Intranasal Oxytocin for the Treatment of Children and Adolescents With Autism Spectrum Disorders (ASD): Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Intranasal Oxytocin for the Treatment of Children and Adolescents With Autism Spectrum Disorders (ASD) (NCT01908205), a randomized phase 2 study. The primary outcome is Efficacy of Intranasal Oxytocin vs. Placebo on Measures of Social Function.

2. Clinical Question

3. Trial Design

The registry identifies this as a phase 2, randomized, parallel-group trial comparing intranasal oxytocin with placebo in children and adolescents with autism spectrum disorder. Its status is completed. The recorded enrollment is 60. The safety records list 30 participants at risk in each arm; those arm-specific safety figures should not be treated as a substitute for a full account of enrollment, analysis populations, or participant flow.

The trial is described as quadruple-masked. The registry record does not specify which roles were masked or explain how masking was maintained. It also does not report the allocation procedure beyond identifying the study as randomized. The sponsor is Holland Bloorview Kids Rehabilitation Hospital. The recorded study start is 2013-09, and primary completion is recorded as 2015-09.

The registry describes the proposed dosing schedule for both groups as 0.4 IU/kg twice daily, with a maximum of 24 IUs per dose. The word “proposed” matters: the record does not give adherence, dose adjustments, treatment exposure, or deviations from the intended schedule. It also does not report additional details about the treatment period or follow-up beyond the primary outcome time points of 12 and 24 weeks.

Randomization and masking are features of the design, not results. They can support a fair comparison when implemented as intended, but the information in the registry record does not establish how successfully they were implemented. No detailed protocol, participant flow, baseline characteristics, or analysis-population description is reported in the registry record.

4. Participant Flow and Accounting

The registry reports how participants moved through each stage of the study. All counts below are exactly as posted; missing counts are not inferred from other stages.

Overall Study

Reasons for not completing this period:

5. Baseline Characteristics

The table reproduces the baseline characteristics posted in the registry, which describe the participants analysed at baseline and help show whether the randomized groups were comparable.

6. Primary Endpoint

The registry lists the primary outcome as Efficacy of Intranasal Oxytocin vs. Placebo on Measures of Social Function, measured over 12 and 24 weeks.

The primary endpoint is the change in score on the Aberrant Behavior Checklist (ABC) Social Withdrawal Subscale. The registry describes the scale as ranging from 0 to 48 and states that lower scores indicate improvement. The outcome is expressed in units on a scale, and the planned summary is a mean with a 95% confidence interval. Measurements are listed at 12 and 24 weeks.

A change score captures how a participant’s recorded score differs from an earlier assessment. Comparing changes between randomized groups can describe whether the groups followed different average trajectories over the assessment period. The registry record does not specify the exact reference assessment used to define change, how missing assessments would be handled, or whether the comparison would be adjusted for baseline score.

The ABC Social Withdrawal Subscale is the chosen measure of social function for this trial. A scale gives researchers a structured way to record a defined aspect of behavior, rather than relying on an unstructured judgment. The scale’s stated direction is essential for interpretation: a lower score is the direction designated as improvement. However, the record does not state what size of change would be considered important to participants or families, or give a threshold for a clinically meaningful difference.

No primary-outcome group means, changes, between-group contrast, or confidence interval are reported in the registry record. The planned measure and its interpretation can therefore be described, but the size and precision of any observed efficacy result cannot be assessed from this record.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The registry describes a randomized, quadruple-masked, parallel-group phase 2 study. These design labels convey how the comparison was intended to be structured, but they do not replace a report of the actual analysis. The registry record does not describe the randomization method, the roles covered by masking, or the statistical procedures used to analyze the primary endpoint.

What does the mean describe?

The primary measure is a continuous score on the ABC Social Withdrawal Subscale, and the specified summary parameter is the mean. A mean describes the average score or average change in a group. A comparison of group means can describe the average separation between treatment groups, but interpretation depends on exactly which quantity is compared and when it is measured. The registry record identifies change in score and assessments at 12 and 24 weeks, but does not report the group summaries or define the precise analysis contrast.

Because lower scores indicate improvement, a reduction in score is the favorable direction according to the registry’s scale description. For a between-group result, readers would also need to know the contrast’s sign convention. The record does not state how a reported difference would be ordered or whether a baseline-adjusted estimate was planned. Without the estimate and its definition, the endpoint description cannot establish an observed treatment-group difference.

How should a confidence interval be read?

In general, a confidence interval communicates statistical uncertainty around an estimated quantity. It gives a range of values compatible with the data and the method used, rather than a guarantee that the true effect lies within a particular realized interval. Its width is informative about precision, while its position relative to values of interest helps readers judge the range of effects consistent with the analysis. A confidence interval is not a measure of clinical importance by itself. The registry names a 95% confidence interval as the dispersion measure for the primary outcome but does not report its limits.

To assess the practical meaning of a score difference, readers would ideally have a justified threshold for an important change, along with the estimated contrast and its uncertainty. Such a threshold is not reported in the registry record. Statistical uncertainty and clinical importance are related questions, but they are not interchangeable: an estimate can be precise without being important, or uncertain while still leaving a range of potentially important values.

What does randomization contribute?

In general, random assignment aims to make treatment groups comparable in expectation, so differences in outcomes can be interpreted as comparisons of assigned groups rather than as comparisons formed by participant choice. Masking can reduce the influence of expectations on care, assessment, or reporting when the relevant people are unaware of assignment. These are methodological purposes, not guarantees. The registry record does not report baseline balance, adherence, masking success, deviations from assignment, or the procedures used to address these issues.

The parallel-group label means the registry identifies separate oxytocin and placebo arms. It does not imply that all participants completed follow-up or were included in an outcome analysis. The record does not report a participant-flow account or define whether the analysis was based on all randomized participants, participants who received treatment, or another population.

What do the planning assumptions mean?

The registry describes sample-size illustrations based on a two-sided significance level of 0.05, equal allocation between arms, and a normal approximation. It states that the effect to detect is a stated fraction of the observed effect. These are assumptions for planning illustrations, not a report of the trial’s own design justification. The registry record does not report a sample-size justification, the effect size used in the original design, or a statistical analysis plan.

Sample-size calculations estimate the amount of information a future or planned study would need under specified assumptions. They do not report how much power the completed trial actually had after enrollment, follow-up, missing data, and the observed outcome variability. Any sample-size illustration is a planning aid, not “observed power.” The registry record does not provide the primary-outcome effect estimate needed to judge what the observed effect was in this trial. It also does not report interim analyses or stopping rules.

Why can repeated outcomes complicate interpretation?

The primary outcome has assessments at 12 and 24 weeks, while the registry also lists multiple secondary outcome domains. In general, examining several outcomes or time points creates more than one opportunity to observe an apparently notable result. A statistical analysis plan may address this through a prespecified hierarchy, adjustment, or a clearly exploratory interpretation. The registry record does not report whether any such approach was used. Therefore, the result of any individual test, if reported elsewhere, should not be interpreted as though the multiplicity question had been resolved by the information in this record.

The record lists 11 outcome measures and zero analysis records. That means the registry information available for this analysis gives outcome descriptions but no registered analysis outputs. It does not establish that no analysis was conducted; it means analysis details and results are not reported in the registry record. No recalculated efficacy estimate can be presented from the outcome information recorded.

10. Multiplicity, Missing Data and Analysis Populations

The registry lists a primary social-function outcome assessed at 12 and 24 weeks, alongside secondary outcomes covering social cognition, social function, social responders, repetitive behaviors, anxiety, and quality of life. It lists 11 outcome measures. When a study examines multiple outcomes or time points, the chance of obtaining at least one apparently notable finding can be affected by how many comparisons are made. In general, a prespecified testing hierarchy or another multiplicity strategy can help clarify which comparisons are confirmatory and how results should be interpreted.

The registry record does not report a multiplicity adjustment, testing hierarchy, or analysis plan for the multiple outcomes and assessment times. It also does not report primary-outcome estimates or secondary-outcome results. Without these details, readers cannot determine which comparisons were treated as primary for inference, whether results were adjusted, or whether the analyses followed a prespecified plan.

Missing-data methods are also not reported in the registry record. There is no account of missing outcome assessments, reasons for missingness, imputation, or sensitivity analyses. The record does not describe analysis populations, such as whether results were intended to include all randomized participants or only those with available outcome data. These omissions matter because the people contributing observed scores may differ from those without recorded follow-up, and analysis choices can affect the estimated group comparison.

The record lists 11 outcome measures and zero analysis records. That does not show that analyses were never performed; it means their methods and results are not reported in the registry record. Conclusions about multiplicity, missingness, and analysis populations therefore cannot be drawn from the registry information available for this analysis.

11. Safety Results

The registry reports serious adverse events as affected participants divided by participants at risk.

Deaths

Other (non-serious) adverse events

Participants with at least one non-serious event at or above the registry's 0% reporting threshold.

Most frequent non-serious adverse event terms

The registry safety record lists 30 participants at risk in each arm. It reports zero serious adverse events in the intranasal oxytocin arm and zero in the placebo arm. It reports other adverse events for 28 of 30 participants in the oxytocin arm and 25 of 30 participants in the placebo arm. Deaths are recorded as zero in both arms.

These figures are counts as recorded, not a complete description of the safety experience. The registry does not list serious adverse event terms, and it does not provide the types of other adverse events, their severity, timing, duration, or relationship to study treatment. The meaning of “other adverse events” is therefore limited to the category and counts recorded; the record does not give enough detail to compare event profiles.

A zero count in the registry should be read as no events recorded in that category for the listed at-risk participants, not as proof that an event is impossible or that all relevant safety information is captured. The record does not report follow-up completeness or how adverse events were collected. The counts alone cannot establish a difference in safety between the groups.

12. What the Result Does, and Does Not, Mean

The registry record describes a randomized comparison of intranasal oxytocin and placebo in children and adolescents with autism spectrum disorder. It specifies a primary measure of change in ABC Social Withdrawal Subscale score at 12 and 24 weeks, where lower scores indicate improvement. It also records safety counts for serious and other adverse events and deaths.

What the record does not provide is an efficacy estimate for the primary outcome. There are no group-level changes, between-group contrasts, confidence limits, or test results in the information available for this analysis. As a result, the posted record cannot show whether the observed social-function scores differed between groups, how large any difference was, or how precisely it was estimated.

The stated scale direction is not an outcome finding: it explains how to interpret a score, not what happened to participants’ scores. Similarly, the study’s randomized and masked design describes intended methods, not proof that implementation was successful or that bias was eliminated. The registry does not report the analysis population, missing-data approach, or detailed analysis plan needed to evaluate the robustness of an efficacy estimate.

The safety counts offer a limited descriptive account. They show no serious adverse events or deaths recorded in either arm, and they list other adverse events in both arms. They do not include event descriptions or sufficient context to characterize or compare safety outcomes comprehensively.

The trial’s completion status and recorded enrollment do not fill these reporting gaps. Nor do planning assumptions for sample-size illustrations establish the trial’s observed efficacy or power. The registry record can identify what the study set out to measure and what safety counts were entered, but it does not support a conclusion about the size, precision, or clinical importance of an oxytocin effect on social function.

13. Why This Trial Matters Statistically

This trial illustrates why design information and outcome information need to be read separately. The registry identifies a randomized, quadruple-masked, parallel-group comparison and names a specific social-function measure. Those details tell readers what kind of comparison was planned. They do not provide the estimate needed to understand the result. For the primary outcome, the registry record does not report group changes or a between-group contrast.

The endpoint description also makes clear that direction matters. The ABC Social Withdrawal Subscale ranges from 0 to 48, with lower scores designated as improvement. To interpret an actual result, readers would need not only a directionally defined scale, but also an effect estimate, its uncertainty, and context for whether a change is important. The registry record does not report a threshold for meaningful change or the result’s confidence interval.

The listed assessment times and secondary outcome domains raise statistical questions that are common in clinical research. Multiple outcomes and time points require clear prespecification and transparent analysis reporting. Missing-data handling and the analysis population also affect how a result should be understood. Those details are not reported in the registry record, so the study cannot be evaluated on these dimensions from the available information.

The safety counts demonstrate a further distinction: registry entries can offer limited numerical descriptions without enough context for a full interpretation. Counts of serious and other adverse events do not explain event types, timing, severity, or relatedness.

Statistically, the central lesson is that trial design, outcome definition, estimation, and reporting each contribute different information. Here, the design and endpoint are described, but the efficacy results and important analysis details are not reported. Keeping those distinctions explicit prevents a study’s rationale or planned methods from being mistaken for evidence about its observed effect.

14. Limitations

• The posted summary statistics are the only data these results rest on; patient-level data, full protocols and statistical analysis plans are not available.
• Recalculated results are simple, unadjusted calculations from posted summaries. They do not replace the sponsor's prespecified analysis.

The main limitation for interpreting this trial from the registry record is the absence of reported primary-outcome results. Although the record describes the ABC Social Withdrawal Subscale, the direction of improvement, the mean summary, the 95% confidence interval, and assessments at 12 and 24 weeks, it does not give score changes or a between-group estimate. Efficacy magnitude and precision therefore cannot be assessed.

The record does not provide participant flow, baseline characteristics, adherence, treatment exposure, or reasons for missing follow-up. It also does not describe the analysis population or missing-data methods. These details would help determine how the analyzed participants and observed scores relate to the randomized groups.

The statistical analysis plan is not reported in the registry record. In particular, the record does not explain the handling of the two primary assessment times, the multiple secondary outcomes, or multiplicity. It does not report whether analyses were adjusted for baseline scores, identify any interim analyses, or describe the method used for confidence intervals. The exact implementation of randomization and masking is also not described.

Safety reporting is limited to counts. Serious adverse event terms are not listed, and other adverse events are not described by type, severity, timing, or relationship to treatment. The registry record does not provide enough context to assess completeness of event collection or follow-up.

Finally, the record lists a completed trial but does not provide a full results account. Its sample-size assumptions describe planning illustrations, not the trial’s original justification or observed power. These reporting limitations do not establish that the unreported methods or results do not exist; they mean they are not reported in the registry record.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: Intranasal Oxytocin for the Treatment of Children and Adolescents With Autism Spectrum Disorders (ASD) — NCT01908205.
• Registry record last updated 2025-07-16; status last verified 2020-10.
• Results first posted 2020-11-16.
• Data retrieved from ClinicalTrials.gov on 2026-11-09.
• Group sizes, means, event counts and sponsor-reported p-values are reproduced as posted. Effect estimates, confidence intervals and p-values not attributed to the sponsor are independently calculated from the posted summary statistics.

18. Record Summary

The registry identifies NCT01908205 as a completed, randomized, quadruple-masked, parallel-group phase 2 trial sponsored by Holland Bloorview Kids Rehabilitation Hospital. It records enrollment of 60 and a study start of 2013-09, with primary completion in 2015-09. The population is children and adolescents with autism spectrum disorder.

The comparison is intranasal oxytocin, identified as Syntocinon, versus placebo. The proposed schedule for both arms is 0.4 IU/kg twice daily, with a maximum of 24 IUs per dose. The primary outcome is change in ABC Social Withdrawal Subscale score at 12 and 24 weeks; lower scores indicate improvement.

The registry record does not report primary efficacy estimates or analysis details. Safety entries list zero serious adverse events and zero deaths in both arms, with other adverse events recorded for 28 of 30 participants in the oxytocin arm and 25 of 30 in the placebo arm. The event details and context needed for a fuller safety interpretation are not reported in the registry record.