psychiatric symptoms in people with White-Sutton syndrome, a rare genetic condition. Researchers will interview 30 children and adults and use standard questionnaires to identify anxiety, OCD, autism, and other issues. The goal is to better understand these sy…

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 follows 150 children and adults worldwide who have a rare genetic condition affecting CAMK2 genes. Researchers will track development, seizures, behavior, and social skills over time. The goal is to create a clear picture of how the disorder progresses, which can help …

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 looks at how children and adults with Developmental Coordination Disorder (DCD) sense touch and body position, and how they update their body image when using tools. Researchers will compare 280 participants with and without DCD to see if sensory deficits explain motor…

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 watches how babies move in their first months to see if it predicts their development later. Researchers will video-record preterm and full-term infants and score their movements. When the children turn 18 and 36 months old, parents will fill out a questionnaire about …

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 collects information from people with ASXL-related disorders (such as Bohring-Opitz syndrome) to better understand how these conditions progress and are managed. No new treatments are tested; instead, participants share their medical history and records through surveys…

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 is collecting blood, tissue, and medical information from up to 1,000 people with RASopathies—a group of genetic conditions that affect development and raise cancer risk. Researchers will store these samples and data in a database for future studies. The goal is to lea…

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 how hearing sensitivity (hyperacusis) might help language skills in children and adults with Williams syndrome. Researchers will use EEG and NIRS brain scans along with questionnaires to compare participants with Williams syndrome, Down syndrome, and typical d…

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 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 collects medical information and biological samples (like blood or tissue) from up to 12,000 people with leukodystrophies—rare disorders that damage the brain's white matter. Researchers will use this data to find new genetic causes, develop biomarkers for future trial…

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 creates a registry to collect health information from 200 people with rare forms of primordial dwarfism, such as MOPDII and Meier-Gorlin syndrome. Researchers hope to learn how these conditions change over a person's lifetime and improve future care. Participants provi…

This study explores whether an advanced practice nurse can effectively screen for neurodevelopmental disorders (like learning or behavior problems) in children aged 1 to 5 who have congenital heart disease. Researchers will compare the nurse's screening results with a full assess…

This study looks at whether bright light therapy can help reduce depressive symptoms in adults with intellectual disabilities. Researchers will see how acceptable and practical this treatment is in two care organizations. About 105 participants, including staff and patients, will…

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…

The Dragonfly study is an international observational project tracking the development of 250 children and adults with CTNNB1 neurodevelopmental syndrome. Researchers will collect medical history, perform neurological exams, and use questionnaires to understand how symptoms and a…

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 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…