This page provides an independent statistical analysis and educational interpretation of publicly reported results. ClinicalTrials.gov provides the official trial registry record. Numerical trial results on this page are restricted to the results posted in the ClinicalTrials.gov record; calculations added by Clinical Biostats are labelled as such.
1. Trial at a Glance
This page covers the registry results for Actigraphic Analysis of Treatment Response (NCT00723580), a na na study. The primary outcome is Actigraphic Measurement of Treatment Conditions.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00723580 |
| Phase | NA |
| Condition | Sleep Disorders, Circadian Rhythm, Insomnia, Psychomotor Agitation |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 1 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Child Psychopharmacology Institute |
| Results posted | Yes |
2. Clinical Question
Population
A single child anticipating pharmacological treatment, described in the official title as a six-year-old girl with Kabuki syndrome. The primary-outcome population is described as a single subject with repeated actigraphic, observational, and psychometric measurements.
Intervention
The registry lists risperidone as the drug intervention. It describes actigraphic measurement across treatment conditions, including baseline and a period when pharmacological treatment was initiated. Details for all treatment conditions are not reported in the registry record.
Comparator
This is a single-group study, not a randomized comparison. Baseline and other treatment conditions are repeated measurement periods within the same subject, rather than a separate control group. The record does not identify a like-for-like comparator.
Primary question
The primary question is whether actigraphic measures of activity and sleep vary across treatment conditions. The description identifies impulsivity and inability to sleep as concerns and says they will be assessed actigraphically. Numerical outcome results and a statistical comparison are not reported in the registry record.
3. Trial Design
This completed record describes an individual, single-group study, with an enrollment of 1. Allocation is listed as NA, and masking is listed as NONE. The record therefore does not describe random assignment, blinded assessment, or a parallel control group. Its structure is based on repeated assessment of one child across treatment conditions.
The official title identifies a six-year-old girl with Kabuki syndrome. The listed conditions also include sleep disorders, circadian rhythm; insomnia; and psychomotor agitation. These registry entries describe the context of the study, but the record does not establish that each listed condition was a separately measured diagnosis or outcome.
Actigraphic measurements were obtained using a watch device attached to the child's non-dominant wrist. The primary-outcome population description refers to actigraphic readings at thirty second intervals, alongside observational measurements every two months and psychometric assessments at baseline and at 23 months. It also describes three 21 day actigraphic periods across five treatment conditions. The arm description refers to baseline and treatment initiation, but detailed descriptions of every condition are not reported in the registry record.
The study start is recorded as 2008-05, primary completion as 2010-07, and status as COMPLETED. The primary-outcome timeframe is May 12- July 14, 2010. The sponsor is Child Psychopharmacology Institute. These design details describe a case-based repeated-measurement record, not a comparative trial capable of separating treatment effects from time, changes in circumstances, or other influences.
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: The child had previously not responded to multiple medication trials, was stimulated by diphenylhydramine, over-sedated on clonidine and had mood destabilization when tried on mirtazapine. The child received no medication for the month preceding the initiation of the study.
Overall Study
| Stage | Actigraphic (Activity Count) Measurement of Sleep and Activity |
|---|---|
| STARTED | 1 |
| Baseline Measurements | 1 |
| COMPLETED | 1 |
| NOT COMPLETED | 0 |
Analysis population for the primary outcome, as defined in the registry: Single subject repeated Actigraphic(at thirty second intervals), observational (every two months) and psychometric (baseline and at 23 months) measurements of a child anticipating pharmacological treatment. Actigraphic measurements (three 21 day periods) over five treatment conditions that included a non-medication baseline and spanned 23 months.
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.
| Characteristic | Unit | Actigraphic (Activity Count) Measurement of Sleep and Activity |
|---|---|---|
| Participants analysed | 1 | |
| Age, Categorical: <=18 years | Participants | 1 |
| Age, Categorical: Between 18 and 65 years | Participants | 0 |
| Age, Categorical: >=65 years | Participants | 0 |
| Age, Continuous | years | 6 (SD 0) |
| Sex: Female, Male: Female | Participants | 1 |
| Sex: Female, Male: Male | Participants | 0 |
| Region of Enrollment: United States | participants | 1 |
6. Primary Endpoint
The registry lists the primary outcome as Actigraphic Measurement of Treatment Conditions, measured over May 12- July 14, 2010.
| Element | Registry entry |
|---|---|
| Result type | Mean |
| Measure of spread | Standard Deviation |
| Unit | activity |
| Endpoint classification | Continuous Endpoint |
The primary outcome is titled Actigraphic Measurement of Treatment Conditions. It is a continuous measure summarized as a mean, with standard deviation identified as the measure of dispersion and activity listed as the unit. The description says actigraphic assessment was intended to capture impulsivity and inability to sleep across treatment conditions. The actigraphic device was worn on the non-dominant wrist, and the population description specifies readings at thirty second intervals.
Actigraphy provides repeated measurements of movement over time. In general, such measurements can help characterize patterns of activity and rest in the context of a person's daily routine. Repeated readings may also make it possible to describe how measured activity varies among defined periods. The record's stated rationale is to examine the utility of actigraphic measurements for assessing treatment response.
The registry identifies three 21 day actigraphic periods across five treatment conditions, but condition-specific definitions and the precise construction of the primary summary are not reported in the registry record. Most importantly, no mean, standard deviation, treatment-condition estimate, or numerical comparison is reported. The endpoint definition therefore describes what was intended to be measured, not what the study found.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record establishes the study's single-subject, repeated-measurement structure and names actigraphic activity as the primary outcome. It does not report numerical results for that outcome. There is no posted mean or standard deviation, nor a reported estimate of change between baseline and another treatment condition. As a result, the magnitude, direction, and precision of any observed difference cannot be evaluated from the registry record.
The record lists 12 outcome measures and 0 analyses. The primary endpoint is described, and secondary outcomes include ratings of impulsivity, irritability, distractibility, and hyperactivity, as well as scales from the Personality Inventory for Children. However, outcome descriptions are not the same as results. No numerical findings for these measures are reported in the registry record.
The design also affects what a comparison could mean. Measurements from the same child across periods may reveal within-person patterns, but the study has no randomized control group. If activity differed across conditions, that pattern alone would not establish that risperidone caused the difference. Changes over time, circumstances, or measurement conditions could also contribute. The registry record does not report an analysis that addresses these possibilities.
Precision cannot be assessed without outcome estimates and measures of uncertainty. In general, a confidence interval describes a range of values compatible with data under a stated statistical model; it conveys more about precision than a point estimate alone. No confidence interval or other uncertainty estimate for the primary outcome is reported in the registry record. The listed standard deviation is a measure intended to describe variability, not a reported numerical result.
The enrollment of 1 is central to interpretation. Repeated measurements can provide detailed observations for an individual, but they do not create independent participants or support a population-level comparison in the same way as a larger controlled study. The registry record does not report a sample-size justification, a statistical analysis plan, interim analyses, or an inferential analysis. These omissions prevent readers from determining how the recorded measurements were intended to be analyzed or how the results would have been interpreted under a prespecified plan.
Accordingly, the registry record supports a description of the study's purpose and measurement approach, but not a conclusion about the size or certainty of a treatment-associated change. The record is marked COMPLETED, but that status does not itself indicate that results were posted. The appropriate statistical reading is that the outcome and design are described while numerical findings and analyses are not reported in the registry record.
9. Statistical Methods Explained
The study is a single-group repeated-measurement design. The same subject was assessed across treatment conditions, with actigraphy as the primary measurement approach and observational and psychometric assessments also described. This structure differs from a randomized parallel-group trial: it does not allocate separate groups to treatment and control, and it does not provide a randomized between-group contrast. The registry lists allocation as NA and masking as NONE.
What does a mean and standard deviation describe?
The primary endpoint is specified as a mean of a continuous activity measure, with standard deviation as its dispersion summary. In general, a mean describes the central level of a set of measurements. A standard deviation describes how dispersed individual measurements are around that mean. The two summaries answer different questions: the mean characterizes average activity, while the standard deviation characterizes variability in the measurements.
Neither summary is reported numerically in the registry record. The endpoint's statistical specification should therefore be read as a description of the intended summary, not evidence that a particular mean or amount of variability was observed. The record also does not explain whether the summary would be calculated over each period, condition, or another defined unit of analysis.
Why does repeated measurement matter?
Actigraphy is described as being collected at thirty second intervals, with three 21 day periods across five treatment conditions. In general, repeated observations from one person are related: readings close together in time may be more similar than readings far apart. Treating every reading as if it came from an independent person can understate uncertainty. A statistical model for repeated data may account for this dependence, but the registry record does not report a model, a variance structure, or a method for handling within-period correlation.
Repeated measurements can offer a detailed account of a person's course over time. They do not, by themselves, resolve the distinction between treatment-associated changes and other time-related changes. Without a randomized comparator, observed differences across periods may reflect treatment, time, changing circumstances, measurement conditions, or a combination. The registry record does not report an analysis designed to separate these explanations.
What would an estimate of uncertainty add?
In general, a confidence interval accompanies an estimate and conveys its statistical uncertainty under a specified model and assumptions. A p-value addresses compatibility between the observed data and a stated null hypothesis under the analysis assumptions; it does not measure the size or practical importance of an effect. Neither a confidence interval nor a p-value for the primary outcome is reported in the registry record. No inferential analysis is listed.
Over-dispersion is a general concern when observed variability exceeds what a statistical model assumes. If a model does not accommodate that extra variation, uncertainty estimates may be too small and inferential conclusions may be unreliable. Whether over-dispersion was assessed or relevant to these measurements is not reported in the registry record. In this single-subject repeated-measurement setting, the dependence and variability structure would need to be described before readers could judge the adequacy of any model.
How should planning calculations be understood?
In general, sample-size calculations estimate how many participants a hypothetical study might need under specified assumptions about an effect, variability, allocation, and statistical testing. The planning assumptions associated with this record describe a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. They also define the target effect as a stated fraction of an observed effect. These are planning conventions, not observed results or evidence that the original study had a two-arm design.
The record itself is single-group and has an enrollment of 1. A planning illustration based on an observed effect is not a report of observed power, and a calculated participant count would not change the study's actual design or enrollment. Sample-size illustrations are planning scenarios, not a way to establish that an individual result is statistically reliable. The registry record does not report the trial's own sample-size justification.
More broadly, the registry does not report the statistical analysis plan, the model used for repeated observations, the handling of missing measurements, or the analysis population. Those details are important because they determine how repeated readings are summarized and how uncertainty is calculated. In their absence, interpretation should remain descriptive and should not imply an unreported inferential method.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists 12 outcome measures and 0 analyses. Several secondary outcomes concern observed impulsivity, irritability, distractibility, and hyperactivity; others are scales from the Personality Inventory for Children. The record does not report how many outcomes were planned for formal testing, which outcomes were prioritized beyond the primary designation, or whether any adjustment for multiple comparisons was planned.
In general, testing many outcomes can increase the chance that at least one appears unusual by chance. Multiplicity methods can address that issue, but they involve choices about which outcomes belong to a testing family and how error rates are controlled. No such method is reported in the registry record. Since numerical results and analyses are not reported, there is no basis for assessing the impact of multiplicity on findings.
Missing-data procedures and the analysis population are also not reported in the registry record. The enrollment is 1, but that does not establish how many actigraphic readings were usable or whether every planned observational and psychometric assessment was completed. The record does not state how gaps, invalid readings, or incomplete assessments were handled. It also does not specify a rule for including or excluding measurements from analyses.
In general, missingness can affect interpretation when the availability of measurements is related to the outcome or to the circumstances under study. The appropriate approach depends on the pattern and reasons for missing data, neither of which is reported in the registry record. These omissions limit reproducibility and make it impossible to assess whether any analysis represented all planned observations.
11. Safety Results
The registry reports serious adverse events as affected participants divided by participants at risk.
| Arm | Participants with serious adverse events | Participants at risk | Percentage |
|---|---|---|---|
| Actigraphic (Activity Count) Measurement of Sleep and Activity | 0 | 1 | 0.0% |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 0% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Actigraphic (Activity Count) Measurement of Sleep and Activity | 0 of 1 |
The adverse-event record lists one participant at risk for serious adverse events and zero affected by serious adverse events. It also lists one participant at risk for other adverse events and zero affected by other adverse events. No serious-event terms are listed. The death field is null, so deaths are not reported in the registry record.
These entries are registry counts for the recorded study and should be read narrowly. Zero affected means that no events are recorded in the corresponding field; it does not establish that no events occurred outside the registry's reporting or observation process. The record does not provide event definitions, assessment procedures, observation duration for adverse events, or additional context for interpreting these counts.
Because the study enrolled 1, the safety information concerns a very limited experience. The registry does not provide a comparative safety assessment, and its single-group design cannot establish whether an event count differs from what would have occurred under another condition. The figures describe what is entered in the adverse-event record; they do not support broader conclusions about safety.
12. What the Result Does, and Does Not, Mean
The study addresses a measurement question: whether actigraphic assessment can be used to characterize activity and sleep-related treatment conditions for an individual child. It combines actigraphy with observational and psychometric measurements. The registry identifies risperidone as the intervention and describes baseline and treatment-initiation periods, while also referring to repeated measurements across treatment conditions.
The record does not report numerical outcome results. It therefore cannot establish how activity differed across conditions, whether any change was large or small, or how precisely a change was estimated. It also does not report a statistical analysis linking the measurements to a treatment effect. The COMPLETED status indicates the study's registry status, not that the findings are numerically available.
Even if a difference across periods had been reported, the design would require careful interpretation. A single-subject repeated-measurement study can describe patterns for that individual. Without randomization or a separate control group, it cannot isolate treatment from time-related changes or other influences. The registry record does not report methods to address those alternative explanations.
In general, a study's statistical evidence depends on the outcome data, analysis choices, assumptions, and uncertainty estimates—not solely on the stated aim or the number of repeated readings. Those elements are not reported for the primary outcome in this record. Consequently, the study description supports understanding of the planned measurement approach, but not a conclusion about whether treatment changed activity or sleep.
The distinction is important: a stated purpose is not a result, and a completed registry entry is not itself evidence for or against a treatment effect. The record can be informative about an individual measurement strategy while remaining insufficient for a quantitative assessment of response or for generalization beyond the subject studied.
13. Why This Trial Matters Statistically
Statistically, this record is notable for placing repeated measurement in a single-subject setting. Actigraphic readings at thirty second intervals and multiple treatment-condition periods can yield a detailed within-person measurement history. Such data raise practical issues about how readings are summarized, how observations that are close in time are related, and how uncertainty should be represented.
The design also illustrates the difference between collecting many measurements and having many independent participants. Repeated readings may enrich the description of one person's activity, but they do not turn an enrollment of 1 into a multi-participant trial. The unit of inference matters: evidence about a measured pattern in one subject is not automatically evidence about a broader population.
The registry's stated aim is to examine and illustrate the utility of actigraphic measurements for assessing treatment response. That is a measurement-focused aim, distinct from establishing a comparative treatment effect. The absence of numerical results means the record does not allow readers to assess the performance of the measurement strategy or the observed course in this subject.
The record also highlights why transparency about statistical methods matters. For repeated data, readers need to know how periods and conditions were defined, how readings were summarized, how dependence and missingness were handled, and how uncertainty was assessed. Those analysis details are not reported in the registry record. A clear measurement schedule alone cannot answer those questions.
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 principal limitation is the enrollment of 1 in a single-group design. This record has no randomized comparator and no like-for-like control group. It can describe repeated measurements in one subject, but it cannot by design provide a randomized estimate of the difference between treatment and control conditions.
The registry record does not report numerical results for the primary activity measure or the listed secondary outcomes. It lists 12 outcome measures and 0 analyses, leaving no reported estimates, uncertainty measures, or inferential findings to evaluate. The measure's dispersion is identified as standard deviation, but no numerical standard deviation is reported.
The description refers to five treatment conditions and three 21 day actigraphic periods, but the condition-by-condition details are not reported in the registry record. The intervention list names risperidone, while the record does not fully describe the timing or content of all treatment conditions. That makes it difficult to determine exactly which periods would be compared or how they relate to the intervention.
The statistical analysis plan, sample-size justification, interim analyses, analysis population, missing-data procedures, and methods for addressing repeated-measure dependence are not reported in the registry record. The record also does not describe whether measurement quality or adherence was assessed. These limitations prevent a detailed appraisal of the intended analysis and the completeness of the measurements.
Finally, a single-subject study has limited generalizability. Even a detailed individual record cannot establish how measurements or treatment-associated patterns would apply to other people. In this case, the absence of posted numerical results further limits what can be concluded from the registry entry.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Actigraphic Analysis of Treatment Response — NCT00723580.
- Registry record last updated 2015-08-18; status last verified 2015-07.
- Results first posted 2010-12-14.
- 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.
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18. Record Summary
NCT00723580 is a completed, single-group study titled Actigraphic Analysis of Treatment Response. The official title describes a six-year-old girl with Kabuki syndrome. Enrollment was 1; allocation was NA and masking was NONE. Risperidone is listed as the drug intervention.
The primary outcome concerns mean actigraphic activity across treatment conditions, with standard deviation identified as the dispersion measure. The record describes wrist-worn actigraphy, readings at thirty second intervals, three 21 day periods, and five treatment conditions. Its primary timeframe is May 12- July 14, 2010.
The registry lists 12 outcome measures and 0 analyses. Numerical results for the primary endpoint, uncertainty estimates, and statistical methods are not reported in the registry record. The adverse-event fields record zero serious and zero other adverse events among one participant at risk; deaths are not reported in the registry record.