Effects of Folfirinox and Stereotactic Body Radiation Therapy for Advanced Pancreatic Cancer: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Effects of Folfirinox and Stereotactic Body Radiation Therapy for Advanced Pancreatic Cancer (NCT02128100), a na phase 2 study. The primary outcome is Grade 3 or Higher Adverse Event(s) as a Measure of Safety and Tolerability.

2. Clinical Question

3. Trial Design

This is a phase 2, completed, single-group study sponsored by the University of Louisville. The registry records enrollment of 5 participants with non-resectable pancreatic cancer. Its official title focuses on locally advanced, non-resectable disease and evaluates FOLFIRINOX together with stereotactic body radiation therapy. The design fields identify no random allocation and no masking.

Because there is a single group, the record does not describe a randomized comparison or a concurrent control arm. The study can describe outcomes among participants enrolled in the combined-treatment group, but it cannot by itself separate outcomes associated with FOLFIRINOX from those associated with SBRT, or distinguish treatment-related changes from other explanations. A like-for-like control comparison is not part of the recorded design.

The study start date is 2014-09-19, and the primary completion date is 2021-03-15. Its primary endpoint is the count of participants with grade 3 or higher adverse events, assessed up to 24 months post treatment. The registry also lists overall response rate at 12 months as a secondary outcome. The record reports 3 outcome measures in total, but the other outcome details are not reported in the registry record.

The intervention description names both components, but the arm description is incomplete: it contains some FOLFIRINOX dosing details and an unfinished SBRT description. The complete intervention schedule, eligibility criteria, recruitment details, and rationale for the enrollment target are not reported in the registry record. These omissions limit how fully the study procedures can be reconstructed from 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

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 Grade 3 or Higher Adverse Event(s) as a Measure of Safety and Tolerability, measured over Assessed up to 24 months post treatment.

The primary endpoint is the number of participants with grade 3 or higher adverse events, described as a measure of safety and tolerability. The registry states that toxicity was assessed using CTCAE at specified time points by the treating physician, with assessment up to 24 months post treatment. In practical terms, this endpoint is a participant count: it asks how many people experienced at least one event meeting the stated grade threshold, rather than reporting an average score or a time-to-event estimate.

Safety endpoints of this kind are used to describe the occurrence of treatment-related or other adverse experiences during a defined observation period. The endpoint title identifies the grade threshold, but the primary result itself is not reported in the registry record. It is therefore not possible to determine the observed count for this endpoint from the record.

The listed serious adverse-event count should not be substituted for the primary endpoint result. Seriousness and CTCAE grade are distinct ways of describing adverse events; the registry’s serious-event figures do not establish how many participants had grade 3 or higher events. The record also does not report the event definitions, detailed grading results, attribution to treatment, or the timing of individual events. Those details are needed to interpret the primary safety outcome more fully.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The registry identifies a single-group design and records no statistical analyses. It names a participant-count primary outcome and a response-rate secondary outcome, but does not report an analysis plan, effect estimates, confidence intervals, or p-values. The methods that can be discussed are therefore limited to the design and outcomes recorded, plus general explanations of how such statistics are ordinarily interpreted.

What does a participant count describe?

The primary outcome is a count of participants with grade 3 or higher adverse events. A count reports how many participants meet the outcome definition. It is not, by itself, a complete account of risk: interpretation also depends on the number of participants observed, the event definition, and the observation period. The record gives an assessment period of up to 24 months post treatment, but does not report the primary endpoint count. The separate serious and other adverse-event counts do not fill that gap because they refer to separately recorded categories.

For a binary outcome such as whether a participant experienced an event, a proportion can be calculated when both the event count and the relevant denominator are known. That can make a result easier to compare across groups, but it does not correct for differences in study design or remove uncertainty. No proportion for the primary endpoint can be stated because its event count is not reported. The record’s serious-event and other-event counts have their own denominators, but they are not the primary endpoint.

Why does the single-group design matter?

A single-group study describes outcomes among participants who received the intervention under study. Without a separate comparison group, it does not directly estimate a contrast between treatment strategies. General statistical explanation: a control group helps distinguish an outcome pattern associated with an intervention from patterns that could also arise because of participant characteristics, the course of illness, other care, or chance. This design does not supply that comparison. Even a precise count would remain a description of the observed group rather than a randomized estimate of relative treatment effect.

Random allocation, masking, and the study model are recorded as not applicable, none, and single-group, respectively. The record does not report further details about allocation procedures or a statistical model. Since the outcome analysis is not reported, it is not possible to assess whether any adjustment or other analytical method was used.

What would uncertainty measures add?

General explanation: a confidence interval gives a range of estimates compatible with the observed data under a stated statistical method. Its width communicates precision; a wide interval reflects more uncertainty than a narrow interval. A p-value, when used, measures how compatible the observed data are with a specified null model. Neither measure alone establishes clinical importance or causation. No confidence interval or p-value is reported for the outcomes in the registry record, and the record lists 0 analyses.

With limited enrollment, estimates can be sensitive to individual observations. That is a general property of small datasets, not a claim about an unreported estimate in this study. The count outcome also provides no information about the distribution of event timing or severity beyond the specified grade threshold. More detailed event-level data and a clear analysis plan would be needed to evaluate those features.

How should planning illustrations be understood?

General explanation: a sample-size calculation asks how many participants a future study might need under specified assumptions about the effect to detect, the significance level, allocation, and statistical method. It is a planning exercise, not an observed study result and not a measure of the probability that this completed study detected an effect. The planning assumptions recorded for the illustrations are a two-sided significance level of 0.05, equal allocation between two arms, a normal approximation, and an effect defined as a stated fraction of the observed effect. The record says that time-to-event illustrations use a total event count and other illustrations use participants per arm.

Those assumptions describe a hypothetical comparison and should not be confused with the actual single-group design. The registry does not report that the study used this planning approach, and the planning assumptions are explicitly not the trial’s own design. The sample-size rationale and the study’s original statistical plan are not reported in the registry record. Any sample-size illustrations should therefore be read as planning scenarios, not as observed power, evidence of success, or a reconstruction of the original study design.

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("Folririnox With SBRT")
n      <- c(5)
events <- c(3)
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  Folririnox With SBRT  5  3  60.0  14.7  94.7  23.1  88.2  22.3  88.2

11. Multiplicity, Missing Data and Analysis Populations

The registry reports 3 outcome measures in total and lists the primary safety endpoint plus one secondary outcome: overall response rate assessed by contrast-enhanced CT of the abdomen at 12 months. The details of the other outcome measures are not reported in the registry record. It records 0 analyses, and no statistical analysis plan or multiplicity procedure is reported.

Multiplicity arises when a study evaluates multiple outcomes, time points, or statistical comparisons. General statistical explanation: the more opportunities there are to identify a result as noteworthy, the more important it is to understand which analyses were planned and whether any adjustment or other strategy was specified. The record does not describe such a strategy. It is therefore not possible to assess how multiple outcomes were handled or whether the listed outcomes were analyzed as planned.

Missing-data methods and analysis populations are not reported in the registry record. The record does not state how participants with incomplete assessments, withdrawals, or missing imaging were handled, or which participants were included in any outcome analysis. The reported enrollment of 5 is not enough to establish the denominator used for every endpoint or the completeness of follow-up. These omissions matter because outcome counts and response assessments depend on who was assessed and how missing observations were treated.

The primary endpoint is assessed up to 24 months post treatment, while the listed response outcome is assessed at 12 months. The record does not explain the handling of participants without an assessment at those time points. No assumptions about imputation, censoring, or analysis populations can be made from the information recorded.

12. Safety Results

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

Deaths

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

The registry records 0 serious adverse events among 5 participants at risk in the FOLFIRINOX with SBRT group. It also records other adverse events in 4 participants among 5 at risk and 3 deaths. These are counts from distinct categories in the record; their overlap and timing are not reported. No specific serious adverse-event terms are listed.

These figures should be read as descriptive counts, not as a comparison with another treatment. There is no separate control group in the recorded design. The count of serious events does not establish the result of the primary endpoint, which is the number of participants with grade 3 or higher adverse events. Seriousness and grade are different classifications, and the primary endpoint result is not reported in the registry record.

The record does not state whether the deaths were related to treatment, when they occurred, or whether the participants had other adverse events. It also does not report event-level detail, exposure time, or an explanation of how adverse events were attributed. The counts therefore cannot support conclusions about the causes or timing of these events. They are useful as a limited account of what the registry records, but should not be expanded into a treatment comparison or a complete safety profile.

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

The record establishes that a phase 2, single-group study of FOLFIRINOX with SBRT enrolled 5 participants with non-resectable pancreatic cancer. It identifies a safety-focused primary endpoint and records limited adverse-event counts. It also identifies a secondary response outcome assessed at 12 months. These details describe the study’s intended focus and some posted observations.

The record does not report the result for the primary endpoint: the number of participants with grade 3 or higher adverse events. It also does not report the result for the listed response outcome. The serious adverse-event count, other adverse-event count, and death count cannot substitute for either missing outcome result. No treatment-effect estimate, confidence interval, p-value, or analysis is reported.

General statistical interpretation: an observation in a single group does not show what would have occurred under a different treatment or without the intervention. Without a control group, a difference from an external expectation would not be a randomized comparison. The record therefore does not establish that the combined treatment caused a particular outcome, nor does it allow a relative benefit or harm to be estimated.

The adverse-event counts are descriptive. The record does not report event attribution, timing, overlap, or the details needed to characterize the grade 3 or higher endpoint. The 3 deaths should not be assumed to be treatment-related. Likewise, the absence of recorded serious adverse events does not mean there were no other adverse events: 4 participants are recorded with other adverse events.

Overall, the record documents a small single-group study and some safety counts, but leaves the principal safety result and the secondary response result unreported. It supports neither a firm comparative conclusion nor a full account of safety or response. The missing results, details of follow-up, and analysis methods constrain what can be inferred from the registry record.

14. Why This Trial Matters Statistically

This study illustrates why design and outcome reporting need to be considered together. The registry identifies a clinically focused question about FOLFIRINOX combined with SBRT, but records a single-group design with enrollment of 5 participants. Such a design can describe outcomes among enrolled participants; it does not provide a concurrent comparison that would estimate how outcomes differ under another strategy.

The distinction between endpoint definition and endpoint result is also important. The primary outcome is clearly described as the count of participants with grade 3 or higher adverse events, assessed up to 24 months post treatment. Yet that result is not reported in the registry record. Separate counts for serious and other adverse events are available, but they do not answer the primary endpoint as specified. A well-defined outcome cannot be interpreted empirically without its result and enough information to understand who was assessed.

The record further shows why safety categories should not be collapsed. It reports 0 serious adverse events, 4 participants with other adverse events, and 3 deaths, while leaving the relationship among those categories, timing, and treatment attribution unreported. Presenting those counts separately avoids implying that they describe the same endpoint or establish a causal relationship.

From a statistical perspective, the missing analysis results and uncertainty measures limit assessment of precision. The response outcome is listed without its result, and the registry records 0 analyses. The reported planning assumptions describe illustrations rather than the trial’s own design. Together, these features make the study a useful example of the difference between a registry’s planned question, its recorded design, and the results available for 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 enrollment of 5 participants and the single-group design limit the scope of interpretation. There is no recorded control arm, so the study does not support a randomized comparison. The record does not report the primary endpoint result or the secondary response result, leaving the main outcome questions unanswered in the posted record.

Additional study details are incomplete. The full SBRT schedule is not reported in the registry record, and the arm description is unfinished. Eligibility criteria, participant characteristics, and details of recruitment are not reported in the registry record. The record also does not explain the timing and completeness of follow-up, how missing assessments were handled, or which analysis populations were used.

The adverse-event information is limited to counts. Event terms for serious adverse events are not listed, and attribution, timing, overlap, and the relationship of deaths to treatment are not reported. The primary endpoint’s grade 3 or higher count cannot be inferred from the serious-event count. The record lists 3 outcome measures but provides details for the primary endpoint and one secondary outcome; the remaining outcome details are not reported in the registry record.

No statistical analysis plan, multiplicity approach, confidence intervals, p-values, or outcome analyses are reported. The sample-size rationale is not reported in the registry record. Planning assumptions associated with illustrations should not be mistaken for the study’s original design or an observed-power calculation. These limitations mean that the registry record offers only a partial account of study procedures and results.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Effects of Folfirinox and Stereotactic Body Radiation Therapy for Advanced Pancreatic Cancer — NCT02128100.
• Registry record last updated 2024-03-05; status last verified 2024-02.
• Results first posted 2024-03-05.
• 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

NCT02128100 is a completed phase 2, single-group study sponsored by the University of Louisville. It enrolled 5 participants with non-resectable pancreatic cancer and evaluated FOLFIRINOX with stereotactic body radiation therapy. The recorded study start date is 2014-09-19, and the primary completion date is 2021-03-15.

The primary endpoint is the count of participants with grade 3 or higher adverse events, assessed up to 24 months post treatment. Its result is not reported in the registry record. Overall response rate assessed by contrast-enhanced CT of the abdomen at 12 months is listed as a secondary outcome, but its result is also not reported. The registry records 0 serious adverse events among 5 participants at risk, other adverse events in 4 participants among 5 at risk, and 3 deaths. The design has no separate control group, and the record does not report statistical analyses or a comparative treatment effect.