Observational Study With Neupro® to Evaluate the Patient´s Perception of Pain Associated With Parkinson´s Disease: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Observational Study With Neupro® to Evaluate the Patient´s Perception of Pain Associated With Parkinson´s Disease (NCT01606670), a registered phase not stated study. The primary outcome is Change From Baseline to Visit 2 in the Sum Score Calculated From the 4 Items of the Affective Dimension of the Pain Description List (Schmerzbeschreibungsliste SBL, Question 10) of the German Pain Questionnaire.

2. Clinical Question

In statistical terms, this is a question about change in a continuous score during routine treatment, not a randomized estimate of a treatment effect against a comparator. The endpoint captures the applicability of affective pain descriptors, rather than pain in every possible sense. The record also lists changes in UPDRS Part II, UPDRS Part III, their combined score, and the PDQ-8 total score as secondary outcomes, but their results are not reported in the registry record.

3. Trial Design

The registry record describes a multicenter, non-interventional study of routine Neupro® treatment in people with idiopathic Parkinson's disease. Its stated objective is to evaluate patients' perception of pain among those receiving Neupro® for the first time. Enrollment was 93. The record lists the study as completed, with a start date of 2012-05 and primary completion date of 2014-07.

The treatment group is described as receiving Neupro® according to routine practice and the approved EU label applicable in Germany. Dose options listed in the record range from 2 to 16 mg/24 h. The record does not describe a comparator group, random assignment, or a treatment allocation method. It therefore does not present a randomized head-to-head comparison. The duration at maintenance dose before the primary follow-up is specified as at least 4 weeks.

The primary measurement is the change from baseline to Visit 2 in the sum of four affective items from the Pain Description List, part of the validated German Pain Questionnaire. The primary population is the Full Analysis Set. Its definition requires at least one application of the patch and valid primary-variable data at baseline and a subsequent routine visit. This definition means the primary population is not simply all enrolled patients.

There are five outcome measures listed, including the primary pain measure and secondary measures of Parkinson's disease symptoms and quality of life. The registry record reports zero analyses. It does not report a statistical analysis plan, a sample-size justification for the study, details of the timing or conduct of interim analyses, or results for the listed outcomes. Those omissions limit what can be concluded about how the planned study questions were addressed.

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: Participant Flow refers to the Enrolled Set (ES).

Overall Study

Reasons for not completing this period:

Analysis population for the primary outcome, as defined in the registry: Full Analysis Set (FAS). The FAS includes all enrolled patients who had applied the rotigotine transdermal patch at least once and for whom valid data for the primary effectiveness variable were available from Baseline and a subsequent routine visit.

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.

Population: Baseline Characteristics refer to the Safety Set, including all enrolled patients who had applied the rotigotine transdermal patch at least once.

6. Primary Endpoint

The registry lists the primary outcome as Change From Baseline to Visit 2 in the Sum Score Calculated From the 4 Items of the Affective Dimension of the Pain Description List (Schmerzbeschreibungsliste SBL, Question 10) of the German Pain Questionnaire, measured over From Baseline (Day 0) to Visit 2 (after at least 4 weeks on a maintenance dose of Neupro®).

The primary endpoint is the change from baseline to Visit 2 in the sum score for the four affective items of the Pain Description List, question 10, within the German Pain Questionnaire. The instrument's twelve adjectives are rated from 0, meaning not applicable, to 3, meaning exactly applicable. Eight items describe sensory aspects of pain; the four affective items produce a sum score from 0 to 12. The registry record notes that values above 8 can be considered noticeable.

This endpoint focuses on the affective description of pain: how applicable the listed affective descriptors are to a participant's experience. It is distinct from the sensory descriptors, and it is a score rather than a direct count of pain episodes or a measure of all aspects of Parkinson's disease. A change score compares the measured value at follow-up with the baseline value. The record specifies Visit 2 after at least 4 weeks on a maintenance dose.

General: a continuous score can retain gradations that would be lost if responses were reduced to categories. The mean change can describe the average direction and size of change in the analyzed population, while variability describes how dispersed individual scores are. Neither type of summary alone explains why scores changed. The registry record does not report the mean change, standard deviation, or other primary endpoint results, so the observed pattern and its precision are not available in the record.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The primary endpoint is continuous: it is a sum score, and the registry record specifies the mean as the summary parameter and the standard deviation as the dispersion measure. Those choices describe different features of the score distribution. The mean describes its central tendency; the standard deviation describes how widely individual measurements vary around that mean. Neither measure is reported for the primary outcome in the registry record.

General: a change score is formed by comparing a participant's follow-up measurement with that participant's baseline measurement. A mean change then summarizes these within-person changes across the analyzed population. Its interpretation depends on the scale's meaning and on the direction used to define change. Here, the score ranges from 0 to 12, with higher item ratings meaning that an adjective is more applicable. But because no observed change is reported, the direction and size of any average shift cannot be described.

General: a standard deviation is not the same as the standard error of a mean. The standard deviation reflects variation among observations; the standard error reflects uncertainty in an estimated mean and depends on the amount of information available. A confidence interval commonly communicates uncertainty about an estimate. The registry record does not report a confidence interval, standard error, or test result for the primary endpoint, so inferential precision cannot be reconstructed from the recorded information.

The study's primary endpoint population is the Full Analysis Set. It includes enrolled patients who applied the patch at least once and had valid primary-variable measurements at baseline and at a later routine visit. This definition makes availability of paired measurements part of inclusion in the primary analysis population. The record does not report the number meeting this definition, the number excluded from it, or how missing data were addressed. It is therefore not possible to assess how much the analyzed population differed from the enrolled population.

The record lists a planning illustration based on the observed effect, with a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. It describes the effect to detect as a stated fraction of the observed effect. These are assumptions for a planning calculation, not a description of the study's non-interventional design. The record does not report the resulting participant requirements or the observed effect value in the registry record for this interpretation. A planning illustration is not observed power and should not be read as evidence about the study's ability to detect its actual outcome.

General: over-dispersion means that observed variability is greater than a model or method expects. When it is present, a method that understates variability can produce uncertainty estimates that are too narrow. The registry record does not report a model-based analysis or information about over-dispersion for this continuous endpoint; this concept is relevant as a general consideration, not as a reported feature of this study.

The record does not state whether the primary analysis used an adjusted or unadjusted model, whether assumptions were checked, or whether confidence intervals were calculated. It also does not provide a protocol-based statistical plan or a rationale for the selected analysis method. These details are not reported in the registry record, so they should not be inferred from the endpoint's designation as a mean with standard deviation.

Five outcome measures are listed. The primary measure concerns affective pain descriptors, while the secondary outcomes concern UPDRS Part II, UPDRS Part III, their combined score, and the PDQ-8 total score. General: when several outcomes are examined, multiplicity can arise because repeated testing creates more opportunities for apparently notable findings. The registry record does not report a multiplicity adjustment or a hierarchy for interpreting the outcomes. It also reports zero analyses, so no outcome-specific test statistics can be discussed.

The study has no control comparison described. General: a within-group change can describe what happened over time in the observed group, but it cannot by itself distinguish treatment-related change from changes associated with time, other care, measurement variation, or participant characteristics. These are general cautions for uncontrolled before-and-after observations; the registry record does not establish that any such factor caused a change in this study.

10. Confidence Intervals for Each Arm

No two-arm comparison is shown for this trial, but each arm's mean can be given with a t-based 95% confidence interval computed from the posted mean, spread and number of participants.

R code and output

arm <- c("Neupro® Treatment")
n   <- c(70)
m   <- c(-1.3)
sp  <- c(2.8)
sd  <- sp      # posted standard deviations
for (i in seq_along(n)) {
  se <- sd[i] / sqrt(n[i]); h <- qt(0.975, n[i] - 1) * se
  cat(sprintf("CARM|%s|%d|%.3f|%.3f|%.3f|%.3f\n", arm[i], n[i], m[i], sd[i], m[i] - h, m[i] + h))
}

CARM  Neupro® Treatment  70  -1.300  2.800  -1.968  -0.632

11. Multiplicity, Missing Data and Analysis Populations

The registry record lists five outcome measures: the primary affective pain score and secondary scores for UPDRS Part II, UPDRS Part III, their combined score, and the PDQ-8 total score. It does not report outcome results or analyses. It also does not report whether testing across the outcomes was adjusted for multiplicity, how the primary and secondary outcomes were prioritized in interpretation, or whether a prespecified testing sequence was used. Those details are not reported in the registry record.

General: multiplicity matters when several outcomes or comparisons are tested because the chance of at least one apparently unusual result can increase as testing expands. Adjustment methods can address some forms of this issue, but they require a defined analysis plan. No adjustment method or plan is described in the registry record, so no claim about control of false-positive findings can be made.

The Full Analysis Set includes enrolled patients who used the patch at least once and had valid data for the primary variable at baseline and a subsequent routine visit. The record does not state how many enrolled patients met that definition. It does not report the extent or pattern of missing outcome data, the reasons for missingness, or an imputation or other missing-data method. It is therefore not possible to evaluate whether missing data could have affected the primary estimate.

General: missingness can affect interpretation when the availability of measurements is related to participants' outcomes or characteristics. Whether that concern applies depends on information about who lacked measurements and why. Such information is not reported in the registry record. The stated Full Analysis Set definition should therefore be read as a population definition, not as evidence that missing data were absent or had no effect.

12. Safety Results

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

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

For the Neupro® Treatment group, the record lists 93 as at risk for serious adverse events and 0 as affected. For other adverse events, it lists 93 at risk and 10 affected. These figures describe the registry's affected counts; they are not a complete description of the nature, timing, severity, or duration of individual events. No serious adverse event terms are listed among the top serious terms.

The deaths field is not populated with a numeric result. Deaths are therefore not reported in the registry record; that should not be interpreted as a confirmed zero. The figures also do not establish how events were collected or assessed, since those details are not reported in the registry record.

General: safety counts are descriptive. They depend on the definitions, observation period, and reporting procedures used, and an affected count is not a measure of causality. With no comparator group described, these data do not provide a comparative estimate of safety. They should be read alongside the study's non-interventional design and the limits of the registry information.

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

This record identifies a study of pain perception during routine Neupro® treatment in people with idiopathic Parkinson's disease. Its primary endpoint is the change in an affective pain-description score from baseline to Visit 2, after at least 4 weeks on a maintenance dose. The endpoint uses four items scored as part of the German Pain Questionnaire, and the record describes its possible sum-score range and the meaning of higher item ratings.

What the record does not show is the primary endpoint result. There is no mean change, standard deviation, confidence interval, or analysis result reported. The listed secondary outcomes likewise have no reported results in the registry record. It is not possible to say from this record whether pain scores changed, by how much, or with what precision. No result should be inferred from the study's completion status or its stated objective.

The design also limits causal interpretation. The record describes non-interventional routine treatment and no comparator group. A change from baseline would describe change over time in the analyzed group; without a comparison group, it would not by itself establish that treatment caused the change. This is a general interpretation of uncontrolled observations, not a conclusion about any unreported result from this study.

The safety listing reports 0 affected for serious adverse events and 10 affected for other adverse events in the group, with deaths not reported in the registry record. These are descriptive registry figures, not a comparative assessment. The record does not give enough detail to characterize event timing or circumstances.

Overall, the registry record is useful for identifying the population, treatment context, endpoint, and intended analysis population. It does not provide the outcome evidence needed to characterize the observed pain-score change or to compare treatment with an alternative. Missing analysis and data-handling details further limit assessment of statistical precision and potential bias.

14. Why This Trial Matters Statistically

This study is statistically notable for asking a patient-centered question through a defined continuous score rather than relying only on a broad clinical measure. The primary measure focuses on the affective dimension of pain, and its item scoring gives readers a stated interpretation of what the scale values represent. Distinguishing the affective items from the sensory items matters because they describe different dimensions of pain.

The registry record also makes clear that the study concerns routine, non-interventional treatment. This context affects the question the design can answer. A before-and-after description can characterize change among participants with the required measurements, but the absence of a comparator limits attribution of any change to treatment. General: understanding the design is as important as understanding the endpoint when interpreting a clinical estimate.

The definition of the Full Analysis Set is another important statistical feature. Requiring valid baseline and follow-up measurements defines which patients contribute to the primary outcome analysis. Without the number meeting that definition or information about missing data, readers cannot assess how broadly an eventual analysis would represent all enrolled patients.

Finally, the registry record lists multiple outcomes but reports zero analyses and no outcome results. This makes transparency about reporting especially important: a stated endpoint and a completed study are not substitutes for a numeric result and its uncertainty. The record supports careful description of what was planned and measured, while leaving the principal empirical question unanswered in the registry record.

15. 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 most direct limitation is that no primary endpoint estimate is reported in the registry record. The record does not provide the mean change or its standard deviation, and it gives no confidence interval or test result. Results for the secondary outcomes are also not reported. Although the study is marked completed, completion alone does not indicate what the measurements showed.

The study is described as non-interventional, and no control group is listed. The record does not state an allocation method. This design description does not support a randomized treatment comparison. Any within-group change, if reported elsewhere, would need to be interpreted as an observation over time rather than as a controlled treatment effect.

The Full Analysis Set requires valid primary outcome data at baseline and a subsequent routine visit after at least one patch application. The number meeting this definition, the amount of missing data, and the handling of missing observations are not reported in the registry record. These omissions prevent assessment of how data availability shaped the analyzed population.

The registry record does not report the statistical analysis plan, details of any model or adjustment, or a multiplicity strategy across the listed outcomes. It does not provide the primary result's precision or enough information to assess the assumptions behind an analysis. The deaths field is also not populated with a numeric result. These gaps mean that outcome interpretation and a fuller evaluation of safety are limited to the descriptive information recorded.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Observational Study With Neupro® to Evaluate the Patient´s Perception of Pain Associated With Parkinson´s Disease — NCT01606670.
• Registry record last updated 2018-04-04; status last verified 2018-03.
• Results first posted 2015-04-29.
• 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.

19. Record Summary

NCT01606670 is a completed, multicenter, non-interventional study of routine Neupro® treatment in people with idiopathic Parkinson's disease. Enrollment was 93; the recorded start and primary completion dates are 2012-05 and 2014-07. The primary endpoint is change from baseline to Visit 2 in the sum of four affective items from the German Pain Questionnaire's Pain Description List. Visit 2 was after at least 4 weeks on a maintenance dose.

The primary analysis population is the Full Analysis Set, defined by patch use and valid baseline and subsequent routine-visit data for the primary variable. Four secondary outcomes are listed. The registry record reports zero analyses and no outcome results, so the primary change and its statistical precision are not reported. The adverse-event listing records 0 affected for serious events and 10 affected for other events; deaths are not reported in the registry record.