This page provides an independent statistical analysis and educational interpretation of publicly reported results. ClinicalTrials.gov provides the official trial registry record. Numerical trial results on this page are restricted to the results posted in the ClinicalTrials.gov record; calculations added by Clinical Biostats are labelled as such.
1. Trial at a Glance
This page covers the registry results for Neoadjuvant Folfirinox Followed by Capecitabine and Limited Field Radiation for Localized Pancreatic Head Adenocarcinoma (NCT01677988), a na phase 2 study. The primary outcome is Estimate the R0/R1 Resection Rate.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01677988 |
| Phase | Phase 2 |
| Condition | Adenocarcinoma of Head of Pancreas |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 3 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Medical University of South Carolina |
| Results posted | Yes |
2. Clinical Question
Population
People with localized adenocarcinoma of the pancreatic head were the intended population. The registry describes screening for eligibility and reports enrollment of 3 participants. Further eligibility criteria and the characteristics of those enrolled are not reported in the registry record.
Intervention
The planned treatment sequence was neoadjuvant modified FOLFIRINOX chemotherapy, followed by chemoradiation with capecitabine and limited-field radiation, and then surgical resection. The chemotherapy description specifies treatment on Day 1 and Day 15 of 28 day cycles for 3 cycles, with growth factor support.
Comparator
There was no separate control group. The study is described as single-group, with allocation listed as NA and masking as none. Consequently, the design does not provide a concurrent randomized comparison of this treatment sequence with another approach.
Primary question
The primary objective was to estimate the R0/R1 resection rate at surgery: the proportion of patients with either R0 or R1 resection status. The registry describes R0 as macroscopic complete tumor removal without microscopic residual tumor, and R1 as macroscopic complete removal with microscopic residual tumor. Although the outcome description refers to an ITT population, the population note says only subjects who had surgery were included in the outcome data. A numerical primary result is not reported in the registry record.
This is a descriptive treatment-sequence question rather than a randomized treatment-effect question. A single group can describe outcomes among participants represented in its records, but without a concurrent control group it cannot by itself establish how those outcomes compare with what would have occurred under another treatment. The registry lists radiographic tumor response, histopathologic tumor response, time to recurrence, and overall survival as secondary outcomes. Numerical results for these outcomes are not reported in the registry record.
3. Trial Design
This was a phase II, single-group study of neoadjuvant chemotherapy followed by chemoradiation and surgical resection for localized pancreatic head adenocarcinoma. The study title and intervention description identify modified FOLFIRINOX as the chemotherapy component and capecitabine with concurrent limited-field radiation as the chemoradiation component. The planned sequence placed surgery after the preoperative treatment.
The brief study description says that participants would begin chemotherapy after eligibility screening. After 3 cycles of chemotherapy, they would begin chemoradiation. Surgery was described as taking place within 4 to 8 weeks after radiation therapy was completed. The description also refers to post-treatment evaluations and follow-up every 3 months for 3 years after initial registration. These are descriptions of the intended study process; the registry record does not report how many participants completed each stage.
The registry lists enrollment of 3 and a terminated status. The start is recorded as 2012-07 and primary completion as 2015-10. The reason for termination, the date on which termination occurred, and whether the study stopped before or after any particular participant reached surgery are not reported in the registry record. Allocation is NA, masking is none, and the model is single-group. Thus, there is no randomized allocation or masked comparison described.
The primary endpoint was an estimate of the R0/R1 resection rate at the time of surgery. The outcome description refers to an ITT population, but its population note says that only subjects who had surgery were included in the outcome measure data. The registry does not clarify how these statements were reconciled, identify the numerical denominator used, or report a primary outcome count. The sponsor is the Medical University of South Carolina. Other protocol details, including the statistical analysis plan and sample-size justification, 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
| Stage | Chemotherapy, Chemoradiation, Surgery |
|---|---|
| STARTED | 3 |
| COMPLETED | 1 |
| NOT COMPLETED | 2 |
Reasons for not completing this period:
| Reason | Chemotherapy, Chemoradiation, Surgery |
|---|---|
| Lack of Efficacy | 2 |
Analysis population for the primary outcome, as defined in the registry: Only subjects who had surgery were included in the outcome measure data.
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.
| Characteristic | Unit | Chemotherapy, Chemoradiation, Surgery |
|---|---|---|
| Participants analysed | 3 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 2 |
| Age, Categorical: >=65 years | Participants | 1 |
| Sex: Female, Male: Female | Participants | 1 |
| Sex: Female, Male: Male | Participants | 2 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 0 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 3 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 |
| Race (NIH/OMB): Asian | Participants | 0 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 |
| Race (NIH/OMB): Black or African American | Participants | 1 |
| Race (NIH/OMB): White | Participants | 2 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 |
6. Primary Endpoint
The registry lists the primary outcome as Estimate the R0/R1 Resection Rate, measured over at time of surgery.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint was the R0/R1 resection rate, described as the proportion of patients with either R0 or R1 resection status at surgery. R0 means macroscopic complete removal by a non-contaminated operation with neither macroscopic nor microscopic residual tumor. R1 means macroscopic complete removal by a non-contaminated operation with microscopic residual tumor. Combining these categories makes the endpoint a binary outcome: a participant is counted as having R0 or R1 status, or not counted in that combined category.
A resection-status endpoint can describe the surgical result after a preoperative treatment sequence. It is used to address the study’s stated objective of estimating the frequency of R0/R1 status. As a general statistical point, a proportion is interpretable only alongside a clear denominator and a definition of which participants contribute to it. The registry’s description refers to an ITT population, while its population note says only participants who had surgery were included in the outcome data. The record does not explain the relationship between those descriptions or provide the analyzed denominator.
The outcome timeframe is at the time of surgery. The numerical R0/R1 count and rate, and any uncertainty interval around the estimate, are not reported in the registry record. Therefore the size and precision of the primary endpoint estimate cannot be evaluated from the registry information described. The outcome definition also does not make this single-group endpoint a comparison against another treatment. It can be used to describe the recorded resection status, but comparative conclusions would require a suitable comparator and analysis, neither of which is described for this study.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record identifies an intended treatment sequence and a primary surgical endpoint, but does not report a numerical primary outcome result. In particular, it does not give the count of participants with R0 or R1 status, the denominator used for the rate, or an uncertainty interval. The primary objective was to estimate the R0/R1 resection rate, but the estimate itself cannot be characterized from the registry information. No conclusion about the observed frequency or precision of the endpoint is warranted from the record alone.
The registry reports enrollment of 3 participants and a terminated status. Enrollment describes the study’s reported recruitment count; it does not establish how many participants received all intended treatment, proceeded to surgery, or contributed to the primary endpoint. The population note says only participants who had surgery were included in the outcome data, but does not state how many had surgery. The mismatch between this note and the outcome description’s reference to an ITT population makes the analysis population unclear. The record does not report how participants without surgery, if any, were handled in the primary analysis.
The design is single-group, with allocation listed as NA and masking listed as none. This means the registry does not describe a randomized control group for evaluating a difference between treatment strategies. As a general statistical point, outcomes observed in one group may reflect the enrolled participants, the treatment sequence, clinical decisions, and other influences. Without an appropriate comparison, the observed outcome cannot isolate the effect of the treatment sequence from those other factors. In this case, even a descriptive numerical primary result is not reported.
The study status is terminated. The reason for termination and its implications for enrollment, follow-up, or endpoint collection are not reported in the registry record. The record gives a primary completion date of 2015-10, but does not provide the course of recruitment or follow-up that led to termination. The listed follow-up plan describes evaluations every 3 months for 3 years after initial registration; completion of that planned follow-up is not established by the information recorded.
Radiographic tumor response, histopathologic tumor response, time to recurrence, and overall survival are listed as secondary outcomes. The registry identifies 8 outcome measures and 0 analyses, but does not report numerical results for these secondary outcomes. The existence of listed endpoints should not be mistaken for evidence that their results are available or that they were analyzed using a particular method. The registry record does not report p-values, confidence intervals, an analysis plan, or adjusted estimates.
Overall, the available information supports a description of the study design, intended treatment sequence, outcome definition, and limited enrollment. It does not support an estimate of the primary endpoint or a comparative assessment of treatment effect. The record’s small reported enrollment and unclear endpoint denominator are important constraints on interpreting any estimate that might otherwise be considered.
9. Statistical Methods Explained
What kind of endpoint is being measured?
The primary outcome is a binary status: whether a participant had R0 or R1 resection status at surgery. The registry describes R0 and R1 separately, then defines the primary measure as the proportion in either category. In general, a binary endpoint records whether a specified event or condition is present, rather than measuring a continuously varying quantity. A proportion summarizes the count meeting the definition relative to a stated analysis denominator.
The denominator matters because the outcome description refers to an ITT population, while the population note says only subjects who had surgery were included in the outcome data. Those descriptions may represent different analysis populations, but the registry does not explain the intended handling. It does not state the denominator for the reported endpoint or describe how people who did not reach surgery would be treated in an ITT analysis. The numerical outcome itself is not reported in the registry record.
How should uncertainty be considered?
As a general statistical concept, a confidence interval describes the range of values reasonably compatible with an estimate under a specified statistical model and procedure. It conveys uncertainty in the estimate; it does not guarantee that the true value lies in the interval. For a proportion, precision depends in part on the amount of information available and on how the outcome and analysis population are defined. When the sample is limited, estimates can be imprecise, and small changes in observed counts can matter substantially.
No confidence interval for the primary outcome is reported in the registry record, and there is no numerical outcome estimate from which to assess precision. The record also does not report a primary-outcome p-value. Because the design is single-group, a test comparing treatment arms is not described. In general, a one-group estimate and its uncertainty answer a descriptive question; they do not by themselves establish a causal treatment effect.
What does the design imply for inference?
The model is single-group, allocation is listed as NA, and masking as none. There is no concurrent randomized comparator described. Randomization in a comparative trial is generally used to help balance prognostic factors between groups, while masking can reduce some forms of knowledge-related influence on treatment or outcome assessment. These are general design concepts, not claims that any particular bias occurred in this study. The registry does not report additional protections, adjudication procedures, or a statistical plan for outcome assessment.
Without a comparator, differences between observed outcomes and outcomes under another treatment cannot be estimated directly from this design. A historical or external comparison would require specific data and assumptions; none is described in the registry record. The treatment sequence also includes chemotherapy, chemoradiation, and surgery, so the single-group design would not separate the contribution of each component even if numerical outcomes were reported.
How should sample-size planning be read?
The sample-size planning illustrations are described as calculations based on the observed effect, using a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. They describe planning for fractions of an observed effect. For time-to-event outcomes, the stated calculations refer to a total event count using the Schoenfeld method; other calculations refer to participants per arm.
These assumptions describe planning illustrations, not the design or analysis actually used in this single-group study. In particular, an equal-allocation, two-arm calculation does not turn the study into a randomized comparison. The numerical observed effect and the numerical planning results are not reported in the registry record. Any such planning illustration is not observed power: it cannot show the probability that this completed study detected an effect, and it does not establish that an effect exists. The record does not report the trial’s own sample-size justification, target enrollment, or a formal power calculation for its primary endpoint.
What is known about analysis choices?
The record lists 8 outcome measures and 0 analyses. It does not report a statistical analysis plan, model specifications, covariate adjustments, handling of missing data, or multiplicity procedures. It also does not report numerical estimates or inferential statistics for the primary or listed secondary outcomes. As a general matter, analytic choices should be specified in advance and reported clearly so that readers can understand how outcomes, denominators, uncertainty, and multiple endpoints were handled. Those details cannot be reconstructed from the registry information.
The registry’s primary outcome definition names the ITT population, while the population note limits outcome data to participants who had surgery. An ITT approach generally seeks to analyze participants according to their assigned group, regardless of whether they complete the intended treatment. In a single-group study, the exact meaning depends on the protocol and outcome definition. The record does not supply enough detail to determine how that approach was intended to work. It would be inappropriate to assume that participants without surgery were classified as failures, excluded, or handled in some other way.
For the listed safety data, the registry records participant counts for serious and other adverse events. Counts of participants affected are not necessarily counts of all event episodes. As a general point, one participant may experience more than one event, and categories or event terms may overlap. The registry record does not report event-level details, exposure-adjusted rates, or a statistical comparison of adverse events.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists radiographic tumor response, histopathologic tumor response, time to recurrence, and overall survival as secondary outcomes, in addition to the primary R0/R1 resection-rate outcome. It also reports 8 outcome measures and 0 analyses. A list of several outcomes raises a general multiplicity question: when many hypotheses are examined, the chance of at least one apparently unusual result can increase if each is assessed independently. Statistical procedures can address multiplicity, but the appropriate approach depends on the objectives and analysis plan.
No multiplicity adjustment, hierarchy of endpoints, or prespecified strategy for interpreting secondary outcomes is reported in the registry record. Numerical results and analyses for the listed secondary outcomes are also not reported. Therefore, it is not possible to determine whether multiple testing was considered, or how any secondary finding would have been interpreted alongside the primary objective.
Missing-data methods are not reported. The primary endpoint’s description refers to an ITT population, whereas its population note says only participants who had surgery were included in the outcome data. The registry does not report the number who reached surgery, the number with an evaluable resection status, or the treatment of participants without a recorded endpoint. It also does not specify whether missingness was imputed, treated as a particular outcome category, or handled by another method. No such approach should be assumed.
The analysis-population definition is therefore uncertain. The record gives enrollment of 3, but that does not establish the denominator for the primary endpoint. A clear account of participant flow and outcome availability would be needed to distinguish enrollment from treatment receipt, surgery, and inclusion in the endpoint analysis. Those details are not reported in the registry record.
11. Safety Results
The registry reports serious adverse events as affected participants divided by participants at risk.
| Arm | Participants with serious adverse events | Participants at risk | Percentage |
|---|---|---|---|
| Chemotherapy, Chemoradiation, Surgery | 2 | 3 | 66.7% |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 0% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Chemotherapy, Chemoradiation, Surgery | 3 of 3 |
Most frequent serious adverse event terms
| Event term | Organ system | Chemotherapy, Chemoradiation, Surgery |
|---|---|---|
| bile duct stenosis | Hepatobiliary disorders | 1 of 3 |
| abdominal pain | Gastrointestinal disorders | 1 of 3 |
| chills | General disorders | 1 of 3 |
| fever | General disorders | 1 of 3 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Chemotherapy, Chemoradiation, Surgery |
|---|---|---|
| fatigue | General disorders | 3 of 3 |
| diarrhea | Gastrointestinal disorders | 2 of 3 |
| nausea | Gastrointestinal disorders | 2 of 3 |
| vomiting | Gastrointestinal disorders | 2 of 3 |
| pain | General disorders | 2 of 3 |
| anemia | Blood and lymphatic system disorders | 1 of 3 |
| leukocytosis | Blood and lymphatic system disorders | 1 of 3 |
| ear infection | Ear and labyrinth disorders | 1 of 3 |
| chills | General disorders | 1 of 3 |
| fever | General disorders | 1 of 3 |
Serious events with the highest arm-level rate: bile duct stenosis (33.3%), abdominal pain (33.3%), chills (33.3%), fever (33.3%).
The registry lists one study group, titled “Chemotherapy, Chemoradiation, Surgery.” For serious adverse events, it records 3 participants at risk and 2 affected. For other adverse events, it records 3 participants at risk and 3 affected. These are registry counts for the recorded group; they do not provide a comparison with another treatment or with an untreated group. The death count is not reported in the registry record.
The serious adverse event terms listed are bile duct stenosis, abdominal pain, chills, and fever, each with a reported frequency of 33.3%. The registry does not provide further event-level detail in the information recorded, such as timing, duration, severity beyond the serious classification, or whether a participant experienced more than one listed term. The listed terms should not be added together: a participant may have more than one event, and the record does not establish that the categories are mutually exclusive.
As a general statistical point, adverse-event counts describe events recorded under the study’s reporting definitions. They do not establish whether an event was caused by treatment. Interpretation also depends on observation time and completeness of reporting, details not reported in the registry record. The figures should therefore be read as descriptive safety information for this single group, not as comparative evidence about risk.
12. What the Result Does, and Does Not, Mean
The registry record documents a phase II, single-group study intended to evaluate a sequence of neoadjuvant modified FOLFIRINOX, chemoradiation, and surgery for localized pancreatic head adenocarcinoma. It defines the primary endpoint as the proportion of participants with R0 or R1 resection status at surgery. It also records enrollment of 3 and a terminated status. These registry entries describe the study’s purpose and recorded design; they do not give the primary endpoint result.
The central numerical limitation is that the registry does not report the R0/R1 count, rate, denominator, or confidence interval. The outcome population is also unclear because the endpoint description names an ITT population while the population note says only those who had surgery contributed to outcome data. Consequently, no estimate of the primary endpoint’s size or precision can be drawn from the registry record. The record does not establish how many participants received the planned sequence or reached surgery.
The design does not include a randomized control group. As a general principle, a single-group study can describe what was recorded among its participants, but it cannot provide a direct randomized comparison with another treatment. Even if a primary outcome estimate were available, the design would not isolate the effect of this treatment sequence from other influences, and it would not distinguish the effects of chemotherapy, chemoradiation, and surgery from one another.
The listed secondary outcomes include radiographic tumor response, histopathologic tumor response, time to recurrence, and overall survival, but numerical results for these outcomes are not reported in the registry record. The safety information is also descriptive and does not include a comparator. The study’s terminated status is known, but the reason and consequences for data collection are not reported.
Accordingly, the record supports a limited account of the intended study design, endpoint definition, enrollment, status, and recorded adverse-event counts. It does not support a conclusion about the frequency of R0/R1 resection, the precision of that estimate, comparative effectiveness, or comparative safety. Those conclusions require outcome data and analysis details that are not reported in the registry record.
13. Why This Trial Matters Statistically
This study illustrates why an endpoint definition alone is not enough for statistical interpretation. The registry specifies what counts as R0 and R1 resection status and identifies the time of assessment, but does not report the endpoint count or rate. Without those results, readers cannot assess the estimated frequency of the primary outcome or how precisely it was measured.
The population wording also matters. The primary description refers to an ITT population, while the population note says outcome data included only participants who had surgery. A reader needs to know the denominator and how participants without surgery were handled to understand what the proportion represents. The record does not clarify this, and enrollment of 3 cannot be substituted for the missing endpoint denominator.
The single-group design is another key statistical feature. It can be used to describe outcomes in the enrolled group, but it does not provide a randomized treatment comparison. This distinction prevents the study design from answering a comparative causal question. A treatment sequence involving chemotherapy, chemoradiation, and surgery also does not separate the contribution of each component.
Finally, terminated status, a small reported enrollment, multiple listed outcomes, and absent numerical results all shape what can be concluded. The record does not explain why the study terminated, describe its analysis plan, or report the primary or secondary outcome estimates. These omissions show why transparent reporting of participant flow, denominators, uncertainty, and analysis choices is essential: without them, even a clearly named endpoint cannot be interpreted as a complete statistical result.
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.
Several limitations are specific to the information recorded for this study. The registry reports enrollment of 3, but does not report the number who completed chemotherapy, received chemoradiation, underwent surgery, or contributed to the primary outcome. The primary endpoint population is described both as ITT and as including only subjects who had surgery. The registry does not reconcile those statements or identify the denominator used for the endpoint.
The primary R0/R1 outcome result is not reported in the registry record. Neither its count nor its rate nor an uncertainty interval is available in the information described. Numerical results for the listed secondary outcomes are also not reported. The record lists 8 outcome measures and 0 analyses, but does not explain the analysis status or provide an analysis plan.
The study is single-group, with no randomized comparator described. Accordingly, the design has no concurrent control for separating treatment effects from other influences. Masking is listed as none. The record does not report additional procedures for independent outcome assessment or adjudication. These features constrain interpretation but do not establish that bias occurred.
The registry reports that the study was terminated, but the reason for termination and its impact on participant follow-up or outcome collection are not reported. Although follow-up every 3 months for 3 years is described in the study summary, completion of that planned follow-up is not established. The trial’s sample-size justification, detailed statistical plan, missing-data methods, and multiplicity strategy are not reported in the registry record.
These limitations concern what can be established from the record. They should not be used to infer unreported participant outcomes, reasons for termination, or analytic decisions.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Neoadjuvant Folfirinox Followed by Capecitabine and Limited Field Radiation for Localized Pancreatic Head Adenocarcinoma — NCT01677988.
- Registry record last updated 2016-01-15; status last verified 2015-07.
- Results first posted 2016-01-15.
- 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.
Continue through the Clinical Biostats statistical pathway
Use this trial's endpoint as a starting point for deeper study of effect measures, confidence intervals, and sample-size planning.
18. Record Summary
NCT01677988 is a phase II, single-group study of neoadjuvant modified FOLFIRINOX followed by capecitabine with concurrent limited-field radiation and surgical resection for localized pancreatic head adenocarcinoma. The registry lists enrollment of 3, no masking, and terminated status. The start is recorded as 2012-07 and primary completion as 2015-10.
The primary objective was to estimate the R0/R1 resection rate at surgery. The endpoint description refers to an ITT population, while the population note says only subjects who had surgery were included in the outcome data. The numerical primary result and its denominator are not reported in the registry record. Listed secondary outcomes include radiographic and histopathologic tumor response, time to recurrence, and overall survival; numerical results are not reported.
The registry records serious adverse events in the single study group and lists four serious adverse event terms at 33.3% each. The reason for termination, the detailed analysis plan, and the handling of missing outcomes are not reported in the registry record.