Mode
Home
Research
Clinical Trials Drug Pipeline Weekly Digests
Reference
Conditions Medications
Community
Questions
Text Size
Log in / Sign up
Home › Conditions › Autism Spectrum Disorder

Autism Spectrum Disorder

64 published articles · Updated continuously

Clinical Trial Landscape

Clinical Trials for Autism Spectrum Disorder

191 trials tracked for Autism Spectrum Disorder: 28 in phase 3 or 4 and 8 with published results. The most-cited published study has 118 citations.

191Trials tracked
28Phase 3 & 4
0Recruiting
8With published results
Phase distribution
Phase 4 11 Phase 3 17 Phase 2 52 Phase 1 10 Other / NA 101
  1. Phase 3 Treatment of Overweight Induced by Antipsychotic Medication in Young People With Autism Spectrum Disorders (ASD) Completed · 118 cited
  2. Phase 4 Neural and Behavioral Effects of Oxytocin in Autism Spectrum Disorders Completed · 45 cited
  3. Phase 3 Behavioral and Neural Response to Memantine in Adolescents With Autism Spectrum Disorder Completed · 45 cited
  4. Phase 3 Effectiveness of Atomoxetine in Treating ADHD Symptoms in Children and Adolescents With Autism Completed · 45 cited
  5. Phase 3 Mirtazapine Treatment of Anxiety in Children and Adolescents With Pervasive Developmental Disorders Completed · 31 cited
  6. Phase 4 Methylphenidate ER Liquid Formulation in Adults With ASD and ADHD Completed · 12 cited
Show 44 more trials
  1. Phase 3 Pediatric Open-Label Extension Study Completed · 11 cited
  2. Phase 3 A Trial of CM-AT in Children With Autism With All Levels of FCT (The Blum Study) Completed · 7 cited
  3. Phase 4 Dose Response Effects of Quillivant XR in Children With ADHD and Autism: A Pilot Study Completed
  4. Phase 4 Milnacipran in Autism and the Functional Locus Coeruleus and Noradrenergic Model of Autism Completed
  5. Phase 4 Improving Driving in Young People With Autism Spectrum Disorders Completed
  6. Phase 4 Natural Treatments for the Management of Emotional Dysregulation in Youth With Non-verbal Learning Disability (NVLD) and/or Autism Spectrum Disorders (ASD) Completed
  7. Phase 4 Atomoxetine, Placebo and Parent Management Training in Autism Completed
  8. Phase 4 A Study of Memantine Hydrochloride (Namenda®) for Cognitive and Behavioral Impairment in Adults With Autism Spectrum Disorders Completed
  9. Phase 4 FMRI Brain Activation of Aripiprazole Treatment in Autism Spectrum Disorders Completed
  10. Phase 4 A Study of the Effectiveness and Safety of Two Doses of Risperidone in the Treatment of Children and Adolescents With Autistic Disorder Completed
  11. Phase 4 Minocycline to Treat Childhood Regressive Autism Completed
  12. Phase 3 Study of Paliperidone ER in Adolescents and Young Adults With Autism Completed
  13. Phase 3 Folinic Acid in Autism Completed
  14. Phase 3 A Study of Oxytocin for the Treatment of Social Impairment in Individuals With High Functioning Autism Spectrum Disorder Completed
  15. Phase 3 Treatment of Encopresis in Children With Autism Spectrum Disorders Completed
  16. Phase 3 Intranasal Vasopressin Treatment in Children With Autism Completed
  17. Phase 3 Methylphenidate for Attention Deficit Hyperactivity Disorder and Autism in Children Completed
  18. Phase 3 Cognitive-Behavioral Treatment for Anxiety Disorders in Children With Autism Spectrum Disorders Completed
  19. Phase 3 Pilot Study of the Effect of Hyperbaric Oxygen Treatment on Behavioral and Biomarker Measures in Children With Autism Completed
  20. Phase 3 Lurasidone Pediatric Autism Study Completed
  21. Phase 3 Pharmacogenomics in Autism Treatment Completed
  22. Phase 3 A Pilot Study of Daytrana TM in Children With Autism Co-Morbid for Attention Deficit Hyperactivity Disorder (ADHD) Symptoms Completed
  23. Phase 2 A Study of Oxytocin in Children and Adolescents With Autistic Disorder Completed
  24. Phase 2 Sulforaphane for the Treatment of Young Men With Autism Spectrum Disorder Completed
  25. Phase 2 Fatty Acid Supplementation in Children With ASD Completed
  26. Phase 2 Efficacy and Tolerability Study of Betahistine to Ameliorate Antipsychotic Associated Weight Gain Completed
  27. Phase 2 A Trial of Sertraline in Young Children With Autism Spectrum Disorder Completed
  28. Phase 2 Divalproex Sodium vs. Placebo in Childhood/Adolescent Autism Completed
  29. Phase 2 Nuedexta for the Treatment of Adults With Autism Completed
  30. Phase 2 Intranasal Oxytocin Treatment for Social Deficits in Children With Autism Completed
  31. Phase 2 Acamprosate in Youth With Fragile X Syndrome Completed
  32. Phase 2 Rapalogues for Autism Phenotype in TSC: A Feasibility Study Completed
  33. Phase 2 Safety of L1-79 in Adolescent and Adult Males With Autism Completed
  34. Phase 2 Intranasal Oxytocin and Learning in Autism Completed
  35. Phase 2 Allogenic Cord Blood Transfusion in Patients With Autism Completed
  36. Phase 2 Open-label Study of Safety and Tolerability of Memantine in Children With Autism Completed
  37. Phase 2 Study of Oxytocin in Autism to Improve Reciprocal Social Behaviors Completed
  38. Phase 2 University of California, San Diego (UCSD) Suramin Autism Treatment-1 (SAT1) Trial Completed
  39. Phase 2 Sulforaphane Treatment of Children With Autism Spectrum Disorder (ASD) Completed
  40. Phase 2 ScanBrit Dietary Intervention in Autism Completed
  41. Phase 2 Omega-3 Fatty Acids for Hyperactivity Treatment in Autism Spectrum Disorder Completed
  42. Phase 2 Riluzole to Treat Child and Adolescent Obsessive-Compulsive Disorder With or Without Autism Spectrum Disorders Completed
  43. Phase 2 A Feasibility Study of N-acetylcysteine for Self-injurious Behavior in Children With Autism Spectrum Disorder Completed
  44. Phase 2 The Effect of Donepezil (Aricept(Registered Trademark)) on REM Sleep in Children With Autism Completed

Showing the 50 most-cited and recently-updated of 191 trials. Browse the full registry →

Trial data sourced from ClinicalTrials.gov. Counts describe the research landscape and are not a treatment recommendation. Informational only — not medical advice.

What the trials found Updated — new results For clinicians

Autism Spectrum Disorder: what the trials found

Several established trials have investigated pharmacological interventions for autism spectrum disorder (ASD) and associated symptoms. In a phase 4 trial, atomoxetine showed no significant difference in ADHD response rates (45.2% vs 46.9% vs 29.0% vs 19.4%, p=0.47) or ASD response rates (22.6% vs 43.8% vs 38.7% vs 16.4%, p=0.95) compared to placebo 13. Another phase 3 trial of atomoxetine reported ADHD Rating Scale scores of 28.7 vs 34.0 and Vineland Adaptive Behavior Scales composite scores of 73.4 vs 74.9 20. A phase 4 trial of methylphenidate extended-release liquid formulation showed a mean change of -22.8 in Adult ADHD Investigator Symptom Report Scale score 8. For irritability, a phase 4 trial of aripiprazole was conducted 16, and a placebo-controlled trial found significant changes in Aberrant Behavior Checklist Irritability subscale (-3.5, -7.4, -12.4, p<0.001) and Clinical Global Impression Severity (-0.3, -0.4, -1.0, p<0.001) 17. A phase 3 trial of lurasidone (20-80 mg) reported changes from baseline in PANSS total score (76.0 to -11.9, -15.6, -18.4) and subscales 22. Other established trials include buspirone 10, milnacipran 11, very low dose Quillivant XR 12, minocycline 14, memantine 15 (with 15 participants showing reduction in ASD symptom severity 15), metformin 18, and memantine in another phase 3 trial 19. A phase 4 trial of open-label omega-3 fatty acids + inositol showed a mean change in Parent-Young Mania Rating Scale score of 0.0 to -2.6 9. A phase 4 trial of Syntocinon (oxytocin) reported changes in brain activity via fMRI at 4, 8, and 52 weeks 7. A placebo-controlled trial found a mean 10-week change in Pediatric Anxiety Rating Scale of -4.9 vs -3.2 and response rates of 0.47 vs 0.20 21.

Recent results — preliminary, needs further review

  • A recent trial (N=296) of Flore showed no significant improvement in Social Responsiveness Scale (80.2 vs 78.6, p>0.05) but a significant improvement in Gastrointestinal Symptom Rating Scale (2.25 vs 1.96, p<0.05) 1.
  • A recent trial (N=24) of Sentido's Model showed significant improvements in Vineland-3 Adaptive Behavior Composite Score (74.8 to 90.8, p<0.001), Goal Attainment Scaling scores (0 to 20, p<0.001), and Family Outcome Survey total score (68 to 91, p<0.001) 2.
  • A recent trial (N=8) of The Listening Program with bone conduction headphones reported changes in ABAS-3 score from 71.00 to 73.83 3.
  • A recent trial (N=27) evaluated Families FORWARD (Focusing on Relationships, Well-being, and Responsibility aheaD) 4.
  • A recent trial (N=41) evaluated transcranial photobiomodulation (tPBM) 5.

For the clinician treating this condition

  • For irritability in ASD, aripiprazole and placebo-controlled data show significant improvements in Aberrant Behavior Checklist Irritability and Clinical Global Impression Severity 1617.
  • Atomoxetine did not demonstrate significant benefit over placebo for ADHD or ASD response rates in a phase 4 trial 13, though a phase 3 trial reported numerical differences in ADHD rating scores 20.
  • Lurasidone (20-80 mg) showed dose-dependent reductions in PANSS total and subscale scores in a phase 3 trial 22.
  • Recent evidence suggests potential benefits of Sentido's Model for adaptive behavior and goal attainment 2, and Flore for gastrointestinal symptoms 1, but these findings are not yet corroborated.

AI synthesis of 21 cited trials, updated Jun 23, 2026. Informational only — not medical advice; trial data sourced from ClinicalTrials.gov. How we use AI.

HCP Mode — summaries include clinical detail, trial data, and statistical outcomes.
Patient Mode — summaries use plain language, avoiding clinical jargon.
Sensory severity predicts insula decoupling in autistic participants during motion stimulation
Radiology & Imaging Cohort
Sensory severity predicts insula decoupling in autistic participants during motion stimulation Sensory severity in autism forms a continuous pattern, not distinct categories
This cohort study examined 223 behavioral participants and 63 neurobiological participants with autism spectrum disorder.
New data show sensory processing differences in autism are a continuous spectrum rather than separate types, changing how we understand thes…
medRxiv Apr 30, 2026
Neuroimaging features predict ASD identification and cognitive decoding accuracy in large cohorts
Psychiatry Cohort
Neuroimaging features predict ASD identification and cognitive decoding accuracy in large cohorts New AI Model Finds Brain Pattern That Predicts Autism Symptoms
This cohort study analyzed individuals from three independent large-scale neuroimaging cohorts (HCP-task, ABIDE-I, ABIDE-II).
A new tool finds a clear brain signal that predicts autism symptoms with high accuracy.
medRxiv Apr 30, 2026
Observational study finds Touch Activated Phlebotomy reduces pain in autistic and non-autistic adults compared to traditional venipuncture
Nutrition & Obesity Medicine
Observational study finds Touch Activated Phlebotomy reduces pain in autistic and non-autistic adults compared to traditional venipuncture Touch Activated Phlebotomy Causes Less Pain Than Traditional Blood Draws
This primary research article reports on an observational study of 23 participants, including 11 autistic adults and 12 non-autistic adults.
A small study found a new blood draw device caused less pain than standard methods for both autistic and non-autistic adults.
medRxiv Apr 30, 2026
Review of human postmortem studies finds region-dependent microglial variation in autism spectrum disorder brains
Neurology Cohort
Review of human postmortem studies finds region-dependent microglial variation in autism spectrum disorder brains Brain scans show microglia vary by region and stage in autism
This review analyzed human postmortem studies using bulk transcriptomics and single-cell atlases to examine microglia in autism spectrum dis…
A review of brain tissue suggests immune cells in autism differ by location and development rather than showing one single pattern across al…
Frontiers Apr 29, 2026
Qualitative review highlights barriers to recognizing autism in women with borderline personality disorder diagnoses
Psychiatry
Qualitative review highlights barriers to recognizing autism in women with borderline personality disorder diagnoses Many women with BPD may actually be autistic
This qualitative review synthesizes interviews with 15 clinicians and 15 women or people assigned female at birth regarding autism recogniti…
Hundreds of women told doctors they were misdiagnosed with borderline personality disorder when they may actually be autistic, raising urgen…
medRxiv Apr 29, 2026
Review of language impairment definitions in 75 speaking autistic adolescents and young adults
Psychiatry
Review of language impairment definitions in 75 speaking autistic adolescents and young adults Stricter language definitions change how many autistic teens are classified
This observational study review examined how different epidemiological definitions of language impairment (LI) affect classification in 75 s…
Stricter language definitions increase how many autistic adolescents are classified as having impairment, but results vary by measure.
medRxiv Apr 28, 2026
ADHD and ASD polygenic risk scores correlate with antipsychotic responsiveness in schizophrenia postmortem brains
Psychiatry Cohort
ADHD and ASD polygenic risk scores correlate with antipsychotic responsiveness in schizophrenia postmortem brains Genes for ADHD and Autism May Predict Schizophrenia Drug Response
This postmortem brain study in 24 patients with schizophrenia and 48 controls examined polygenic risk scores for ADHD and ASD.
Genes tied to ADHD and autism may predict how well antipsychotic drugs work in schizophrenia patients.
Frontiers Apr 27, 2026
Children with autism lack auditory looming bias seen in typical development
Neurology
Children with autism lack auditory looming bias seen in typical development Brain scans show autistic kids hear approaching sounds differently
This primary trial compared EEG responses to looming vs receding sounds in 3-4-year-old children with autism, sensory concerns, or typical d…
Brain scans reveal preschoolers with autism process approaching sounds differently than typical children do, pointing to unique sensory wiri…
medRxiv Apr 26, 2026
Genetic study of autism in African Americans shows limited transferability of European-derived polygenic scores
Genetics & Precision Medicine Cohort
Genetic study of autism in African Americans shows limited transferability of European-derived polygenic scores Autism genes differ by ancestry—new clues in Black families
This cohort study of 38,483 autistic individuals compared genetic findings between African and European ancestries.
Black children with autism carry different genetic clues than white children, a landmark study shows—meaning better answers and fairer care …
medRxiv Apr 25, 2026
Review of vagus nerve stimulation for autism spectrum disorder and maternal immune activation
Neurology Sys. Review
Review of vagus nerve stimulation for autism spectrum disorder and maternal immune activation Vagus nerve stimulation shows promise for autism and maternal immune activation
This narrative review examines vagus nerve stimulation (VNS) in the context of autism spectrum disorder and maternal immune activation.
A review suggests vagus nerve stimulation could help people with autism spectrum disorder or those affected by maternal immune activation.
Frontiers Apr 24, 2026
Review of serotonergic signaling in autism spectrum disorder and other neurodevelopmental conditions
Drug Pipeline Sys. Review
Review of serotonergic signaling in autism spectrum disorder and other neurodevelopmental conditions Serotonin’s Hidden Role in Brain Development Depends on Sex
This narrative review examines the role of serotonergic signaling in autism spectrum disorder and other psychiatric and neurodevelopmental c…
This discovery may help explain why conditions like autism affect boys and girls so differently.
Frontiers Apr 24, 2026
Bumetanide shows individual-level improvement in heterogeneous neurodevelopmental cohort
Psychiatry RCT
Bumetanide shows individual-level improvement in heterogeneous neurodevelopmental cohort Bumetanide helps mood and sleep in kids with sensory issues
In a post-trial access study using a multiple-baseline single-case experimental design, 113 youth (7–19 years) with ASD, ADHD, epilepsy, or …
Kids with autism, ADHD, or epilepsy who feel sensory overload saw better mood and sleep after taking bumetanide.
medRxiv Apr 24, 2026

Questions about Autism Spectrum Disorder

Do specific autism neurosubtypes show opposite electrical brain activity patterns in preclinical models?

Research using animal models and human EEG data indicates that autism consists of distinct neurosubtypes with opposing electrical brain activity patterns related to excitation and inhibition balance.

Full answer →
Does having different gut bacteria affect children with Autism Spectrum Disorder and ADHD?

Yes, children with ASD and ADHD often have different gut bacteria compared to neurotypical children, and this imbalance may influence symptoms and gut health.

Full answer →