Telehealth Delivery of Treatment for Sleep Disturbances in Young Children With Autism Spectrum Disorder: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Telehealth Delivery of Treatment for Sleep Disturbances in Young Children With Autism Spectrum Disorder (NCT03668873), a randomized na study. The primary outcome is Change From Baseline on MSPSQ-CSI.

2. Clinical Question

The clinical question concerns the comparative results of two telehealth-delivered programs, not a comparison with no intervention. Both arms received a program intended for families of young children with autism spectrum disorder and sleep problems. The primary measure was completed by the child’s primary caregiver, and higher scores indicate worse sleep-related outcomes on the index.

The record describes a randomized clinical trial with a parallel design. Random assignment supports a comparison of groups as assigned, but the information available does not include the numerical primary outcome results needed to judge the direction, size, or precision of any difference. Therefore, the record supports describing the study question and measurement approach, but not stating which program had a more favorable observed result.

3. Trial Design

The registry describes a randomized, single-masked, parallel-group clinical trial sponsored by The Cleveland Clinic. The trial status is completed. The recorded enrollment is 91, while the study-design summary says ninety children would be recruited. The record does not explain the difference between those enrollment descriptions.

Children were assigned to Sleep Parent Training or Sleep Parent Education. Both are identified as behavioral interventions and were delivered individually through a telehealth platform. The design summary describes five sessions in each program over a 10-week period, with real-time parent-child coaching. The Sleep Parent Training description says sessions could be reordered after Session A to address child-specific problems. The Sleep Parent Education description includes a sleep hygiene session modeled from the RUBI manual. Further details of either program are not reported in the registry record.

The primary measure was collected at baseline, week 5, and week 10. The brief design summary also describes a week 16 follow-up intended to assess durability. The primary outcome timeframe, however, is listed as baseline, 5 weeks, and 10 weeks. Week 16 primary outcome results are not reported in the registry record.

The study began on 2018-11-01, and primary completion is listed as 2022-11-20. The record identifies masking as single but does not say which people were masked. It does not report further details about the randomization process, allocation concealment, or procedures used to maintain masking. The registry lists phase as NA; a phase designation is therefore not specified.

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.

Before assignment: 140 children were pre-screened and 102 were found to be eligible for a full screen. Of those, 91 consented. There were 14 screen failures (4 whose CSI was less than 5, 7 whose ASD diagnosis could not be corroborated, and 3 who did not follow through with full screening). Leaving 77 participants to be randomized, data were available for 36 participants randomized to SPT and 38 participants to SPE after 2 drop out in SPT and 1 in SPE before the intervention was initiated.

Overall Study

Reasons for not completing this period:

Analysis population for the primary outcome, as defined in the registry: A modified intention-to-treat principle was used for the primary analysis, in which participants who attended at least one treatment session were analyzed in the group to which they were randomized.

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 Change From Baseline on MSPSQ-CSI, measured over Baseline, 5 weeks, and 10 weeks.

The primary endpoint is change from baseline on the Modified Simonds & Parraga Sleep Questionnaire-Composite Sleep Index (MSPSQ-CSI). The child’s primary caregiver completed the modified questionnaire at baseline and at weeks 5 and 10. The index combines scores for the frequency of targeted sleep problems—bedtime resistance, night waking, early waking, and sleeping in places other than bed—with scores for the duration of sleep latency and night wakings.

The index ranges from a minimum score of 0 to a maximum score of 12; higher scores indicate a worse outcome. As a general interpretation, a change in a repeated measure can describe whether scores moved over time, while a comparison between randomized groups can help assess whether their observed changes differed. The registry record identifies the planned measure and its direction, but does not report the group-specific changes or a between-group estimate.

This caregiver-reported composite is used to represent several sleep-related concerns in a single outcome. Combining targeted problems can make the endpoint more concise than interpreting each questionnaire item separately, but it also means the overall score does not show which particular sleep problem accounts for a change. The record says the sample sizes reflect participants who completed surveys at each follow-up. It does not report the number of completed surveys at each time point in the information available.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

What does the primary estimate represent?

The registry identifies the primary parameter as a least-squares mean and lists a 95% confidence interval as its dispersion measure. In general, a least-squares mean is an estimated mean from a statistical model. It can be useful when a model accounts for features of the data or study design, rather than relying only on an unadjusted arithmetic average. The registry record does not describe the model used to produce the primary estimates, the covariates included, or the exact contrast used for the treatment comparison.

The outcome is a continuous score on the MSPSQ-CSI. The stated score range is 0 to 12, with higher scores indicating worse outcomes. The endpoint title refers to change from baseline, and the recorded time points are baseline, week 5, and week 10. A change score describes movement relative to a participant’s baseline measurement; a comparison of changes between randomized groups addresses whether the groups’ observed trajectories differed. No numerical estimates for either group or their comparison are reported in the registry record.

Why is a confidence interval useful?

In general, a confidence interval communicates statistical uncertainty around an estimate. A narrower interval indicates greater precision under the model and assumptions used; a wider interval indicates more uncertainty. The record specifies a 95% confidence interval but does not provide its bounds. The interval therefore cannot be used to judge the precision of the primary estimate or whether it includes values that might be considered important. A confidence interval is not a guarantee that the true effect lies within the displayed bounds, and it does not by itself establish clinical importance.

What does randomization contribute?

Random allocation is intended to make the groups comparable, on average, at the start of a trial, including with respect to factors that were not measured. In a parallel-group design, participants are assigned to one program and remain in that assigned group for the comparison. This helps provide a basis for interpreting an outcome contrast as a comparison of assigned interventions, subject to the analysis population, missing data, adherence, and other study limitations. The registry identifies the trial as randomized and parallel, but does not give details of the sequence generation or allocation concealment.

The masking designation is single. The record does not identify who was masked or explain how masking was implemented. Because the outcome was completed by a child’s primary caregiver and the interventions involved parent-facing sessions, details about masking and outcome assessment would be relevant to understanding potential sources of bias, but they are not reported in the registry record.

How should planning calculations be read?

In general, sample-size calculations estimate how many participants would be needed under specified assumptions to detect a chosen effect with a chosen probability, while controlling a chosen significance level. The registry’s planning assumptions describe a two-sided significance level of 0.05, equal allocation between arms, and a normal approximation. They also describe the target effect as a stated fraction of the observed effect. Such calculations are planning illustrations, not observed power and not evidence that the trial detected an effect.

The record does not provide the numerical planning counts or the fractions used in the calculations in the information described. It also does not report the original design’s sample-size justification or its assumptions. The planning illustration should not be treated as a substitute for the trial’s observed estimate and confidence interval. A calculation based on an observed effect can be especially dependent on that observed estimate; without the estimate and the calculation outputs, readers cannot assess the resulting planning values.

Why do analysis populations matter?

The primary analysis used a modified intention-to-treat rule: participants who attended at least one treatment session were analyzed according to their randomized group. In general, analyzing participants in their assigned groups retains the comparison created by randomization for those included. A modified rule can depart from a full intention-to-treat analysis when some randomized participants are excluded; the registry’s rule specifies attendance at one or more sessions as the inclusion condition. The record does not report how many participants were included under that rule, how many were excluded, or whether additional analysis populations were used.

For a repeated caregiver-reported outcome, follow-up completion can affect which observations contribute to estimates. The registry says sample sizes reflect participants who completed surveys at each follow-up. It does not describe imputation, model handling of incomplete repeated measures, sensitivity analyses, or whether missingness was examined by treatment group. These details are important because complete-case results and model-based results can depend on assumptions about why measurements are missing. No such assumptions or methods are reported in the registry record.

10. Multiplicity, Missing Data and Analysis Populations

The registry lists six outcome measures and five analyses. It identifies the MSPSQ-CSI as the primary outcome and names changes in ABC-Irritability, PSI, and PSOC among the secondary outcomes. The primary timeframe includes baseline, week 5, and week 10, while the design summary also describes follow-up at week 16. These multiple measures and assessment occasions can create multiple opportunities to observe an apparently notable result.

In general, multiplicity refers to the increase in false-positive risk that can arise when many statistical tests are conducted. Researchers may address it by specifying a primary comparison, using an adjustment procedure, or interpreting secondary analyses as exploratory. The registry record does not report a multiplicity adjustment, a hierarchy of tests, or a complete statistical analysis plan. It is therefore not possible to determine how the analyses were prioritized or how any multiplicity was handled.

The record states that primary-analysis sample sizes reflect participants who completed surveys at each follow-up, and defines the modified intention-to-treat population as participants who attended at least one treatment session, analyzed in their randomized group. It does not give follow-up completion counts, missing-data methods, or sensitivity analyses. It also does not report whether there were interim analyses or how the analysis population was determined for the secondary outcomes. Those items are not reported in the registry record and should not be inferred.

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 serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: pica event (3.1%).

The registry reports serious adverse events for 32 participants at risk in Sleep Parent Training and 36 participants at risk in Sleep Parent Education. One serious adverse event is recorded in each group. Deaths are recorded as 0 in both groups. The record lists a PICA event as the top serious term, with 3.1% shown alongside it; it does not provide further context for that term in the information available.

The registry also reports other adverse events for 32 participants at risk in Sleep Parent Training and 36 in Sleep Parent Education. There are 15 affected participants in the Sleep Parent Training group and 14 in the Sleep Parent Education group in that category. These are reported counts, not a basis for calculating new rates or for determining whether an event was caused by a program. No additional details about event types, severity definitions, timing, or attribution are reported in the registry record.

Adverse-event counts describe events recorded under the registry’s categories; they do not establish comparative safety on their own. Interpretation depends on the event definitions, ascertainment procedures, exposure, and completeness of reporting. The registry record does not supply those details, and the counts should not be treated as proof of either harm or absence of harm.

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

The trial is relevant statistically because it randomized participants between two active, telehealth-delivered behavioral programs and specified a caregiver-reported sleep composite as its primary endpoint. The record gives the direction of the score, the measurement schedule, and a rule for the primary analysis population. These elements help readers understand what was intended to be compared and how the outcome was defined.

However, a design description is not an outcome result. The registry record does not report the primary group estimates, their confidence intervals, or a between-group contrast. It therefore does not establish whether scores improved, worsened, or differed between assigned groups. It also does not show the magnitude or precision of any observed difference. A missing result is not evidence that no effect occurred, and it is not evidence that an effect occurred.

The primary composite combines several sleep concerns and duration measures into a score. An overall score can provide a concise measure, but without item-level results it cannot show which component contributed to any change. The record does not report the endpoint results needed to make even that broader interpretation.

The study design summary describes week 16 follow-up to assess durability, but the primary outcome timeframe is listed through week 10. Week 16 primary results are not reported in the registry record. Nor are numerical results for the named secondary outcomes included in the information available. The safety record gives event counts but does not establish attribution or comparative safety.

Accordingly, the defensible interpretation is limited to the trial’s design, intended outcome, and reported safety counts. Any claim that one program was more effective, that the programs were equivalent, or that benefits persisted would go beyond the numerical results reported in the registry record.

13. Why This Trial Matters Statistically

Several statistical features make this trial useful to examine as a study design. It is described as randomized and parallel-group, with two behavioral programs delivered through telehealth. That creates a direct comparison between alternative approaches rather than a comparison of a program with no treatment. The primary outcome is a continuous caregiver-reported composite, and the registry specifies its score direction and repeated assessment schedule.

The outcome definition also illustrates a common trade-off in clinical measurement. A composite can collect several related concerns into one score, potentially offering a compact summary. At the same time, an overall score can conceal variation among its components. The record describes the components of the MSPSQ-CSI, but does not report their separate results or the overall primary estimates.

The modified intention-to-treat rule is another important interpretive feature. Participants who attended at least one session were analyzed according to their randomized group. That rule is clearly stated, but the registry does not report the number included or details of missing follow-up data. Knowing the analysis rule is helpful; evaluating its consequences requires the associated counts and missing-data methods.

Finally, this record illustrates why a trial’s outcome reporting matters as much as its design. Without group estimates and uncertainty intervals, readers cannot assess the comparative result, regardless of the rigor suggested by randomization. The registry supports a clear description of the question and endpoint, while leaving the primary statistical conclusion unresolved in the information reported.

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.

A central limitation of the available registry information is that it does not report the numerical primary outcome estimates or a between-group comparison. Without these results, the size, direction, and precision of the primary result cannot be assessed. The registry identifies a 95% confidence interval but does not give its bounds.

The analysis details are also limited. The record defines a modified intention-to-treat population but does not report how many participants were analyzed, how many attended no treatment sessions, or how many completed surveys at each follow-up. Missing-data procedures, sensitivity analyses, model specifications, and any adjustment for baseline measures are not reported in the registry record.

The masking designation is single, but the record does not say who was masked or how. Details of random sequence generation and allocation concealment are likewise not reported. The registry lists six outcomes and five analyses, but does not provide a full statistical analysis plan or explain how multiplicity was handled.

The design summary describes week 16 follow-up, while the primary outcome timeframe lists baseline, week 5, and week 10. Week 16 primary results are not reported in the registry record. The brief design description says ninety children would be recruited, whereas enrollment is listed as 91; the record does not explain this difference. These limitations restrict interpretation of the available information, especially for comparative effectiveness and durability.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: Telehealth Delivery of Treatment for Sleep Disturbances in Young Children With Autism Spectrum Disorder — NCT03668873.
• Registry record last updated 2024-09-19; status last verified 2024-05.
• Results first posted 2024-09-19.
• 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 Cleveland Clinic sponsored a completed randomized, single-masked, parallel-group trial of Sleep Parent Training and Sleep Parent Education delivered by telehealth. The registry lists enrollment as 91; the design summary describes ninety children ages 2 to less than 7 years with autism spectrum disorder and sleep disturbance. Both programs were described as five individually delivered sessions over 10 weeks.

The primary outcome was change from baseline on the MSPSQ-CSI, measured at baseline, week 5, and week 10. Scores range from 0 to 12, with higher scores indicating worse outcomes. The primary analysis used a modified intention-to-treat rule for participants attending at least one session, analyzed in their randomized group.

The registry record does not report the primary group results or treatment contrast. It reports serious adverse events, other adverse events, and deaths by group, but does not establish event attribution. The record began on 2018-11-01, with primary completion listed as 2022-11-20.