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 Exploring the Use of Transdermal Methylphenidate to Reduce Fall Risk in Patients With Dementia. (NCT01825577), a na phase 4 study. The primary outcome is Timed Get Up and Go Test - Measure of Mobility.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01825577 |
| Phase | Phase 4 |
| Condition | Dementia, Accidental Falls, Apathy |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 14 |
| Arms in the primary outcome | n/a |
| Lead sponsor | St. Louis University |
| Results posted | Yes |
2. Clinical Question
Population
Elderly patients living in community nursing homes, identified as fall risks and diagnosed with probable Alzheimer’s disease.
Intervention
Transdermal methylphenidate: once daily 10mg for 2 weeks, followed by once daily 15mg for 2 weeks. The patch was to be worn for approximately 7-10hrs each day.
Comparator
There was no separate control group. The study is registered as a single-group, open-label study, with screening or baseline measurements serving as the reference for the planned post-treatment assessment.
Primary question
Does use of transdermal methylphenidate correspond to a reduction in fall risk, assessed through gait and mobility measures, in patients with dementia? The primary endpoint was the Timed Get Up and Go Test.
The registry frames fall risk as something assessed through measures that predict the likelihood of future falls. The primary aim was to determine whether transdermal methylphenidate reduced fall risk. The stated hypothesis was that gait and mobility assessment scores would improve after treatment compared with scores at screening.
This was not a randomized comparison of methylphenidate with placebo or another treatment. A change from baseline in a single group can describe what happened to participants over the study period, but it cannot by itself establish that treatment caused the change. Changes over time may also reflect factors not separated from treatment in this design.
The registry describes apathy as an additional area of interest and lists the Performance Oriented Mobility Assessment as a secondary outcome. The primary outcome was mobility measured by the Timed Get Up and Go Test. Although the record lists outcome analyses, it does not report numerical endpoint results or an estimated change. It is therefore not possible to assess from the registry record whether the planned improvement was observed or how precisely it was estimated.
3. Trial Design
This was a phase 4, single-group, open-label study sponsored by St. Louis University. Allocation is listed as not applicable, and the registry gives no masking. Enrollment was 14. The record lists the study status as terminated, with a start date of 2012-11 and primary completion in 2015-02. The reason for termination is not reported in the registry record.
Participants were to receive transdermal methylphenidate on a once-daily schedule: 10mg for 2 weeks, then 15mg for 2 weeks. The patch was to be worn for approximately 7-10hrs per day. The primary outcome timeframe was baseline and a post-test at 4 weeks. The registry identifies the intended population as elderly patients living in community nursing homes who were considered fall risks and had a diagnosis of probable Alzheimer’s disease.
Because the study had no separate comparator arm, its main planned comparison was within participants, between baseline and the post-test. That design can describe change over time in the enrolled group. It does not provide the concurrent control needed to distinguish a treatment-associated change from changes that might have occurred over time for other reasons. The open-label design also means the registry does not describe blinded outcome assessment.
The registry record does not report the reason for early termination, participant flow, the number who completed the treatment period, or the number included in each endpoint analysis. It also does not report a protocol-level statistical analysis plan, the planned handling of missing measurements, or a sample-size justification for this single-group study. The record’s sample-size assumptions describe an illustrative two-arm calculation, rather than the design registered for this trial; those assumptions should not be mistaken for the trial’s own design rationale or observed evidence.
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
| Stage | Single Arm Transdermal Methylphenidate |
|---|---|
| STARTED | 14 |
| COMPLETED | 6 |
| NOT COMPLETED | 8 |
Reasons for not completing this period:
| Reason | Single Arm Transdermal Methylphenidate |
|---|---|
| Physician Decision | 2 |
| Withdrawal by Subject | 1 |
| Withdrawn by family members | 2 |
| Lack of Efficacy | 3 |
Analysis population for the primary outcome, as defined in the registry: Elderly patients living in community nursing homes identified as fall risks with a diagnosis of probable Alzheimer's disease.
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 | Single Arm |
|---|---|---|
| Participants analysed | 14 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 0 |
| Age, Categorical: >=65 years | Participants | 14 |
| Sex: Female, Male: Female | Participants | 8 |
| Sex: Female, Male: Male | Participants | 6 |
| Region of Enrollment: United States | participants | 14 |
6. Primary Endpoint
The registry lists the primary outcome as Timed Get Up and Go Test - Measure of Mobility, measured over Baseline and Post-test at 4 weeks.
| Element | Registry entry |
|---|---|
| Result type | Mean |
| Measure of spread | Standard Deviation |
| Unit | seconds |
| Endpoint classification | Continuous Endpoint |
The primary endpoint was the Timed Get Up and Go Test, a measure of mobility reported as the mean time in seconds. The test records how long it takes a participant to rise from a chair, walk 10 feet, turn around, walk back, and sit down. The registry notes that the test takes less than 5 minutes to complete.
The test is used because it captures several elements of functional mobility within a single timed task: standing up, walking, turning, returning, and sitting. In this study, gait and mobility scores were intended to serve as assessments related to fall risk. The registry’s stated hypothesis was that scores would improve after treatment compared with screening scores. The primary endpoint timeframe was baseline and post-test at 4 weeks.
As a continuous measure, the endpoint can be described by a mean and a measure of variability. A mean summarizes the group’s average test time, while the standard deviation describes how spread out individual test times are around that mean. Neither summary alone shows the change from baseline. Interpreting a treatment-period change would require the relevant baseline and post-test results, and an estimate of uncertainty would help indicate how precisely the change was measured.
The registry record does not report the numerical mean, standard deviation, change from baseline, confidence interval, or significance test for this endpoint. It therefore does not permit an assessment of the direction, size, or precision of the primary outcome result.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record identifies a clear primary measure and assessment timeframe: mobility on the Timed Get Up and Go Test at baseline and post-test at 4 weeks. It also records a single-group, open-label design and enrollment of 14. However, it does not report the primary outcome values or an estimated within-participant change. Without those results, the direction and size of any change cannot be determined from the record.
The record lists 2 outcome measures and 2 analyses, but does not provide the numerical results of those analyses. In particular, no primary endpoint mean, standard deviation, treatment-period change, confidence interval, or p-value is reported. The status of the study is terminated, but the reason for termination and its relationship, if any, to the availability of results are not reported in the registry record.
Even if a baseline-to-post-test change were reported, this design would support a description of change in the enrolled group rather than a controlled estimate of the effect of methylphenidate. There was no concurrent control group and no random allocation. A within-group difference cannot separate treatment-associated change from other influences that may affect mobility over time. The open-label design also leaves outcome assessment potentially open to knowledge of treatment, although the record does not say whether any assessor was blinded.
Precision would matter as well as the estimated change. A confidence interval, in general, describes a range of values compatible with the data and the method used to calculate it; a wider interval indicates less precise estimation. No confidence interval is reported for the primary endpoint, so the precision of any change cannot be judged. A p-value, if reported, would address compatibility with a specified statistical null under a model; it would not by itself establish that an observed change was caused by treatment or indicate its practical importance. The registry record does not report a p-value for the primary endpoint.
The safety figures report 0 participants affected by serious adverse events and 0 affected by other adverse events among 14 at risk in the single treatment group. These counts are useful as recorded safety information, but they do not establish that no adverse events of any kind occurred or characterize risks beyond the study participants and observation recorded. Deaths are not reported in the registry record.
Overall, the record describes the study question and treatment schedule, but does not give the primary efficacy results needed to evaluate observed mobility change. Neither a benefit nor a lack of benefit can be concluded from missing endpoint results. The single-group design would also limit causal interpretation even if numerical changes were available.
9. Statistical Methods Explained
The registry describes a continuous primary outcome: time to complete the Timed Get Up and Go Test, summarized as a mean with standard deviation. The outcome is measured at baseline and post-test at 4 weeks. Those are repeated measurements on participants, not measurements from separate treatment and control groups. General statistical interpretation should therefore distinguish the average value at each time from the average within-participant change.
What does a mean describe? In general, a mean is the arithmetic average of observed measurements. It provides a compact description of the group, but it does not show whether participants had similar or widely varying test times. The standard deviation describes the spread of measurements around the mean. It is not the same as the uncertainty in the estimated mean or in a change between assessments. The registry record does not give the endpoint means or standard deviations.
Why does the repeated-measure structure matter? General methods for repeated measurements account for the fact that measurements from the same participant tend to be related. A baseline value and a later value from the same person are not independent observations. A within-participant analysis uses that pairing; an analysis that treats all measurements as unrelated may represent the data structure poorly. The registry record does not identify which statistical test or model was used for the primary analysis, so no particular method should be attributed to the investigators.
What would an estimate of change show? An estimated baseline-to-post-test change would summarize the direction and average size of observed change during the study period. A confidence interval, in general, expresses uncertainty around an estimate under the assumptions of the analysis. Its width is informative about precision, while the estimate itself communicates the direction and scale of change. The record reports neither a change estimate nor a confidence interval. A statistical significance test, if used, would answer a different question from whether a change is large or meaningful; no such result is reported.
Why is the absence of a control group important? This is general design reasoning: a single-group pre-post comparison does not measure what would have happened over the same period without treatment. An observed change may reflect treatment, but may also reflect other time-related influences, changes in care, natural variation, or measurement variation. Randomization and a concurrent comparison group can help address some of these alternative explanations. This trial is registered as single-group, with allocation listed as not applicable, so a treatment-versus-control effect is not available from its design.
How should the planning assumptions be read? The record describes an illustrative sample-size calculation using a two-sided significance level of 0.05, equal allocation between two arms, a normal approximation, and an effect expressed as a fraction of the observed effect. It says that the illustration is not the trial’s own design. The registered trial is single-group, so the two-arm assumptions do not describe its actual allocation. A planning illustration concerns hypothetical future study requirements; it is not an observed result, an estimate of this trial’s precision, or observed power. No sample-size justification for the registered design is reported in the registry record.
The record does not report the statistical test, model specification, covariate adjustment, assumptions checking, treatment of outliers, or method for calculating uncertainty. No analysis method can be inferred from the fact that analyses are listed. Any recalculation would require numerical summary statistics, which are not reported for the primary outcome. There is consequently no recalculated effect to compare with a sponsor analysis, and no discrepancy between such estimates can be assessed.
In general, over-dispersion means that variability is greater than a model assumes. It can make standard errors too small and confidence intervals too narrow if a model fails to account for that extra variation. This concept is not necessarily applicable to every endpoint or analysis; the registry record does not report a model or diagnostics that would establish whether over-dispersion was relevant to this study. For this continuous mobility measure, the available record is insufficient to evaluate distributional assumptions or the adequacy of any fitted model.
The secondary outcome is the Performance Oriented Mobility Assessment, described as a measure of gait and balance. The record lists 2 outcome measures and 2 analyses but does not report the analysis methods or numerical results for either outcome. The statistical plan, including any approach to multiple outcomes or multiple analyses, is not reported in the registry record.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists 2 outcome measures. The primary outcome is the Timed Get Up and Go Test, and the secondary outcome is the Performance Oriented Mobility Assessment, described as a measure of gait and balance. It also lists 2 analyses. The record does not explain whether those analyses correspond to the outcomes, timepoints, or other comparisons, and numerical results are not reported.
When more than one outcome or statistical test is considered, the chance of obtaining at least one apparently unusual result can increase. General methods for addressing multiplicity include prespecifying a primary outcome, adjusting significance thresholds, or interpreting secondary analyses as exploratory. The record identifies a primary outcome but does not report whether a multiplicity adjustment was planned or used. No statistical analysis plan is reported, so the intended hierarchy among analyses beyond the primary and secondary outcome labels is unclear.
Missing-data methods and analysis populations are also not reported in the registry record. The record does not state how many participants completed each assessment, whether any measurements were missing, how missing values were handled, or which participants were included in each analysis. Enrollment alone does not establish the number with an available post-test or the analysis denominator. No assumptions about completion or missingness should be made from the registry information.
In general, missing outcome data can affect both the estimated change and its precision, particularly when missingness is related to participants’ health or mobility. The registry record does not provide enough information to assess whether missing data were present or how they might affect interpretation. The termination status does not, by itself, establish why the study ended or whether outcome data were incomplete.
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 |
|---|---|---|---|
| Single Arm Transdermal Methylphenidate | 0 | 14 | 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 |
|---|---|
| Single Arm Transdermal Methylphenidate | 0 of 14 |
For the single-arm transdermal methylphenidate group, the registry reports 14 participants at risk and 0 participants affected by serious adverse events. It also reports 14 at risk and 0 affected by other adverse events. These are counts of participants recorded as affected in the respective categories; they are not estimates of comparative risk because there was no control group.
The record lists no serious adverse event terms. That should be read as the registry’s reported event information, not as proof that no event of any kind occurred. The figures do not describe event severity beyond the recorded categories, timing, duration, or an independent assessment of causality. The registry record does not report deaths.
Safety counts from a small single-group study are descriptive. Without a comparator, they cannot establish whether the treatment changed the likelihood of an adverse event relative to another course of care. The recorded counts also do not support broad conclusions beyond the participants and follow-up captured in the registry. They should be interpreted alongside the absence of reported endpoint and participant-flow details, rather than as a complete characterization of safety.
12. What the Result Does, and Does Not, Mean
The registry record defines the clinical question, treatment schedule, primary mobility measure, and planned assessment timeframe. It records that the study was terminated and enrolled 14 participants. But it does not report numerical primary outcome results. The central result needed to describe whether test times changed, and how precisely any change was estimated, is therefore unavailable in the registry record.
This absence is not evidence that mobility improved, worsened, or stayed the same. It also does not establish that treatment did or did not reduce falls. The primary measure was a mobility test related to fall-risk assessment; the registry does not report a count of falls as the primary endpoint. Nor does the record report a numerical result for the secondary gait and balance outcome.
The design also sets limits on what a reported change could mean. In a single-group, open-label study, a baseline-to-post-test difference would describe change during the treatment period in the enrolled group. Without a concurrent control and random allocation, it would not isolate the effect of methylphenidate from other potential causes of change. A result from this design would therefore be less conclusive about causation than a well-conducted randomized comparison.
The absence of reported uncertainty estimates matters too. An estimated average change without a confidence interval or another measure of precision could be difficult to distinguish from substantial uncertainty. The registry does not provide these results, so neither the size nor the precision of a possible change can be assessed. Significance testing, if conducted, is also not reported, and a significance result alone would not determine clinical importance or prove causation.
The safety entries record no participants affected in the serious or other adverse-event categories, but those counts are not a complete account of all possible harms. The record does not report deaths, participant completion, or the reason for termination. The most defensible reading is therefore limited: the registry describes a planned evaluation and records the stated safety counts, while leaving the primary outcome findings and key analysis details unavailable.
13. Why This Trial Matters Statistically
This trial raises a clinically relevant statistical question: whether a mobility assessment changes during treatment in a population described as being at fall risk and having probable Alzheimer’s disease. Its primary endpoint is a timed functional task, and the registry also identifies a gait-and-balance measure as secondary. These details make clear what was intended to be measured, even though the numerical findings are not reported.
Statistically, the study illustrates the distinction between measuring change and attributing change. Repeated assessments can show how a group’s outcome differs over time. A single-group design, however, cannot directly show how that change compares with what would have happened without treatment. A comparator and random allocation address a different question from a pre-post description, and the registry record contains no randomized comparison.
The record also illustrates why outcome estimates need measures of uncertainty and transparent analysis details. Means and standard deviations, a change estimate, and its uncertainty would help readers assess the observed data. Information about participant flow, missing measurements, analysis populations, and methods would clarify which observations underpin the results. Those elements are not reported in the registry record for the primary endpoint.
The termination status is relevant context but does not explain itself. The reason for termination and its implications for data completeness are not reported, so neither should be inferred. Likewise, the stated safety counts are descriptive and do not replace comparative safety data. The statistical importance of this record lies in the contrast between a defined outcome question and the limited results available for evaluating it.
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.
Several limitations are specific to the information recorded for this trial. The study was single-group, open-label, and had no randomized comparator. Any within-group change would therefore be difficult to distinguish from changes related to time, measurement, or other influences. The registry does not describe blinded outcome assessment, so whether assessors knew treatment status is not reported.
The study status is terminated, but the reason for termination is not reported in the registry record. The record also does not report participant flow, completion of the treatment period, the number with baseline and post-test measurements, or analysis denominators. Enrollment of 14 does not establish how many participants contributed data to a particular outcome analysis.
Most importantly, numerical results for the primary Timed Get Up and Go Test are not reported. The record contains no mean or standard deviation at either assessment, estimated change, confidence interval, or significance test. Numerical results for the secondary outcome are also not reported. These omissions prevent assessment of effect direction, magnitude, and precision.
The analysis plan, missing-data approach, multiplicity handling, and analysis populations are not reported. The sample-size assumptions describe an illustrative two-arm calculation rather than the registered single-group design, and no design-specific sample-size justification is reported. The registry also does not report deaths. Together, these gaps limit both statistical interpretation and evaluation of how complete the outcome and safety record is.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Exploring the Use of Transdermal Methylphenidate to Reduce Fall Risk in Patients With Dementia. — NCT01825577.
- Registry record last updated 2018-07-24; status last verified 2016-08.
- Results first posted 2016-08-19.
- 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.
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18. Record Summary
NCT01825577 is a phase 4, open-label, single-group study sponsored by St. Louis University. It enrolled 14 elderly patients living in community nursing homes who were identified as fall risks and diagnosed with probable Alzheimer’s disease. The registered intervention was transdermal methylphenidate once daily for 4 weeks, increasing from 10mg for 2 weeks to 15mg for 2 weeks.
The primary outcome was the Timed Get Up and Go Test, measured at baseline and post-test at 4 weeks. The secondary outcome was the Performance Oriented Mobility Assessment. The registry lists the study as terminated, with primary completion in 2015-02; the reason for termination is not reported in the registry record.
Safety counts record 0 participants affected by serious adverse events and 0 affected by other adverse events among 14 at risk. The primary and secondary outcome results, analysis methods, missing-data handling, and analysis populations are not reported. Consequently, the record does not establish the direction or precision of mobility change.