A pilot study found that long-term intranasal oxytocin was safe and well-tolerated in eight adolescent boys with autism, with six showing improved social communication scores, though overall results were mixed.
6 of 8 boys improved on social communication measuresIn the first long-term safety study of nasal oxytocin in adolescents with autism, most participants showed improved ADOS-G scores with zero side effects over the treatment period.
What the researchers found
Six of eight participants showed improved scores on the communication and social interaction domains of the Autism Diagnostic Observation Schedule (ADOS-G). Caregivers of five participants reported positive effects, particularly in the quality of reciprocal communication.
However, standardized behavioral measures (Child Behavior Checklist T-scores and Aberrant Behavior Checklist scores) did not reach statistical significance, though some subcategories showed mild improvement trends. Critically, all eight participants showed excellent compliance and zero side effects across the entire treatment period, with normal blood pressure, blood, and urine results throughout.
Why it matters
Most previous oxytocin-autism research used single doses in adults, leaving major questions about whether the treatment is safe for children over extended periods. This study provided the first evidence that long-term nasal oxytocin is safe in adolescents with autism — a critical prerequisite for designing the larger, longer clinical trials needed to determine whether oxytocin can meaningfully help young people with social communication difficulties.
How the study worked
This was a single-armed, open-label pilot study in eight male adolescents with ASD (ages 10-14, IQ range 20-101). Oxytocin was administered intranasally with stepwise dose increases every 2 months (8, 16, then 24 IU per dose). A 1-2 week placebo period was inserted before each dose step. Outcomes were measured using the ADOS-G, Child Behavior Checklist (CBCL), and Aberrant Behavior Checklist (ABC). Safety was monitored via blood pressure, blood tests, and urinalysis.
What this study cannot tell us
This was a very small (n=8), open-label study with no control group, making it impossible to separate oxytocin's effects from placebo responses or natural development. The wide IQ range (20-101) adds heterogeneity. Behavioral rating scales did not reach statistical significance. The authors themselves acknowledge the results are too preliminary for definitive conclusions about efficacy.
How to read the evidence
This is a small, open-label pilot study with no control group — the lowest tier of clinical evidence. While it provides important safety data, the lack of blinding and small sample size mean the efficacy signals should be interpreted with extreme caution. It primarily serves as justification for larger controlled trials.
When this study was published
Published in 2013, this was an early and pioneering study in pediatric oxytocin-autism research. Since then, several larger trials have been conducted in both children and adults, with mixed results on efficacy but generally consistent safety findings.
The bigger picture
This pilot study addressed a critical gap in oxytocin-autism research by demonstrating safety in a pediatric population over an extended treatment period. It represents an early step in the journey toward determining whether this natural neuropeptide could become a standard treatment for social difficulties in autism — one of the most sought-after therapeutic goals in developmental neuroscience.
Questions still open
- Would a larger, double-blind, placebo-controlled trial confirm the social communication improvements seen in this pilot study?
- Is there an optimal dose of intranasal oxytocin for adolescents with autism, and does the stepwise dosing approach used here offer advantages?
- Do certain subgroups of children with autism (e.g., those with specific oxytocin system biomarkers) respond better to oxytocin treatment?
Common questions
Is intranasal oxytocin approved for treating autism?
Were there any side effects from long-term oxytocin use in these children?
Read the original research
Long-term administration of intranasal oxytocin is a safe and promising therapy for early adolescent boys with autism spectrum disorders.
Journal of child and adolescent psychopharmacology, 23(2), 123-7
Citation
Tachibana, Masaya; Kagitani-Shimono, Kuriko; Mohri, Ikuko; Yamamoto, Tomoka; Sanefuji, Wakako; Nakamura, Ayumi; Oishi, Masako; Kimura, Tadashi; Onaka, Tatsushi; Ozono, Keiichi; Taniike, Masako. (2013). Long-term administration of intranasal oxytocin is a safe and promising therapy for early adolescent boys with autism spectrum disorders.. Journal of child and adolescent psychopharmacology, 23(2), 123-7. https://doi.org/10.1089/cap.2012.0048