Ongoing Clinical Trials for Autism Spectrum Disorder
Currently, there are 11 clinical trials investigating various treatments for autism spectrum disorder across Europe and other regions. These studies explore different approaches including hormone-based nasal sprays, medications targeting social behaviors, gut microbiome interventions, and medication safety monitoring in children and adolescents with autism.
Clinical trial locations
- Belgium
- France
- Study on Arbaclofen for Improving Social Function in Children and Adolescents with Autism Spectrum Disorder
- Study of Oxytocin Nasal Spray as Add-on Treatment for Children with Autism Spectrum Disorder and Intellectual Disability: Safety and Feasibility Assessment
- Study on the Effects and Safety of Fecal Microbiota Transplant in Children with Autism and Digestive Issues
- Study on the Effects of Pitolisant for Children and Adolescents with Autism Spectrum Disorder
- Study on the Long-Term Safety of Pimavanserin for Treating Irritability in Children and Adolescents with Autism Spectrum Disorder
- Germany
- Hungary
- Italy
- Netherlands
- Norway
- Poland
- Spain
- Study on Arbaclofen for Improving Social Function in Children and Adolescents with Autism Spectrum Disorder
- Study on the Effects of Pitolisant for Children and Adolescents with Autism Spectrum Disorder
- Study on the Long-Term Safety of Pimavanserin for Treating Irritability in Children and Adolescents with Autism Spectrum Disorder
Study of Oxytocin Nasal Spray as Add-on Treatment for Children with Autism Spectrum Disorder and Intellectual Disability: Safety and Feasibility Assessment
This study is designed for children aged 6 to 12 years who have both autism and moderate to severe intellectual disability. The research examines oxytocin nasal spray as an additional treatment alongside standard behavioral and educational therapy.
Who can participate: Children must have a confirmed diagnosis of severe autism based on standardized criteria and moderate to severe intellectual disability with challenging behaviors. They must be receiving ongoing care at one of the study centers and be covered by social security. If the child is taking any psychiatric medications, these must have been at stable doses for at least three months. Parents need to be able to follow medication instructions and complete questionnaires, and both parents must sign informed consent.
Who cannot participate: Children outside the 6 to 12 year age range, those without confirmed diagnoses of both autism and intellectual disability, or those with known allergies to the study medication are excluded. Children who participated in another clinical trial within the past 30 days, have serious medical conditions including heart problems, or cannot attend regular medical check-ups are not eligible. Children with significant nasal problems preventing proper use of the spray or those unable to provide blood samples for monitoring also cannot participate.
Study focus: The main goal is to determine whether it is practical and safe to use oxytocin nasal spray in this specific group of children. The study will test two different doses (4 and 8 international units) to evaluate how well children tolerate the treatment. Healthcare providers and parents will monitor children’s responses and any changes in behavior throughout the 12-week treatment period.
Treatment approach: The investigational treatment is oxytocin, a naturally occurring hormone administered as a nasal spray. It is given daily both at treatment centers and at home, alongside standard psycho-educational therapy that focuses on teaching social, behavioral, and communication skills.
Study on Improving Social Interaction in Children with Autism Using Oxytocin Nasal Spray and Therapy
This Belgian trial explores how a single dose of oxytocin nasal spray might enhance social interactions in children with autism. The study combines the hormone treatment with activities designed to measure social engagement.
Who can participate: Male children aged 8 to 12 years and pre-pubertal females within this age range can join if they have a formal diagnosis confirmed by experienced clinicians. They must have verbal comprehension and visual spatial abilities above a certain threshold, and any medications or therapies must remain unchanged for four weeks before the study visit.
Who cannot participate: Children with other major medical or psychiatric conditions, recent participation in other trials, substance abuse history, severe allergies to medications, uncorrected vision or hearing impairments, poorly controlled neurological disorders, genetic disorders affecting brain function, severe behavioral problems, infectious diseases, or conditions requiring medications that could interfere with the study are excluded.
Study focus: The primary objective is to observe changes in social attunement between the child and researcher during both screen-based and real-life social activities. The study also measures natural hormone levels in saliva samples after treatment to understand biological effects.
Treatment approach: Oxytocin is administered once as a nasal spray called Syntocinon. The treatment is combined with interpersonal sensorimotor synchronization therapy, which involves activities designed to improve coordination and timing between the child and therapist.
Study on Oxytocin Treatment for Children with Autism and Intellectual Disability
This Belgian study evaluates multiple doses of oxytocin nasal spray over time, focusing on improving core autism symptoms and reducing stress in children with both autism and intellectual disabilities.
Who can participate: Children between 4 and 13 years old with confirmed diagnoses of both autism and intellectual disability can participate. Only pre-menstrual girls are included, though those who begin menstruation during the trial may continue. Children must attend a special needs school or meet specific criteria indicating intellectual disability.
Who cannot participate: Children without confirmed diagnoses of both autism and intellectual disability, those outside the age range, or those unable to undergo required assessments or follow study procedures are excluded.
Study focus: The study aims to assess effectiveness on core autism symptoms using standardized tools that measure social communication changes and stress levels through heart rate monitoring. Additional assessments examine repetitive behaviors, adaptive behavior, overall behavior, stress levels, and sleep habits.
Treatment approach: The treatment uses Syntocinon nasal spray containing oxytocin, administered in multiple doses according to the study protocol. The focus is on evaluating both behavioral improvements and physiological stress responses.
Study on Arbaclofen for Improving Social Function in Children and Adolescents with Autism Spectrum Disorder
This multi-country trial spanning Spain and France examines whether a single dose response to arbaclofen can predict long-term benefits in social functioning for young people with autism.
Who can participate: Participants aged 7 to 23 years with confirmed autism diagnoses can join. They must have a reliable caregiver who can bring them to visits and provide regular feedback. Current medications affecting behavior must be stable for at least six weeks, and therapies must be stable for three months. Those with seizure histories must be seizure-free for specified periods.
Who cannot participate: The specific exclusion criteria focus on ensuring participants can safely complete the study procedures and assessments, though detailed exclusions were not fully specified in the source material.
Study focus: The study evaluates whether a single dose response during an initial controlled phase can predict how individuals will respond to longer-term treatment. Researchers measure specific brain activity markers and behavioral changes to assess improvements in social functions.
Treatment approach: Arbaclofen is given as an oral solution in various doses ranging from 5mg to 20mg. The study includes both active treatment and placebo groups for comparison, lasting up to 13 weeks.
Study on Improving Social Interaction in Children with Autism Using Oxytocin Nasal Spray and Therapy
This second Belgian study with similar design explores oxytocin’s effects on social bonding and interaction in children with autism through a single-dose approach.
Who can participate: Males aged 8 to 12 and pre-pubertal females within this range with formal autism diagnoses can participate. They must have intelligence quotients above 70 and maintain stable background treatments for four weeks before the study. No changes in psychosocial interventions are allowed during the study period.
Who cannot participate: Children with other major medical or psychiatric conditions, severe allergies, current participation in other trials, substance abuse history in the past year, uncorrected sensory impairments, poorly controlled neurological disorders, genetic disorders affecting brain function, severe behavioral problems, infectious diseases, or conditions requiring interfering medications are excluded.
Study focus: The study observes changes in dyadic attunement between participants and experimenters using neurophysiological and behavioral measures. Saliva samples help assess changes in natural hormone levels following treatment.
Treatment approach: A single dose of intranasal oxytocin is administered using Syntocinon nasal spray, with the goal of enhancing social attunement and connection abilities.
Study on the Effects and Safety of Fecal Microbiota Transplant in Children with Autism and Digestive Issues
This French trial takes a unique approach by examining gut health interventions for children with autism who also experience digestive problems.
Who can participate: Children aged 36 to 72 months (3 to 6 years) with autism diagnoses and gastrointestinal symptoms lasting over a year can participate. They must have symptoms like diarrhea, abdominal pain, constipation, indigestion, or reflux reaching certain severity levels. All legal guardians must consent, and children must have social insurance coverage.
Who cannot participate: Children outside the specified age range, without autism diagnoses, with other interfering medical conditions, previous fecal transplantation, severe allergies, compromised immune systems, certain gastrointestinal diseases, or those taking interfering medications are excluded.
Study focus: The primary goal is achieving a significant reduction in gastrointestinal symptom scores by week 18. Secondary outcomes include improvements in core autism symptoms, quality of life, and careful monitoring for any side effects from the procedure.
Treatment approach: Fecal microbiota transplantation involves introducing healthy bacteria from a donor through an enema over a maximum period of 14 days. Progress is monitored at multiple time points extending to one year after treatment.
Study on the Effects of Intranasal Oxytocin on Learning in Youth with Autism Spectrum Disorder
This Norwegian study investigates how oxytocin affects brain function and learning abilities in young people with autism.
Who can participate: Participants aged 12 to 21 years with confirmed autism diagnoses who can communicate with the study team are eligible. Both males and females can participate.
Who cannot participate: Individuals without autism diagnoses or outside the specified age range cannot participate. The study excludes vulnerable populations requiring extra research protection.
Study focus: The study examines neural plasticity (the brain’s ability to change and adapt) using brain activity measurements taken 40 minutes after treatment. Cognitive flexibility is assessed through computerized tasks, and heart rate variability provides additional information about bodily responses.
Treatment approach: Single doses of oxytocin nasal spray are administered at different strengths (8 or 24 international units) over three separate treatment periods with washout phases between them to prevent carryover effects.
Study on the Effects of Intranasal Oxytocin on Social and Repetitive Behaviors in Youth with Autism Spectrum Disorder
This second Norwegian trial focuses specifically on how oxytocin influences social and repetitive behaviors over a four-week treatment period.
Who can participate: Males and females aged 12 to 20 years with confirmed autism diagnoses can participate. They must be in good general health and able to communicate effectively with investigators. Participants aged 16 and older provide their own consent, while younger participants provide assent with caregiver consent.
Who cannot participate: Individuals without autism diagnoses or outside the specified age range are excluded.
Study focus: The study monitors changes in social responsiveness and repetitive behaviors using standardized scales completed by caregivers. Behavioral inflexibility is also assessed throughout the four-week period.
Treatment approach: Oxytocin nasal spray at 24 international units is administered twice daily for four weeks. The study uses a double-blind design where neither participants nor researchers know who receives active treatment versus placebo.
Studying the Safety of Aripiprazole and Risperidone in Children and Adolescents with Autism Spectrum Disorder
This study across Germany and the Netherlands examines whether monitoring medication levels in the blood can help reduce weight gain and other side effects from commonly prescribed autism medications.
Who can participate: Children and adolescents aged 6 to 18 years with documented autism diagnoses and behavioral problems who are starting treatment with either risperidone or aripiprazole can participate.
Who cannot participate: Specific exclusion criteria were not detailed in the source material, but generally focus on conditions that might interfere with safe study participation.
Study focus: The primary focus is monitoring changes in body mass index over 6 and 12 months. Secondary assessments include behavior checklists, quality of life measures, clinical improvement evaluations, and various health parameters like glucose, cholesterol, hormones, and blood pressure.
Treatment approach: Participants receive either risperidone or aripiprazole orally, with one group having doses adjusted based on therapeutic drug monitoring (regular blood level checks) and another group receiving standard care without this additional monitoring.
Study on the Effects of Pitolisant for Children and Adolescents with Autism Spectrum Disorder
This multi-country trial spanning Poland, Italy, Spain, and France explores pitolisant’s potential to improve social communication in children and adolescents with autism.
Who can participate: Children and adolescents aged 6 to 17 years with confirmed autism diagnoses and specific social communication difficulties can participate. They must have reliable caregivers providing regular support and interaction. Intelligence quotients must be 70 or higher, and current treatments must remain stable for at least four weeks before and during the study.
Who cannot participate: Individuals outside the specified age range or who are part of vulnerable populations requiring special protection cannot participate.
Study focus: The study evaluates changes in social communication and interaction skills using standardized scoring systems over a 12-week period. Additional assessments examine behavior, sleep patterns, and overall well-being.
Treatment approach: Wakix tablets containing pitolisant in strengths of 4.5mg or 18mg are taken orally. The study uses a double-blind design comparing active treatment to placebo, with participants monitored closely for effects and side effects.
Study on the Long-Term Safety of Pimavanserin for Treating Irritability in Children and Adolescents with Autism Spectrum Disorder
This extensive trial across Poland, Hungary, France, Italy, and Spain examines long-term safety of pimavanserin for managing irritability associated with autism.
Who can participate: Children and adolescents who completed a previous pimavanserin study can continue into this extension trial. They must be medically and behaviorally stable, with consent from parents or legal guardians and assent from participants when possible. Female participants of childbearing potential must use appropriate contraception and have negative pregnancy tests.
Who cannot participate: Individuals without autism-related irritability, those outside the study age range, people with medical conditions interfering with the study, those on medications affecting results, individuals with severe allergies to similar medications, those unable to follow procedures, recent participants in other trials, and those with conditions making participation unsafe are excluded.
Study focus: This 52-week study monitors long-term safety through vital signs, weight, body mass index, electrocardiograms, physical examinations, laboratory tests, and assessments for adverse effects. Effectiveness is measured through changes in irritability levels and behavioral improvements.
Treatment approach: Pimavanserin capsules in doses of 10mg, 20mg, or 34mg are taken orally throughout the 52-week period, with regular safety monitoring and behavioral assessments.
Summary
The current landscape of clinical trials for autism spectrum disorder reveals several notable patterns. A significant number of studies focus on oxytocin nasal spray treatments, with multiple trials examining this hormone-based approach across Belgium, France, and Norway. These studies explore both single-dose and longer-term treatment approaches, indicating strong research interest in oxytocin’s potential to enhance social functioning.
Geographically, France participates in the greatest number of trials, hosting studies on oxytocin, arbaclofen, fecal microbiota transplantation, pitolisant, and pimavanserin. Belgium shows concentrated interest in oxytocin research with three dedicated trials. Several studies span multiple countries, particularly those examining newer medications like pitolisant and pimavanserin, suggesting coordinated European research efforts.
The trials address diverse aspects of autism, from core social communication difficulties to associated challenges like irritability, digestive issues, and medication side effects. Age ranges vary considerably, with some studies focusing on young children (as young as 3 years) while others include adolescents and young adults up to 23 years. Many trials specifically include children with intellectual disabilities alongside autism, representing an important but often underserved population in research.
Treatment approaches span several categories: hormone-based therapies (oxytocin), psychiatric medications (aripiprazole, risperidone, pimavanserin), novel pharmaceuticals (pitolisant, arbaclofen), and innovative approaches like fecal microbiota transplantation. The emphasis on long-term safety monitoring in several trials reflects growing attention to ensuring treatments are well-tolerated over extended periods.
