FOLFIRINOX for Unresectable Locally Advanced and Borderline Resectable Pancreatic Cancer: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for FOLFIRINOX for Unresectable Locally Advanced and Borderline Resectable Pancreatic Cancer (NCT01688336), a na phase 2 study. The primary outcome is Median Overall Survival (OS) of FOLFIRINOX in Patients With Unresectable Locally Advanced (ULA) Pancreatic Cancer.

2. Clinical Question

The study was intended to assess safety and efficacy in both ULA and BR disease, but its stated primary endpoint applies only to the ULA population. The registry lists secondary outcomes concerning OS for BR patients, progression-free survival (PFS), objective response rate, disease control rate, and rate of resectability. Those outcomes address different aspects of the disease course and should not be treated as interchangeable with the primary OS measure.

Because all participants received FOLFIRINOX and there was no control group, the central question is descriptive: what outcomes were observed among the treated participants? A single-group result cannot by itself distinguish outcomes associated with treatment from outcomes that might have occurred without it or under another treatment. The registry record does not report an OS median, a confidence interval, or analyses for the listed outcomes. Consequently, it does not supply a numerical answer to the primary question.

3. Trial Design

This was a phase II, multi-center, single-group clinical trial of FOLFIRINOX for pancreatic cancer described as unresectable locally advanced or borderline resectable. The registry lists enrollment of 9 and identifies UNC Lineberger Comprehensive Cancer Center as the sponsor. Allocation is recorded as not applicable, and masking as none. All subjects were assigned to the FOLFIRINOX group; there was no concurrent comparison arm.

The registry gives a start date of 2012-01 and a primary completion date of 2016-08-22. The current status is terminated. The reason for termination is not reported in the registry record, so the status alone does not explain why the study ended or whether planned recruitment or follow-up was completed.

The primary endpoint was median OS among patients with ULA pancreatic cancer. The endpoint description says that patients receiving at least Day 1 of FOLFIRINOX would be evaluable and followed for up to 3 years. This is a stated outcome population and follow-up plan; the record does not report how many enrolled participants received treatment, how many met the primary endpoint population definition, or how much follow-up was actually observed.

The study also lists secondary outcomes for BR-patient OS, PFS, objective response, disease control, and resectability. The registry records 7 outcome measures, but the record does not provide analyses for them. It reports 0 analyses. A planned outcome and an outcome measure listing indicate what the study intended to assess; they do not establish that a result was observed, analyzed, or reported.

With no randomized or other control group, this design can describe outcomes in the study group but cannot provide a direct between-treatment estimate. Neither a causal effect nor comparative efficacy can be inferred from a treated group alone. Details such as the rationale for the enrollment target, the reason for termination, and any changes to the study plan are 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

Analysis population for the primary outcome, as defined in the registry: Patients with unresectable locally advanced pancreatic cancer.

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 Median Overall Survival (OS) of FOLFIRINOX in Patients With Unresectable Locally Advanced (ULA) Pancreatic Cancer, measured over Up to 3 years.

The primary endpoint is median overall survival (OS) in patients with unresectable locally advanced pancreatic cancer. OS refers to the time from a defined starting point until death from any cause. The registry describes follow-up for up to 3 years for patients who receive at least Day 1 of FOLFIRINOX, but does not state a numerical median or confidence interval among the posted outcome results.

A median is a time-to-event summary: it identifies the point at which the estimated survival distribution reaches its midpoint. Unlike a simple average, it does not require every participant to experience the event during observation. Some participants may still be alive at their last follow-up, making their survival times censored rather than fully observed. Time-to-event methods are designed to use such partial follow-up while accounting for the fact that the eventual event time is unknown.

A confidence interval communicates uncertainty around an estimate. A narrower interval generally indicates greater precision, while a wider interval indicates less precision. The registry specifies a 95% confidence interval as the intended dispersion measure, but the interval itself is not reported in the registry record.

OS is used as a direct time-to-event outcome, but its interpretation depends on the population definition, follow-up, and completeness of outcome ascertainment. Here, the primary population is ULA patients, not all participants with either ULA or BR disease. The record does not give the observed number of primary-population participants or the resulting OS estimate, so the endpoint definition alone cannot establish what OS was in this study.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

Time-to-event analysis is used when the outcome is the time until an event, such as death. A participant who has not had the event by the last known follow-up contributes information up to that point, but the eventual event time is unknown. This is called censoring. A survival analysis can incorporate those partial observations rather than treating each participant's follow-up as if it were complete. The registry identifies median OS as the primary outcome and specifies a 95% confidence interval, but does not report the estimate or interval.

The median is a distribution-based summary, not an average survival time. Its meaning depends on the estimated survival curve and the amount of follow-up and event information available. If the curve does not reach its midpoint during observation, the median may not be estimable from the observed follow-up. The registry record does not state whether that occurred; it does not provide the OS result at all. The planned follow-up was up to 3 years, which should not be read as the actual follow-up duration for every participant.

A confidence interval describes the statistical uncertainty surrounding an estimate under the assumptions of the analysis. It is not a guarantee that a particular interval contains the true value, and it does not address all possible sources of bias. For this trial, the record names a 95% confidence interval as the dispersion measure but does not report its endpoints. Without the estimate and interval, readers cannot judge the precision of the primary OS result.

The registry identifies the study as single-group, with no allocation to different arms and no masking. In a randomized comparison, allocation can support a direct comparison between groups, subject to the design and analysis. Here there is no control group. As a result, even a reported survival estimate would describe the treated study group rather than quantify a treatment effect relative to a concurrent control. General differences between one study's results and another study's results are not equivalent to a randomized comparison.

Hazard ratios are relative summaries used in time-to-event comparisons; their interpretation depends on the groups being compared and the model or analysis producing the estimate. The record does not report a numerical sponsor hazard ratio or any analyses. Thus, there is no reported hazard ratio to interpret. A hazard ratio cannot be recovered from the median endpoint description alone without additional data and assumptions, and no such calculation is warranted from this record.

Over-dispersion is a general statistical issue that arises when observed variation is greater than a model assumes. If it is ignored, uncertainty can be understated and tests can be too optimistic. Its relevance depends on the outcome type and analysis model. The registry record does not report the statistical model or any such diagnostic, so it is not possible to say whether over-dispersion was assessed or applicable to a particular analysis in this trial.

The sample-size illustrations use assumptions stated as a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation, with the effect to detect expressed as a fraction of the observed effect. For time-to-event outcomes, the illustration concerns a total number of events; for other outcomes, it concerns participants per arm. These are planning illustrations, not observed power and not the study's own design justification. In particular, equal allocation between two arms is a hypothetical illustration and does not describe this single-group trial. The sample-size rationale used by the investigators is not reported in the registry record.

Multiplicity arises when many outcomes or analyses are considered. Testing several hypotheses can increase the chance of at least one apparently unusual result under a null setting, unless the analysis plan addresses that issue. The record lists 7 outcome measures and 0 analyses, and names several secondary outcomes, but does not report whether multiplicity adjustments or a hierarchy of testing were planned. Therefore, no claim about multiplicity control can be made.

Analysis populations also matter. The primary description says that all patients who receive at least Day 1 of FOLFIRINOX will be evaluable, and specifies ULA patients as the population for the primary endpoint. The record does not report the number meeting this definition or the number included in an analysis. It also does not report missing outcome data, censoring details, exclusions, or analysis methods. These omissions limit the ability to assess how the intended endpoint relates to the participants who contributed information.

10. Multiplicity, Missing Data and Analysis Populations

The registry lists 7 outcome measures, including the primary OS endpoint and secondary outcomes for OS in BR patients, PFS, objective response rate, disease control rate, and rate of resectability. It records 0 analyses. The record does not explain the status or results of the listed measures beyond their descriptions, and any outcome measure not specified by name is not reported in the registry record.

When a study considers several outcomes or statistical tests, multiplicity can increase the chance of a positive-looking result arising by chance. Statistical plans may address this through prespecified outcome hierarchies or adjustment methods. Whether either approach was used is not reported in the registry record. With no analyses reported, there are no posted inferential results for which to assess multiplicity handling.

The primary endpoint description defines the evaluable group as patients receiving at least Day 1 of FOLFIRINOX and identifies the primary population as ULA patients. Enrollment was 9, but the number who received treatment, met the ULA definition, or contributed to the primary endpoint analysis is not reported in the registry record. The record also does not give the number of participants with complete outcome information.

Missing-data methods, censoring details, analysis exclusions, and the handling of participants who withdrew or lacked follow-up are not reported in the registry record. Because OS is a time-to-event outcome, incomplete follow-up can affect the information available even when a participant's event time is not observed. Without details of follow-up and analysis populations, readers cannot assess the extent or handling of that uncertainty. These are reporting gaps; they do not establish that data were missing or that any particular method was or was not used.

11. 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: death (11.1%), urinary tract infection (11.1%), renal calculi (11.1%).

The registry records 9 participants at risk for serious adverse events and 2 affected. For other adverse events, it records 9 at risk and 9 affected. The group entry also lists 7 deaths. These figures describe registry entries for the FOLFIRINOX group; there is no control group with which to compare them.

The serious-event terms listed are Death, Urinary Tract Infection, and Renal Calculi, each with a reported frequency of 11.1%. The term-level percentages and the separate count of 7 deaths are distinct registry fields. The record does not explain their relationship, so the term percentage should not be used to reinterpret the deaths count. It also does not state whether any listed event was considered related to FOLFIRINOX, how events were adjudicated, or the timing of events.

These entries do not estimate comparative risk and cannot show whether events were more or less frequent than they would have been under another treatment. Affected counts are not a full account of severity, duration, recurrence, or causality. The registry record does not report those details. The figures should therefore be read as descriptive safety reporting rather than as a causal or comparative safety conclusion.

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

The record establishes that a phase II, single-group study of FOLFIRINOX was registered for patients with ULA or BR pancreatic cancer, with a primary OS question focused on ULA patients. It also records enrollment of 9, a terminated status, and descriptive adverse-event entries. These registry entries describe the study and its posted information; they do not supply a numerical primary-outcome result.

The record does not report the median OS, its 95% confidence interval, or analyses for the listed secondary outcomes. That absence means readers cannot assess the magnitude or precision of the primary result from this record. It does not mean that OS was zero, that no participants survived, or that the treatment had no effect. The outcome result is simply not reported in the registry record.

Because there is no comparator arm, the design cannot estimate a direct difference in outcomes between FOLFIRINOX and another intervention. Any observed outcome in a single treatment group could reflect factors beyond treatment. The registry does not offer a basis for causal attribution or for comparing the group with an external population.

The safety entries also need a narrow interpretation. They record counts and serious-event terms, but do not establish causation, comparative risk, or the reason for any death. The status of terminated does not explain why the trial ended, and the planned follow-up does not establish actual follow-up for each participant.

Accordingly, the statistical contribution that can be read from this record is primarily about design, endpoint definition, and the limits of posted reporting. An interpretable efficacy estimate would require outcome results and their analysis details; comparative conclusions would additionally require an appropriate comparison. Those elements are not reported in the registry record. The listed planning illustrations, if considered, are hypothetical design calculations rather than observations from participants and should not be taken as evidence that the trial had a particular probability of detecting an effect.

13. Why This Trial Matters Statistically

This trial is statistically notable for the gap between a clearly stated time-to-event endpoint and the lack of a reported endpoint estimate. The registry identifies median OS, the target population, and the intended confidence-interval measure. Yet it records 0 analyses and does not provide the primary result. Readers can understand what the study set out to measure, but cannot assess the observed survival estimate or its precision from this record.

The single-group design also defines what the study could answer. It can describe outcomes among participants receiving FOLFIRINOX, but it does not support a direct randomized comparison. A numerical outcome, even if reported, would not alone distinguish treatment-associated outcomes from other explanations. This distinction between describing a group and estimating a comparative effect is fundamental to interpreting clinical trial evidence.

The record also illustrates the importance of keeping the primary population separate from the broader study eligibility description. The trial included ULA and BR disease categories, while the primary OS question is specifically for ULA patients. Without a reported analysis population, the enrollment total cannot be assumed to represent the primary endpoint sample.

Finally, the registry's safety entries illustrate why counts, event terms, and causal conclusions must be kept separate. The record reports affected counts and serious-event terms but supplies no comparator or attribution detail. The terminated status adds a further uncertainty because its reason is not reported. Taken together, these features make transparent reporting of outcome estimates, analysis populations, follow-up, and reasons for discontinuation especially important for interpreting the study statistically.

14. 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 available record has substantial limits for outcome interpretation. Although the primary endpoint and its intended confidence-interval measure are identified, no median OS estimate or interval is reported. The secondary outcomes are listed, but analyses and results are not reported. Without those values, neither the magnitude nor the precision of any efficacy outcome can be assessed.

Enrollment was 9, but the record does not state the number of participants who received at least Day 1 of treatment, the number with ULA disease, or the number included in the primary analysis. The actual follow-up duration, censoring pattern, outcome completeness, and missing-data handling are also not reported in the registry record. The planned follow-up of up to 3 years does not resolve these unknowns.

The design has no comparator arm and no random allocation. It therefore cannot provide a direct comparative estimate, and outcomes within the treated group cannot by themselves establish causation. The registry does not report the statistical analysis methods or a sample-size justification. The planning assumptions described for hypothetical sample-size illustrations are not the trial's own design justification or evidence of observed power.

The study status is terminated, but the reason is not reported in the registry record. It is also not possible to determine from the record whether the planned enrollment, follow-up, or analyses were completed. Safety information is limited to recorded counts and terms; event timing, attribution, and comparative rates are not reported. These limitations reflect what can be interpreted from the registry record and do not establish that unreported procedures or information did not exist outside it.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: FOLFIRINOX for Unresectable Locally Advanced and Borderline Resectable Pancreatic Cancer — NCT01688336.
• Registry record last updated 2017-10-06; status last verified 2017-09.
• Results first posted 2017-08-21.
• 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.

18. Record Summary

NCT01688336 is a phase II, multi-center, single-group trial of FOLFIRINOX in patients with ULA or BR pancreatic cancer, sponsored by UNC Lineberger Comprehensive Cancer Center. The registry records enrollment of 9, no masking, and terminated status. Its primary endpoint is median OS in patients with ULA pancreatic cancer, with a stated 95% confidence interval and follow-up up to 3 years.

The record lists secondary outcomes and 7 outcome measures, but records 0 analyses. A primary OS estimate, confidence interval, and secondary outcome results are not reported in the registry record. Safety entries include 2 of 9 participants affected by serious adverse events, 9 of 9 affected by other adverse events, and 7 deaths; serious-event terms listed include Death, Urinary Tract Infection, and Renal Calculi, each at 11.1%. The reason for termination and the details needed for comparative or causal conclusions are not reported in the registry record.