Study of Mogamulizumab + Docetaxel in Subjects With Non-small Cell Lung Cancer: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Study of Mogamulizumab + Docetaxel in Subjects With Non-small Cell Lung Cancer (NCT02358473), a na phase 1 study. The primary outcome is Number of Subjects Reporting Adverse Events.

2. Clinical Question

In plain terms, the record addresses whether adverse events were reported while participants received this treatment sequence and during the specified follow-up period. It does not set up a like-for-like comparison with another treatment or with no treatment. The listed secondary outcomes include overall response rate, progression-free survival by RECIST 1.1, and overall survival. Those outcomes describe tumor response and time-to-event questions, but the registry record does not provide analyses of them.

3. Trial Design

This was an open-label, multicenter phase one study of mogamulizumab in combination with docetaxel in previously treated subjects with non-small cell lung cancer. The registry lists a single-group model, no masking, and allocation as not applicable. Enrollment was 13 participants, and the study status is completed. Its stated purpose was to evaluate safety.

The treatment sequence began with mogamulizumab alone in a 4-week run-in period. Participants then received up to 6 cycles of mogamulizumab in combination with docetaxel at appropriate intervals. The record says participants could then continue to receive mogamulizumab, at the same dose administered in Cycle 1, once every 3 weeks. It does not give further details about the dose or the criteria for continuing treatment.

Because everyone was in one treatment group, the design does not estimate a randomized treatment contrast. The run-in and combination phases are sequential parts of the described regimen, not independent arms assigned for comparison. Open-label means masking was not used; the record does not describe blinding of participants or study personnel.

The study started in 2015-01 and reached primary completion in 2016-12. The registry record does not report a sample-size justification, a protocol for interim analyses, or details of how participants were selected for analysis. These omissions limit what can be inferred about the planning and conduct beyond the information stated in the record.

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:

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 Number of Subjects Reporting Adverse Events, measured over Screening through 90 days after the last dose of study medication.

The primary outcome is the number of participants reporting adverse events. Its stated observation period runs from screening through 90 days after the last dose of study medication. A participant count is a direct summary of how many people experienced or reported an event under the study’s definitions; it is not a measure of event severity or duration by itself.

Safety outcomes are central in early-phase clinical research. In general, documenting adverse events can help characterize the types of problems observed during treatment and follow-up, and can inform decisions about further investigation. A count is useful because it conveys the number of participants affected, but it does not show how often events occurred in total if a participant could have more than one event. Nor does it, by itself, establish that treatment caused an event.

The record also lists serious adverse events and other adverse events for the treatment group. Those categories should not be treated as interchangeable: serious adverse events are a subset defined by seriousness criteria, while adverse events more broadly can include events that are not serious. The record does not give a detailed endpoint definition, event-attribution criteria, or the method used to collect and code events. Consequently, the counts can be described as posted safety data, but their interpretation depends on details not reported in the registry record.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The statistical information in the registry record is limited. It describes a single-group study, gives participant counts for safety categories, names secondary outcomes, and does not report analyses. The concepts below are general explanations of how such data are usually interpreted; they should not be read as claims about methods used in this study unless those methods are explicitly stated in the record.

What does a participant count convey?

A count records how many participants meet an outcome definition. For adverse events, the participant-level count answers how many people had at least one event in the category, rather than how many separate episodes occurred. A participant with more than one event may still contribute only once to a participant count for a given category. The registry record does not describe its counting rules in detail, so the posted numbers should be read according to the labels given rather than extended to event frequency or recurrence.

Counts are descriptive. They can indicate that events were observed in the enrolled group, but they do not by themselves show whether the regimen caused those events. Clinical events can have multiple possible causes, and causal interpretation generally requires more information about timing, attribution, underlying illness, and comparison with an appropriate alternative. Those details are not reported in the registry record.

Why does the single-group design matter?

A treatment comparison asks how outcomes differ under alternative strategies. In a randomized comparison, allocation can help make groups comparable in expectation, so differences in observed outcomes can be interpreted as evidence about treatment contrasts, subject to uncertainty and study conduct. This study is recorded as single-group with allocation not applicable, so it has no concurrent control arm for that purpose. The mogamulizumab run-in is a treatment phase and does not create an independent control group.

Without a control group, the observed safety counts cannot separate treatment-related events from events that might also occur because of the disease, prior treatment, or other circumstances. The counts remain useful descriptions of what was recorded among participants receiving the study regimen, but they do not estimate a comparative effect.

What does uncertainty mean for these results?

In general, a confidence interval gives a range of values compatible with the data and the assumptions of a statistical method. It is often more informative than a point estimate alone because it conveys precision. A narrow interval indicates greater precision under the method’s assumptions; a wide interval indicates greater uncertainty. No confidence interval for the safety counts is reported in the registry record, so the precision of those counts as estimates of broader event rates cannot be assessed from the posted information.

For small studies, estimates can be sensitive to individual observations. This is a general statistical concern, not a claim that a particular result would change under another analysis. The enrollment of 13 participants means the observed group is limited in size, and the registry record gives no inferential estimate to quantify the uncertainty. The percentages attached to serious adverse event terms are descriptive registry values; they do not substitute for confidence intervals or a comparative analysis.

How should serious and other events be interpreted?

Seriousness and frequency are different characteristics. A serious adverse event is classified according to seriousness criteria; the term does not itself indicate that the event was caused by treatment. A participant-level count of serious events also differs from a list of event terms and percentages. The record does not say whether a participant could contribute to more than one listed term, so the term percentages should not be summed or treated as mutually exclusive categories.

Over-dispersion is a general statistical issue for count outcomes. It occurs when variability in counts is greater than a model assumes, which can make standard errors too small if the model is not suitable. It is relevant when analyzing repeated event counts or rates, but the registry record does not report such a model or event-rate analysis. The posted participant counts alone do not permit an assessment of over-dispersion.

What do the planning assumptions mean?

General planning calculations ask how much information a future study might need to detect a specified effect under stated assumptions. The planning illustration associated with this study is based on the observed effect, uses a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation; the effect to detect is expressed as a fraction of the observed effect. For time-to-event outcomes, such illustrations concern total events; other illustrations concern participants per arm.

These are planning illustrations, not results from the enrolled group and not observed power. They do not describe the design of this single-group trial, which had no two-arm allocation. The registry record does not report the study’s own sample-size justification or the assumptions used to plan enrollment. A planning calculation based on an observed effect also depends on that effect estimate and should not be mistaken for independent confirmation of it.

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("Mogamulizumab + Docetaxel")
n      <- c(13)
events <- c(13)
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  Mogamulizumab + Docetaxel  13  13  100.0  75.3  100.0  77.2  100.0  76.8  99.8

11. Multiplicity, Missing Data and Analysis Populations

The record names overall response rate, progression-free survival by RECIST 1.1, and overall survival as secondary outcomes, and lists 6 outcome measures. However, analyses are not reported in the registry record. It therefore does not provide results, analysis methods, or testing procedures for these outcomes.

Multiplicity arises when several outcomes or comparisons are tested. In general, conducting multiple statistical tests can increase the chance of at least one apparently noteworthy result by chance. Researchers may address this through prespecified outcome hierarchies or adjustments to significance levels, among other approaches. Whether any such approach was used in this study is not reported in the registry record. No adjustment for multiple testing should be assumed.

The record identifies the primary outcome as the number of participants reporting adverse events and gives its timeframe as screening through 90 days after the last dose. It does not report how missing observations, incomplete follow-up, withdrawals, or participants who stopped treatment were handled in the analysis. The population used for any outcome analysis is also not reported in the registry record. Thus, the posted safety counts should not be assumed to represent a particular analysis population or a complete follow-up set beyond what the record states.

These omissions do not establish that no statistical plan or missing-data procedures existed. They mean those details cannot be evaluated from the registry record. Without them, readers cannot determine whether the analyses followed prespecified methods, how uncertainty was handled, or whether all participants contributed to each outcome in the same way.

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: pneumonia (15.4%), sepsis (15.4%), pneumonia bacterial (7.7%), septic shock (7.7%), febrile neutropenia (7.7%).

The mogamulizumab plus docetaxel group lists 6 participants with serious adverse events and 12 with other adverse events, among 13 enrolled participants. These are participant counts, not totals of event episodes. The record lists pneumonia and sepsis at 15.4% each among the serious adverse event terms. Pneumonia bacterial, septic shock, febrile neutropenia, and dehydration are each listed at 7.7%.

Read these figures as descriptive reports from this study group. Serious adverse event terms may overlap within participants; the registry record does not state whether each term occurred in a different person. The percentages should therefore not be summed, and the term list should not be interpreted as a count of distinct participants. The record also does not report event attribution, timing by treatment phase, duration, outcome, or a comparison with another group. Seriousness does not by itself establish that an event was caused by study medication.

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

The record documents a completed, open-label phase one study of mogamulizumab with docetaxel in previously treated non-small cell lung cancer. It provides a treatment sequence and participant-level safety counts, including serious adverse events and other adverse events. These data describe what was recorded in the study group; they are not a controlled estimate of comparative safety.

The design has no separate control group. As a result, the observed events cannot be compared with an alternative treatment group within this study, and the findings cannot isolate the contribution of mogamulizumab from docetaxel or other possible causes. The record also does not report a formal analysis with uncertainty estimates for these counts. The percentages for listed serious-event terms are descriptive and may overlap across participants.

The stated primary outcome concerns the number of participants reporting adverse events during screening through 90 days after the last dose. The posted safety information does not give detailed event definitions, attribution methods, or missing-data handling. Those limits affect how much can be inferred from the counts.

The study lists overall response rate, progression-free survival by RECIST 1.1, and overall survival as secondary outcomes, but their analyses are not reported in the registry record. The record therefore does not establish an effect on tumor response, progression, or survival. Nor does a safety description alone establish whether the regimen is appropriate for any individual. In general, results from a small, uncontrolled study are best understood as observations that may motivate further investigation, not as a definitive treatment comparison.

14. Why This Trial Matters Statistically

This study is statistically informative because it illustrates the distinction between describing outcomes and estimating a comparative effect. Its single-group design can report what happened among participants receiving the treatment sequence, but it cannot directly compare those outcomes with a separate control strategy. That distinction is important when interpreting safety counts, especially when serious event terms are presented with percentages.

The record also makes clear why outcome names and outcome results are not the same thing. Overall response rate, progression-free survival by RECIST 1.1, and overall survival are listed, but analyses are not reported in the registry record. A named outcome indicates what the study intended to assess; it does not establish that a result is available or that an effect has been demonstrated.

General statistical principles help frame the safety summaries. Participant counts are straightforward descriptions, but they do not communicate precision unless accompanied by uncertainty estimates. Event terms may overlap, so percentages should not be combined without information about how they relate at the participant level. A small enrolled group also limits how confidently its descriptive experience can be extended beyond the study.

The registry record’s limitations are themselves relevant: it does not report a sample-size justification, interim-analysis plan, missing-data procedures, or a statistical plan. Those details would help readers assess how outcomes were planned and analyzed. Their absence from the record means they cannot be evaluated, not that no such procedures existed.

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 principal design limitation is the single-group structure. There is no concurrent comparator, and the allocation field is not applicable. Consequently, the safety experience cannot be interpreted as a relative risk or as evidence that one treatment strategy is safer than another. The open-label design also means masking was not used, although the registry record does not describe how event reporting or assessment may have been affected.

Enrollment was 13 participants. This provides a limited basis for characterizing uncommon events or estimating broader event frequencies. The record gives participant counts and selected serious adverse event terms but does not report confidence intervals or other uncertainty estimates. It also does not describe whether listed terms overlap within participants.

The treatment was delivered in stages: a 4-week mogamulizumab run-in, followed by up to 6 cycles of combination treatment, with possible continued mogamulizumab thereafter. The registry record does not provide enough detail to compare safety by treatment phase, dose, exposure duration, or reason for stopping treatment. The exact criteria for continuing treatment are not reported.

Additional limitations include the absence of reported analyses for the listed secondary outcomes and the lack of information about missing data, analysis populations, event attribution, or multiplicity procedures. The record does not report a sample-size justification or interim-analysis plan. These gaps constrain interpretation of both the safety findings and any intended efficacy assessment; they should be treated as information not reported in the registry record rather than evidence that a particular method was or was not used.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Study of Mogamulizumab + Docetaxel in Subjects With Non-small Cell Lung Cancer — NCT02358473.
• Registry record last updated 2024-09-19; status last verified 2024-09.
• Results first posted 2018-11-30.
• 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

NCT02358473 is a completed, open-label, multicenter phase one study of mogamulizumab with docetaxel in previously treated non-small cell lung cancer. It enrolled 13 participants in a single group and was designed to evaluate safety. Treatment began with a 4-week mogamulizumab run-in, followed by up to 6 combination cycles; continued mogamulizumab was also described.

The primary outcome was the number of participants reporting adverse events from screening through 90 days after the last dose. The posted safety counts list 6 participants with serious adverse events and 12 with other adverse events. Secondary outcomes included overall response rate, progression-free survival by RECIST 1.1, and overall survival, but analyses are not reported in the registry record. No randomized comparison is available, and the record does not report detailed statistical methods, missing-data handling, or a sample-size justification.