An Efficacy and Safety Study of Transdermal Therapeutic System (TTS)-Fentanyl in Cancer Participants With Inadequately Controlled Pain by Non-Narcotic Analgesics: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for An Efficacy and Safety Study of Transdermal Therapeutic System (TTS)-Fentanyl in Cancer Participants With Inadequately Controlled Pain by Non-Narcotic Analgesics (NCT01060124), a na phase 4 study. The primary outcome is Percentage of Participants Satisfied With Pain Treatment.

2. Clinical Question

The study’s stated purpose was to evaluate efficacy and safety, as well as participant satisfaction, in cancer participants whose pain was inadequately controlled by non-narcotic analgesics. A secondary outcome is described as the difference in pain intensity before and after administration. The record lists five outcome measures, but does not provide numerical values for the primary endpoint in the information available for this analysis. The clinical question is consequently narrower than whether treatment caused a change in pain: the named primary endpoint concerns reported satisfaction at a specified assessment time. Without a comparator group, any observed satisfaction cannot by itself distinguish a treatment-associated change from other explanations, such as changes over time or other aspects of care.

3. Trial Design

This is a Phase 4, single-group study of TTS-Fentanyl D-trans in cancer participants with inadequately controlled pain. The registry record lists enrollment of 103, allocation as NA, and masking as none. It reports a study start of 2007-04 and a primary completion date of 2009-02, with study status recorded as completed. Janssen Korea, Ltd., Korea is named as the sponsor.

The study does not report a separate control group or a randomized allocation scheme. In a single-group design, participants receive the intervention under study and outcomes are assessed within that group. That structure can describe participants’ recorded outcomes, but it does not provide the direct between-group contrast that a controlled comparison would offer. The record itself flags that this is not a randomized comparison and that the groups are not a like-for-like control comparison.

The primary endpoint is the percentage of participants satisfied with pain treatment at Day 29. Its stated analysis population is Per-Protocol: participants who completed the clinical trial without violating the protocol, among those who participated. The number included in that population is not reported in the registry record. Nor does the record state how many participants were assessed at Day 29 or how incomplete assessments were handled.

The brief study description refers to transdermal treatment and participant satisfaction. It does not report the dosing schedule, treatment duration beyond the named Day 29 endpoint assessment, or detailed eligibility criteria. Those design details are not reported in the registry record and should not be inferred from the study title. The record also does not describe the sample-size justification, interim analyses, or a detailed statistical analysis plan.

4. Participant Flow and Accounting

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

Overall Study

Reasons for not completing this period:

Analysis population for the primary outcome, as defined in the registry: Per-Protocol (PP) analysis population included all participants who completed the clinical trial without violating the protocol among the participant who participated in the clinical trial.

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 Percentage of Participants Satisfied With Pain Treatment, measured over Day 29.

The primary endpoint is the percentage of participants satisfied with pain treatment at Day 29 after application of TTS-Fentanyl D-trans. It is a binary-type outcome summarized as a percentage: each participant’s assessment contributes to whether they are counted as satisfied under the study’s endpoint definition. The registry record describes the assessment but does not give the exact response categories or threshold used to classify satisfaction. Those details are not reported in the registry record.

Satisfaction is used to capture participants’ assessment of their pain treatment, a perspective distinct from a pain-intensity measurement. The registry lists difference in pain intensity before and after administration as a secondary outcome, while satisfaction is the named primary endpoint. These outcomes address related but non-identical questions: a satisfaction measure records an overall evaluation, whereas pain intensity records a rating of pain itself. The registry record does not provide the numerical result for either measure in the information.

The primary endpoint is defined for a Per-Protocol analysis population, consisting of participants who completed the clinical trial without violating the protocol. This is not necessarily the same population as everyone enrolled. The number assessed at Day 29, the satisfaction percentage, and its 95% confidence interval are not reported in the registry record. Without these values, the endpoint’s observed level and precision cannot be described.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

What does a percentage endpoint represent?

General statistical explanation: a percentage for a binary outcome describes the share of participants classified in one category, such as satisfied, among the participants included in the analysis. The numerator is the number classified in that category; the denominator is the number whose outcome is included. Interpreting a percentage therefore depends on knowing both the classification rule and the analysis population. The registry record names a satisfaction endpoint and a Per-Protocol population, but does not state the exact satisfaction classification rule, the number included at Day 29, or the resulting percentage.

What does a confidence interval communicate?

General statistical explanation: a confidence interval expresses uncertainty around an estimate under the statistical procedure used to construct it. It helps readers distinguish a relatively precise estimate from one with substantial uncertainty. The primary endpoint specifies a 95% confidence interval, but neither the percentage nor the interval limits are reported in the registry record. Consequently, the precision of the primary endpoint cannot be evaluated. A confidence interval is not a guarantee that the true value lies within its limits, nor does it correct for bias arising from study design, outcome measurement, or missing observations.

How does the design affect the question?

General statistical explanation: a single-group study estimates outcomes within the participants studied. A randomized controlled comparison can estimate a difference between groups under its design and analysis assumptions. Here, the registry describes a single-group study, allocation as NA, and masking as none. It does not report a randomized comparator. The endpoint can therefore be described as satisfaction observed in the study population, if the result is available, but it cannot be treated as a randomized contrast between treatment and control. Other factors occurring during the study period could also contribute to observed outcomes.

What is the role of the analysis population?

The primary endpoint is specified for the Per-Protocol population. The record defines this as participants who completed the clinical trial without violating the protocol among participants who took part. General statistical explanation: restricting analysis to participants who complete a study and meet protocol requirements can change which participants contribute to an estimate. If inclusion is related to outcome, the resulting estimate may not describe all participants who enrolled. The record does not report the Per-Protocol population size, the number with endpoint data, or how exclusions were determined beyond the stated definition.

How should safety counts be read?

General statistical explanation: event counts and percentages describe what was recorded under the study’s safety definitions. They do not, on their own, demonstrate that an intervention caused an event. A comparison group and details of event collection can help interpret differences, but this record describes one group. It reports serious adverse events for 18 of 103 participants at risk and other adverse events for 25 of 103 participants at risk. The record does not provide further definitions or a comparison that would support causal attribution. The serious-event term percentages are recorded as listed, not as estimates of comparative risk.

What does the planning illustration assume?

The record’s planning assumptions describe an illustration based on the observed effect, with a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. The effect to detect is described as a stated fraction of the observed effect. Such calculations are planning illustrations, not observed power and not evidence that the study was designed using those assumptions. The registry record does not report its own sample-size justification. Because the numerical primary result is not reported in the registry record, the observed effect underlying such an illustration cannot be evaluated from these details. No sample-size conclusion should be read as an observed study result.

General statistical explanation: over-dispersion matters when observed variation in count data exceeds what a chosen model expects. A model that does not account for this can understate uncertainty. The registry record does not describe a count-data model or report an analysis that requires an over-dispersion assessment; this concept is therefore general rather than a characterization of the study’s analysis.

10. Exact Estimates for Each Arm

No two-arm comparison is shown for this trial, but each arm's result can still be summarized with exact interval estimates computed from the posted counts. The Clopper-Pearson interval is exact and conservative; the Wilson interval is a common closer-to-nominal alternative; the Bayesian credible interval uses a uniform Beta(1, 1) prior. For small groups the three can differ noticeably.

R code and output

arm    <- c("Transdermal Therapeutic System (TTS)-Fentanyl D-tr")
n      <- c(64)
events <- c(53)
for (i in seq_along(n)) {
  ci <- binom.test(events[i], n[i])$conf.int          # Clopper-Pearson exact interval
  cr <- qbeta(c(0.025, 0.975), 1 + events[i], 1 + n[i] - events[i])   # Bayesian, uniform Beta(1,1) prior
  wi <- (function(x, n) { p <- x / n; z <- qnorm(0.975); d <- 1 + z^2 / n
    c((p + z^2 / (2 * n) - z * sqrt(p * (1 - p) / n + z^2 / (4 * n^2))) / d, (p + z^2 / (2 * n) + z * sqrt(p * (1 - p) / n + z^2 / (4 * n^2))) / d) })(events[i], n[i])
  cat(sprintf("ARM|%s|%d|%d|%.1f|%.1f|%.1f|%.1f|%.1f|%.1f|%.1f\n", arm[i], n[i], events[i], 100 * events[i] / n[i],
              100 * ci[1], 100 * ci[2], 100 * wi[1], 100 * wi[2], 100 * cr[1], 100 * cr[2]))
}

ARM  Transdermal Therapeutic System (TTS)-Fentanyl D-tr  64  53  82.8  71.3  91.1  71.8  90.1  71.7  90.1

11. Multiplicity, Missing Data and Analysis Populations

The registry record lists five outcome measures, identifies satisfaction at Day 29 as the primary endpoint, and reports zero analyses. It does not provide a detailed statistical analysis plan or say how multiple outcomes were handled. When several outcomes are examined, general statistical practice recognizes that the chance of at least one apparently notable finding may increase if no multiplicity strategy is used. The record does not report whether any adjustment, hierarchy, or other approach was planned, so none should be assumed.

The primary endpoint is defined for a Per-Protocol population, but the size of that population and the number with Day 29 assessments are not reported in the registry record. The record also does not describe the amount or pattern of missing outcome data, or the method used to handle missing observations. These omissions prevent assessment of how incomplete follow-up might affect the endpoint estimate. They do not establish that data were missing or that a particular method was used.

The registry record does not report interim analyses, stopping rules, or a sample-size justification. These items should not be inferred from the study’s Phase 4 designation or its completed status. Without a detailed analysis plan and numerical outcome results, it is not possible to determine whether analyses followed prespecified procedures or how uncertainty across the listed outcomes was addressed.

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 5% reporting threshold.

Most frequent serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: death (1.9%), pyrexia (1.9%), disease progression (1.0%), abdominal distention (1.0%), abdominal pain (1.0%).

The record reports one study group, with 103 participants at risk for both serious and other adverse events. It lists 18 participants affected for serious adverse events and 25 affected for other adverse events. These are counts within the study group, not comparisons against an untreated or randomized control group. They describe recorded events and do not establish that TTS-Fentanyl D-trans caused them.

The serious-event terms listed include Death at 1.9%, Pyrexia at 1.9%, Disease Progression at 1.0%, Abdominal distention at 1.0%, Abdominal Pain at 1.0%, and Diarrhoea at 1.0%. The record’s deaths field is null. A null value is not a reported zero, and it does not resolve the meaning of the Death term in the serious-event list. The registry record does not explain that relationship.

General statistical explanation: adverse-event term percentages and participant-level counts can refer to different summaries, and the same participant may experience more than one event. The record does not provide enough detail to reconcile individual terms with the overall serious-event count. Event definitions, attribution assessments, and the collection procedures are not reported in the registry record. The figures should therefore be read as descriptive safety reporting, not as estimates of comparative or causal risk.

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

The record establishes that a completed Phase 4, single-group study evaluated TTS-Fentanyl D-trans in cancer participants with pain inadequately controlled by non-narcotic analgesics. It identifies satisfaction with pain treatment at Day 29 as the primary endpoint and specifies a Per-Protocol analysis population. It also records an enrollment of 103 and lists serious and other adverse-event counts for the study group.

It does not report the numerical satisfaction percentage or its 95% confidence interval. As a result, the recorded primary endpoint’s value and precision are not reported in the registry record. The registry lists a secondary outcome concerning pain intensity before and after treatment, but its numerical result is not reported in the registry record either. These missing numerical results prevent a data-based description of the direction or size of the observed endpoint findings.

Even if a within-group endpoint estimate were available, the single-group design would not provide a randomized treatment-versus-control contrast. General statistical explanation: changes observed after treatment may have several explanations, and a concurrent control group can help separate treatment-associated differences from changes over time or other influences. This record does not provide such a comparison. It therefore cannot quantify the outcome that would have occurred under a different treatment condition.

The safety counts likewise describe events in the study group. They do not establish causation or comparative safety. The registry’s null entry for deaths should not be read as zero deaths, particularly because Death also appears among the listed serious-event terms. The relationship between these entries is not reported in the registry record.

These limitations do not erase the descriptive value of the study question or the safety information recorded. They define what can and cannot be concluded from the registry record: design and endpoint details are available, while the primary numerical result, its uncertainty, and a controlled comparison are not.

14. Why This Trial Matters Statistically

This trial is statistically instructive because its primary question is clear but its numerical primary result is not reported in the registry record. The endpoint is participant satisfaction with pain treatment at Day 29, summarized as a percentage with a specified 95% confidence interval. Naming the estimand and its intended population is useful, but interpretation also requires the estimate, its uncertainty, the denominator, and the endpoint classification rule. Those numerical and operational details are not reported in the registry record.

The single-group design also illustrates the distinction between describing an outcome and estimating a comparative effect. A single-group percentage can characterize participants’ recorded outcomes, while a controlled design addresses how outcomes differ under alternative conditions. Because the record lists no separate comparator and no randomized allocation, the study cannot supply that controlled contrast. This matters when translating an observed endpoint into a claim about what the intervention caused.

The specified Per-Protocol population highlights another important issue: results depend on who is included in an analysis. A definition based on completing the trial without protocol violations may exclude some participants, but the record does not report the population size or how many contributed Day 29 data. Without those details, readers cannot assess the relationship between enrollment and the endpoint analysis.

Finally, safety counts need context. Counts and event-term percentages can describe recorded events, but a single-group study cannot make a direct comparative safety estimate. The null deaths entry alongside a listed Death term also demonstrates why registry fields should be interpreted according to their precise recorded meaning rather than converted into assumptions. Together, these features make the record a useful example of how design, population, endpoint reporting, and safety summaries constrain statistical interpretation.

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 consequential limitation for interpreting the primary endpoint is that its numerical result and 95% confidence interval are not reported in the registry record. The registry identifies the assessment time as Day 29 and the analysis population as Per-Protocol, but does not report the number assessed, the satisfaction percentage, or the interval limits. The exact rule used to classify participants as satisfied is also not reported.

The design is single-group, with allocation listed as NA and masking as none. No like-for-like control group is reported, so the record does not support a randomized between-group comparison. The registry’s own design note cautions against treating arms as a comparable control contrast. Detailed intervention administration, eligibility criteria, and the handling of concomitant care are not reported in the registry record.

The record lists five outcome measures and zero analyses, but does not give a detailed analysis plan, multiplicity strategy, interim-analysis information, or sample-size justification. It does not report the Per-Protocol population size, missing-data amounts, or missing-data methods. The safety information is also limited: the record provides group counts and selected serious-event terms, but not detailed event definitions or an explanation of the null deaths entry alongside Death in the serious-event list. These gaps limit assessment of precision, selection, analysis choices, and causal interpretation.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: An Efficacy and Safety Study of Transdermal Therapeutic System (TTS)-Fentanyl in Cancer Participants With Inadequately Controlled Pain by Non-Narcotic Analgesics — NCT01060124.
• Registry record last updated 2014-04-29; status last verified 2014-04.
• Results first posted 2013-04-26.
• 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

This completed Phase 4 study evaluated TTS-Fentanyl D-trans in cancer participants with pain inadequately controlled by non-narcotic analgesics. It was a single-group study with allocation listed as NA and masking as none. Enrollment was 103; the study start was 2007-04 and primary completion was 2009-02.

The primary endpoint was the percentage of participants satisfied with pain treatment at Day 29, assessed in a Per-Protocol population. Its numerical estimate and 95% confidence interval are not reported in the registry record. A secondary outcome concerned pain-intensity difference before and after treatment. The record lists five outcome measures and zero analyses.

For the study group, the record reports 18 participants affected by serious adverse events and 25 affected by other adverse events, each among 103 at risk. Death and other serious-event terms are listed, while the deaths field is null. The single-group design and missing numerical endpoint result limit comparative and causal interpretation.