Chemotherapy, Stereotactic Body Radiation Therapy & Nelfinavir Mesylate in Locally Advanced Pancreatic Cancer: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Chemotherapy, Stereotactic Body Radiation Therapy & Nelfinavir Mesylate in Locally Advanced Pancreatic Cancer (NCT01959672), a na phase 2 study. The primary outcome is Number of Participants With Progressive Disease,.

2. Clinical Question

3. Trial Design

This is a phase II, single-group study sponsored by the University of Nebraska. The registry lists an enrollment of 11 and a completed status. The recorded start date is 2013-09-06, and the primary completion date is 2018-04-01. Allocation is listed as not applicable, and masking is listed as none. The design therefore does not include a randomized, masked comparison group in the registry record.

The treatment group is labeled as receiving chemotherapy, oregovomab, SBRT, and surgery. The regimen description specifies gemcitabine hydrochloride, leucovorin calcium, and fluorouracil on days 1 and 8, with treatment repeated every 3 weeks for 7 courses. Oregovomab is described for patients with CA125 level >= 10, given on day 15. The available treatment description ends partway through the regimen, so the complete sequence and details of SBRT, nelfinavir, and surgery are not reported in the registry record.

The official title refers to chemotherapy with and without immunotherapy, but the listed study model is single-group and the record does not describe separate groups for those strategies. It would therefore be inappropriate to interpret the study as a randomized test of oregovomab versus no oregovomab. The registry lists secondary outcomes including distant failure-free survival, overall survival, surgical complete resection (negative margin) rate, and tumor response rate evaluated on the pathology specimen. Results for these outcomes are not reported in the registry record. A sample-size justification, interim-analysis plan, and detailed statistical analysis plan are also not reported in the registry 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:

Analysis population for the primary outcome, as defined in the registry: Analysis of the primary endpoint (progression at 4 months) demonstrated that of the ten patients who remained alive, two (20%) had progressed at 4 months.

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 Participants With Progressive Disease,, measured over Up to 4 months.

The primary endpoint is the number of participants with progressive disease through 4 months. The registry defines progression as at least a 25% increase in the longest diameter of a lesion, compared with the longest diameter recorded since treatment started. This is a binary outcome: a participant either meets the stated progression definition within the endpoint period or does not. The endpoint description calls for an exact one-sided 90% confidence interval around the progressive-disease rate.

The registry reports that two of the ten patients who remained alive had progressed at 4 months, with the rate stated as 20%. The actual confidence interval is not reported in the registry record. The outcome description does not explain how participants who died, lacked an assessment, or otherwise could not be evaluated were handled; those details are not reported in the registry record.

In general, a progression endpoint can describe whether disease worsened according to a prespecified measurement rule over a defined period. A diameter-based threshold makes the event definition explicit, while the fixed timeframe specifies when the event is assessed. This endpoint alone does not describe every aspect of disease course, treatment experience, or survival, and a single-group result does not establish how the rate compares with another treatment strategy.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The primary outcome is a binary participant-level event: progressive disease within the stated period. The registry defines the event by a threshold increase in the longest diameter of a lesion, using the longest diameter recorded since treatment started as the reference. It describes the timeframe as up to 4 months. The result is reported as two of the ten patients who remained alive, with a rate of 20%.

What does the confidence interval describe?

The endpoint description says an exact one-sided 90% confidence interval will be constructed around the progressive-disease rate. In general, a confidence interval communicates uncertainty about an estimate under a specified statistical procedure. A one-sided interval focuses on a bound in one direction rather than reporting both a lower and an upper bound as a two-sided interval does. The registry does not report the interval limits or explain which bound was presented. The observed rate and the interval are distinct pieces of information: the rate summarizes the observed group, while the interval is intended to characterize uncertainty under the chosen method.

In general, exact methods for a binary rate use the event count and the number of participants included in the analysis, rather than relying on an approximation that may perform poorly when counts are small. That general rationale does not establish which participants were included in the registry’s analysis. The endpoint text identifies ten patients who remained alive, but the treatment of deaths, missing assessments, or other unavailable outcome data is not reported in the registry record. The actual interval result is also not reported in the registry record.

Why does the study design matter?

The registry lists a single-group model, allocation as not applicable, and no masking. A single-group design can describe outcomes observed among participants receiving a treatment approach, but it does not directly estimate a between-group contrast. In general, a comparison between groups can help distinguish the outcome associated with one strategy from outcomes that might occur under another strategy. Without a listed comparator, the primary result is not a randomized estimate of the effect of the combined treatment approach, and it cannot separate the effects of chemotherapy, oregovomab, SBRT, nelfinavir, or surgery.

The official title refers to chemotherapy with and without immunotherapy. However, the registry lists only one treatment group, and the available record does not set out a randomized comparison of those approaches. The treatment description says oregovomab is given to patients with CA125 level >= 10. This eligibility detail is not a substitute for a comparator arm, and the registry does not report a separate estimate for patients who did or did not receive oregovomab.

What is not available about the analysis?

The registry describes an exact one-sided 90% interval but does not report the resulting interval. It does not report a hypothesis test for the primary result, covariate adjustment, a detailed analysis population, missing-data rules, or an interim-analysis plan. A sample-size justification is not reported in the registry record. The record lists zero outcome analyses, so analysis-level details and results beyond the primary endpoint statement are not available.

In general, a statistical analysis plan clarifies which outcomes and populations will be analyzed, how incomplete observations will be handled, and which methods will be used. Those decisions matter because the same observed count can be interpreted differently depending on the analysis population and endpoint rules. No such details should be inferred when they are not reported.

Any sample-size illustrations associated with the posted results are planning illustrations based on an observed effect, not the trial’s own design rationale and not observed power. In general, power calculations describe the ability of a planned design to detect an effect under stated assumptions; they do not measure whether the completed study has proved an effect. The registry’s sample-size assumptions describe a two-sided significance level of 0.05, equal allocation between two arms, a normal approximation, and effects defined as fractions of the observed effect. Those are assumptions for the illustration, not evidence that this single-group trial used a two-arm design or that its original sample size was justified in that way.

In general, over-dispersion is a concern when observed variability is greater than a statistical model expects, which can make uncertainty estimates too narrow if the model does not account for it. The posted primary endpoint is a participant count, and the registry does not report a model-based analysis or an over-dispersion assessment. It would therefore be inappropriate to claim that such an assessment was performed or that it affected the 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 (Chemotherapy, Oregovomab, SBRT, Surgery")
n      <- c(10)
events <- c(2)
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 (Chemotherapy, Oregovomab, SBRT, Surgery  10  2  20.0  2.5  55.6  5.7  51.0  6.0  51.8

11. Multiplicity, Missing Data and Analysis Populations

The registry lists a primary endpoint and secondary outcomes that include distant failure-free survival, overall survival, surgical complete resection (negative margin) rate, and tumor response rate evaluated on the pathology specimen. It reports six outcome measures in total, but analysis results for the secondary outcomes are not reported in the registry record. A plan for controlling the chance of false-positive findings across multiple outcomes is not reported in the registry record. Without that plan and the corresponding results, the listed outcomes should not be treated as confirmatory findings.

Missing-data rules are not reported in the registry record. The primary-endpoint statement refers to ten patients who remained alive, while enrollment is listed as 11. The record does not explain the endpoint status or analysis handling of the remaining participant, nor does it state whether any assessments were missing or how missing assessments were addressed. No assumptions about those circumstances should be made from the count alone.

The analysis population is not described beyond the sentence reporting the primary endpoint among patients who remained alive. The registry does not state whether the endpoint analysis included all enrolled participants, only participants with an available assessment, or another defined group. It also does not report how deaths before the endpoint assessment were treated in the progression analysis. These details matter because an analysis population defines who contributes to the estimate and how the result should be interpreted.

The registry lists zero outcome analyses and does not report an interim-analysis plan or a detailed statistical analysis plan. These absences mean that multiplicity procedures, missing-data handling, and analysis-population definitions cannot be evaluated from the registry record.

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 5% reporting threshold.

Most frequent serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: leukopenia (54.5%), thrombocytopenia (36.4%), lymphopenia (36.4%), anemia (18.2%), reduced anc (18.2%).

The adverse-event record lists 11 participants at risk for serious adverse events and 6 affected in the treatment group. It also lists 11 at risk for other adverse events and 2 affected. These are participant counts in the registry’s adverse-event summary; they do not describe the timing, duration, severity grades, or relationship of individual events to treatment. Those details are not reported in the registry record.

The serious-event terms listed with percentages are leukopenia (54.5%), thrombocytopenia (36.4%), lymphopenia (36.4%), anemia (18.2%), reduced ANC (18.2%), and ALT elevation (18.2%). The record does not provide the event counts for each term alongside those percentages, nor does it explain the denominator or the coding and grading rules in this summary. The percentages should therefore be read as registry-reported figures, not recalculated or interpreted as comparative risks.

The adverse-event group also lists deaths as 11. The registry entry does not provide the timing, causes, or circumstances associated with that figure. It should not be assumed from the summary alone that the deaths were caused by treatment or that they occurred during a particular follow-up period. No comparison group is listed, so these safety figures do not support a comparative safety estimate.

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

The result establishes what the registry reports for the primary endpoint in this study group: two of the ten patients described as remaining alive had progressive disease at 4 months, a reported rate of 20%. The endpoint is defined by a specified lesion-diameter threshold and timeframe. The record also specifies that an exact one-sided 90% confidence interval was to be constructed, but the interval is not reported. Thus, the reported count and rate are available, while the planned precision estimate is not.

The result does not establish how the progression rate would compare with another treatment approach. This was a single-group study, and no comparator arm is listed. Nor does the result identify which treatment component is associated with the observed outcome. The regimen label includes chemotherapy, oregovomab, SBRT, and surgery; nelfinavir is named among the interventions. The registry does not provide component-specific comparisons or results.

The result also does not resolve how the endpoint was handled for all enrolled participants. Enrollment is 11, while the endpoint statement describes ten patients who remained alive. The status and analysis treatment of the remaining participant are not reported in the registry record. The missing-data rules and exact analysis population are not reported either. Those limitations should remain visible when interpreting the percentage.

In general, an observed rate is a description of the analyzed group under the endpoint definition; it is not by itself proof of a causal effect. A confidence interval would add information about statistical precision, but it would not correct the absence of a comparator or replace unreported outcome-handling details. Results for the listed secondary outcomes are not reported in the registry record. No conclusion about those outcomes can be drawn from their names alone.

14. Why This Trial Matters Statistically

This trial is statistically informative as an example of why design and endpoint details must be read together. The registry states a concrete progression definition, a timeframe of up to 4 months, and an observed count and rate. It also describes an exact one-sided 90% confidence interval as the intended measure of uncertainty. Yet the interval itself is not reported, so a reader has the endpoint estimate without its stated precision summary.

The single-group structure is central to interpreting the result. A rate observed in a treatment group answers a descriptive question about that group, but without a comparator it does not estimate a treatment difference. This matters especially when the treatment approach contains multiple components and the official title refers to chemotherapy with and without immunotherapy. The registry’s listed design does not establish a randomized contrast between those strategies.

The record also illustrates the importance of defining the analysis population and handling incomplete outcomes. Enrollment is listed as 11, while the primary-endpoint statement refers to ten patients remaining alive. No explanation of the remaining participant’s endpoint status or the treatment of deaths and missing assessments is reported. A clearly reported analysis population and disposition would help readers understand precisely what the rate represents.

Finally, the registry names additional outcomes but does not report their analyses. Listing outcomes is not equivalent to presenting estimates, uncertainty, or a multiplicity plan. These distinctions are useful in clinical-trial reading: endpoint definitions, analysis details, uncertainty, and study design each answer different questions, and none can substitute for the others.

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 primary result is reported for ten patients who remained alive, although enrollment is listed as 11. The registry does not explain the endpoint status or analysis handling of the remaining participant. It also does not report how deaths, missing measurements, or other unavailable assessments were handled. This leaves the analysis population and denominator interpretation incompletely described.

The registry states that an exact one-sided 90% confidence interval was to be constructed, but the interval is not reported. Without the interval limits, the stated precision of the progression-rate estimate cannot be evaluated from the record. No sample-size justification, interim-analysis plan, or detailed statistical analysis plan is reported in the registry record.

The study is single-group, with allocation not applicable and no masking. There is no listed comparator, so the primary endpoint cannot be interpreted as a direct comparative effect. The official title refers to chemotherapy with and without immunotherapy, but the registry does not list separate groups for that contrast. It also does not report component-specific estimates that would distinguish the roles of the named treatments.

The treatment description is incomplete, ending partway through the account of the regimen. Although the group label names chemotherapy, oregovomab, SBRT, and surgery, and nelfinavir appears in the interventions, the complete administration and timing details are not reported in the registry record. Secondary outcomes are listed, but their results and analysis details are not reported. These limitations restrict interpretation to the narrow primary-endpoint statement and the registry’s descriptive design information.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Chemotherapy, Stereotactic Body Radiation Therapy & Nelfinavir Mesylate in Locally Advanced Pancreatic Cancer — NCT01959672.
• Registry record last updated 2023-10-10; status last verified 2020-02.
• Results first posted 2019-11-15.
• 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

NCT01959672 is a completed phase II, single-group study sponsored by the University of Nebraska, with enrollment listed as 11. The registry describes a treatment approach involving chemotherapy, oregovomab for patients with CA125 level >= 10, SBRT, nelfinavir, and surgery. The primary endpoint is progressive disease through 4 months, defined by at least a 25% increase in the longest diameter of a lesion relative to the longest diameter recorded since treatment started.

The registry reports progression in two of the ten patients who remained alive, with the rate stated as 20%. It describes an exact one-sided 90% confidence interval but does not report the interval. The record lists secondary outcomes, but their results are not reported. The single-group design does not provide a randomized comparator, and details on the remaining participant, missing-data handling, and analysis population are not reported in the registry record.