Pharmacokinetics, Metabolism, Efficacy, and Safety Study of Two Testosterone Matrix Transdermal Systems: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Pharmacokinetics, Metabolism, Efficacy, and Safety Study of Two Testosterone Matrix Transdermal Systems (NCT01323140), a na phase 2 study. The primary outcome is Percent of Subjects With Testosterone Levels in the Normal Range..

2. Clinical Question

The trial focused on pharmacokinetics, metabolism, efficacy, and safety for two testosterone matrix transdermal systems, identified in the official title by their surface areas of 28 cm2 and 48 cm2. Its primary outcome was the percentage of participants whose testosterone levels met prespecified criteria. Those criteria concerned average concentration (Cavg) and maximum concentration (Cmax), rather than a comparison between treatment groups.

The primary question is therefore descriptive: how many participants met the specified concentration conditions during the study? Since the study had one group and no control arm, it cannot provide a randomized comparison of the system against another treatment or against no treatment. The registry record lists 40 participants as enrolled, but does not report how many met the primary criteria, the corresponding percentage, or the confidence interval. It consequently does not answer the primary question in the posted results.

The record identifies hypogonadism as the condition and describes testosterone delivery through the skin. It does not include enough detail to characterize participants beyond that condition, or to establish how the results might apply to populations with different characteristics.

3. Trial Design

This was a Phase 2, open-label, single-group study sponsored by Watson Pharmaceuticals. The registry record lists 40 participants as enrolled. Allocation is recorded as NA, masking as none, and the study model as single group. These design features mean there was no randomized or masked comparison group in the record.

The official title describes an investigation of pharmacokinetics, metabolism, efficacy, and safety involving two testosterone matrix transdermal systems, with surface areas of 28 cm2 and 48 cm2. The intervention is named as a testosterone matrix transdermal system. The treatment description says participants went through a lead-in dose-proportionality phase on Days 1-2 and a site-to-site bioavailability phase on Days 2-9, then began efficacy analysis on Day 9 at dose level B, described as a single 48 cm2 system. The available treatment description ends mid-sentence after referring to pharmacokinetic analysis, so further dosing details are not reported in the registry record.

The primary outcome was assessed at Day 29/30. It was defined as the percentage of participants with testosterone levels in the normal range, with additional acceptance criteria for Cmax. The registry record lists one outcome measure and no analyses. It records a study start of 2011-04, primary completion of 2011-07, and status as completed.

Because this was a single-group study, the central statistical description would be the proportion meeting the stated criteria and its uncertainty, rather than a treatment contrast between arms. The registry record does not report the primary result or an analysis description. It also does not report the protocol’s sample-size justification, details of any interim analyses, or a fuller statistical plan. Those omissions limit assessment of how the study was intended to evaluate its objectives.

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

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 Percent of Subjects With Testosterone Levels in the Normal Range., measured over Day 29/30.

The primary endpoint is the percentage of participants whose testosterone levels met the stated criteria at Day 29/30. Testosterone serum concentration was determined at that time, with pharmacokinetic parameters including Cavg and Cmax calculated for efficacy assessment. Cavg refers to average concentration and Cmax to maximum concentration.

The endpoint’s acceptance criteria required at least 75% of participants to have Cavg in the normal range, defined as 300 ng/dL to 1030 ng/dL inclusive. They also required at least 85% of participants to have Cmax at or below 1500 ng/dL, no more than 5% to have Cmax between 1800 and 2500 ng/dL, and no participant to have Cmax at or above 2500 ng/dL. These conditions assess both whether average concentrations fall within the defined range and whether maximum concentrations meet the stated limits.

Using a percentage makes the outcome interpretable as the share of the study group meeting a defined condition. General statistical context: a confidence interval around a percentage describes the uncertainty associated with estimating the corresponding population proportion from a finite group. The registry record identifies a 95% confidence interval as the dispersion measure, but does not report the observed percentage or its interval. The result therefore cannot be compared with the stated acceptance thresholds from the posted results.

The endpoint is a pharmacokinetic measure; it is not itself a direct measure of how participants felt or functioned. The registry record does not report a patient-reported outcome as the primary endpoint, and it does not include the result needed to evaluate the specified concentration criteria.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The outcome is a percentage, which is a binary-outcome summary: each participant either meets a specified condition or does not. General statistical context: a percentage is calculated from the number meeting the condition relative to the number assessed. The registry record gives an enrollment count of 40 and identifies a percentage as the primary outcome unit, but does not report the number meeting the criteria or the analysis denominator. No primary percentage can therefore be reconstructed from the record.

What does the confidence interval describe?

The registry record names a 95% confidence interval as the measure of dispersion for the primary outcome. General statistical context: a confidence interval expresses uncertainty about an estimated quantity, such as a proportion. It is not a range containing a specified share of individual participants, and it does not say that the true value has a particular probability of being inside the interval. Its interpretation depends on the statistical procedure and assumptions used.

No interval endpoints or estimation method are reported for this primary outcome. It is consequently not possible to assess precision, check whether an interval crosses an acceptance threshold, or compare the reported uncertainty across outcomes. A confidence interval cannot be inferred from enrollment alone; it requires the observed outcome information and a chosen method.

How do the concentration criteria work?

The primary endpoint combines conditions about Cavg and Cmax. The registry record defines the Cavg normal range as 300 ng/dL to 1030 ng/dL and sets a minimum percentage of participants expected to be in that range. It separately specifies a minimum percentage with Cmax at or below 1500 ng/dL, a maximum percentage with Cmax between 1800 and 2500 ng/dL, and a requirement for no participant at or above 2500 ng/dL.

These thresholds describe distinct parts of the concentration profile. Cavg concerns average exposure over the assessment period, while Cmax concerns the maximum concentration. General statistical context: when an endpoint includes several conditions, each condition may have a different denominator, definition, or uncertainty unless the protocol explicitly standardizes them. The registry record does not explain how the criteria were combined into a single reported endpoint, or whether each was presented separately. It reports one outcome measure, but no analysis details.

What does a single-group design permit?

The study model is single group, with masking listed as none and allocation as NA. There is no concurrent control arm in the registry record. General statistical context: an estimate from a single group can describe the observed group and, under suitable assumptions, inform an estimate of a broader population proportion. It does not estimate a difference between treatments. Without a comparison group, changes or observed levels cannot be separated statistically from other influences in the way a controlled comparison is designed to do.

The record describes earlier dose-proportionality and site-to-site bioavailability phases, followed by dosing for efficacy analysis at dose level B. It does not give the complete treatment details because the available description is truncated. Nor does it report the statistical methods used to analyze pharmacokinetic measurements or the primary endpoint. A particular modeling approach, adjustment, or estimation method should not be assumed.

How should planning calculations be understood?

General statistical context: a sample-size calculation is a planning exercise that depends on assumptions about the outcome, the effect to detect, the significance level, allocation, and analysis method. The assumptions listed for planning illustrations specify a two-sided significance level of 0.05, equal allocation between two arms, a normal approximation, and effects expressed as fractions of the observed effect. These are planning assumptions, not evidence about the study’s actual design or observed results.

Those assumptions describe a two-arm illustration, whereas this trial is recorded as single group. Any sample-size illustration based on those assumptions should be read as a hypothetical planning calculation and not as a direct reconstruction of the trial’s design. General statistical context: power is a property of a design under specified assumptions; it is not a measure of the probability that a result is true. Sample-size planning illustrations are not observed power. The registry record does not report the trial’s own sample-size justification.

No recalculated primary estimate is available in the registry record. There are no posted counts of participants meeting the criteria from which an unadjusted estimate or interval could be reconstructed. Accordingly, the statistical interpretation is limited to explaining the planned endpoint and the design, not evaluating a numerical efficacy result.

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("Treatment")
n      <- c(38)
events <- c(26)
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  Treatment  38  26  68.4  51.3  82.5  52.5  80.9  52.4  80.9

11. Multiplicity, Missing Data and Analysis Populations

The registry record lists one primary outcome measure, defined as the percentage of participants meeting the testosterone concentration criteria. That outcome contains multiple acceptance conditions involving Cavg and Cmax. Although the conditions are part of the endpoint definition, the record does not explain whether they were assessed as separate tests, how they were combined, or whether any multiplicity adjustment was planned. A multiplicity procedure is not reported in the registry record.

The record lists zero analyses. It does not provide an analysis plan, a list of analysis populations, or a description of how participants with missing measurements would be handled. The number of participants with primary endpoint measurements is not reported in the registry record. Enrollment of 40 participants does not establish that every participant contributed a usable Day 29/30 result.

General statistical context: missing outcome measurements can affect both the denominator of a percentage and the interpretation of the estimate. The impact depends on how much information is missing and why it is missing, as well as the analysis strategy. Complete-case analysis, imputation, or other approaches can lead to different results under different assumptions. No particular approach can be attributed to this study from the registry record.

It is also not reported whether analyses were interim or final, whether there were other analysis populations, or whether endpoint definitions changed. These details matter because the method of handling multiple criteria and unavailable measurements can affect the meaning and uncertainty of an endpoint estimate. Since the primary result itself is not reported, none of these analysis choices can be evaluated against a posted efficacy estimate.

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 non-serious adverse event terms

The treatment group lists 40 participants at risk for serious adverse events and 0 affected. For other adverse events, 40 participants were at risk and 6 were affected. These are counts in the registry record, not estimates of comparative risk, because the study has no separate control group.

The record does not list any top serious adverse event terms. Deaths are recorded as null, which means the number of deaths is not reported in the registry record; it should not be interpreted as a count of zero. The serious adverse event count of 0 is reported separately and should be read as the recorded count for that category.

General statistical context: an observed count describes what was recorded in the study group, while the denominator indicates how many participants were at risk. Counts alone do not describe the full range of event types, timing, severity, or duration. The registry record does not give additional detail for the six participants affected by other adverse events, nor does it identify their event terms.

These figures should be interpreted as descriptive safety reporting for this study group. They do not establish comparative safety, and the limited counts do not characterize all possible adverse events. The registry record does not report a detailed safety analysis or further context about event collection and follow-up.

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

The registry record establishes that a Phase 2, open-label, single-group study was completed and identifies an intended pharmacokinetic primary endpoint at Day 29/30. It also gives the concentration thresholds used to define the endpoint and reports limited adverse-event counts. These details describe what the study set out to assess and some of the information recorded for safety.

What the record does not establish is the observed primary outcome. The percentage meeting the Cavg and Cmax criteria and its 95% confidence interval are not reported in the registry record. Without those values, readers cannot determine whether the stated criteria were met, how far the observed estimate was from a threshold, or how precise the estimate was. A defined endpoint is not itself a result.

The design also limits what can be inferred. Because the study was single group and open label, it does not provide a randomized comparison against another treatment or a control condition. A result from this group, even if reported, would describe the observed concentration profile in the participants assessed; it would not by itself quantify a relative effect against a comparator.

The safety counts likewise have a limited interpretation. The registry record reports 0 serious adverse events among 40 at risk and 6 participants affected by other adverse events among 40 at risk. It does not report the number of deaths, event terms for the non-serious category, or enough detail to characterize the safety experience fully.

General statistical context: estimates from a small or otherwise limited study group are subject to uncertainty, and conclusions depend on the study design, measurement process, and analysis. This record does not provide the primary result or enough methods detail to judge those factors comprehensively. It therefore supports a description of the study and its intended assessment, not a conclusion about whether the intervention met its efficacy criteria or how it compares with alternatives.

14. Why This Trial Matters Statistically

Statistically, this trial is useful as an example of the distinction between a clearly defined endpoint and a reported endpoint result. The registry record specifies a percentage outcome, its Day 29/30 assessment time, a 95% confidence interval, and several concentration-based acceptance criteria. Yet it does not report the observed percentage or interval. Without the result, the endpoint’s intended interpretation cannot be carried through to an estimate or an assessment of precision.

The single-group design is also important. It frames the study as a description of pharmacokinetic and safety outcomes in a treated group, rather than a comparative trial. The study’s open-label status and lack of a comparator make it especially important not to interpret a group result as a treatment contrast. That distinction is relevant even when the primary endpoint is measured objectively as a concentration.

The endpoint itself brings together average and maximum concentrations. Statistically, those criteria ask different questions about exposure and impose distinct thresholds. When multiple conditions define an acceptance framework, clear reporting of each component and how they are combined is valuable for understanding the result. The registry record does not report those component values or explain the analysis that would connect them to the primary percentage.

The record also illustrates why enrollment and completed status are not substitutes for outcome reporting. It lists 40 participants as enrolled and marks the study completed, but does not give the efficacy estimate needed to interpret the primary question. In the absence of the outcome, a confidence interval, and analysis details, the available information supports study-design description more strongly than efficacy 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 main limitation for interpreting this trial from the registry record is the absence of the primary outcome result. Although the endpoint and its assessment time are specified, the percentage meeting the criteria and its 95% confidence interval are not reported. The result cannot be compared with the stated acceptance thresholds, and its precision cannot be assessed.

The study is single group, open label, and has no comparator. It therefore does not provide a randomized treatment contrast. Allocation is recorded as NA, and masking as none. This design limits causal interpretation and makes the findings, if fully reported, descriptive of the assessed group rather than directly comparative.

The available treatment description is incomplete. It identifies lead-in phases and a dose level B using a single 48 cm2 system, but ends mid-sentence. The record does not report the complete dosing and treatment procedures. The official title refers to two system sizes, but the posted treatment description does not provide enough information to explain the full relationship between those systems and the study phases.

Further limitations arise from missing methods details. The registry record does not report the sample-size justification, statistical analysis methods, analysis populations, missing-data handling, multiplicity approach, or interim analyses. It lists one outcome measure and no analyses. The number of participants with primary outcome measurements is not reported.

Safety reporting is also limited: the record gives counts for serious and other adverse events, but does not report the number of deaths, detailed event terms for other adverse events, or broader safety analyses. Together, these gaps restrict the assessment of outcome magnitude, uncertainty, and completeness.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Pharmacokinetics, Metabolism, Efficacy, and Safety Study of Two Testosterone Matrix Transdermal Systems — NCT01323140.
• Registry record last updated 2013-01-01; status last verified 2012-11.
• Results first posted 2013-01-01.
• 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 is a completed Phase 2, open-label, single-group study sponsored by Watson Pharmaceuticals, with 40 participants enrolled. It concerns a testosterone matrix transdermal system in men with hypogonadism and has a primary completion date of 2011-07.

The primary outcome was the percentage of participants meeting specified Cavg and Cmax criteria at Day 29/30. The registry record describes the thresholds and a 95% confidence interval, but does not report the primary result or the analysis methods. It also lists 0 serious adverse events among 40 at risk and 6 participants affected by other adverse events among 40 at risk; the number of deaths is not reported in the registry record.

The single-group design does not provide a comparator-based treatment estimate. The available record supports description of the study design and intended endpoint, but not assessment of the primary outcome against its acceptance criteria.