BATS With in Combination With Low Dose IL-1 and GM-CSF for Advanced Pancreatic Cancer: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for BATS With in Combination With Low Dose IL-1 and GM-CSF for Advanced Pancreatic Cancer (NCT02620865), a na phase 1, phase 2 study. The primary outcome is Median Overall Survival (OS).

2. Clinical Question

The broad clinical question was whether this treatment approach could be studied for side effects, dose, and activity in advanced pancreatic cancer. The registry’s stated primary endpoint focuses on overall survival, while listed secondary outcomes include toxicity, progression-free survival, time to progression, immune response, and biomarker analyses. Those outcomes address different aspects of a trial: survival outcomes describe time until an event, toxicity describes harms recorded during treatment, and immune or biomarker outcomes describe laboratory measures.

Because there was no randomized comparator, the study cannot directly estimate how outcomes differed between the treatment approach and a control regimen. Its design is therefore suited to describing observations within the treated group, not to isolating the effect of BATs from chemotherapy, other co-interventions, or the course of the disease.

3. Trial Design

The registry identifies this as a phase Ib/II, completed, single-group trial sponsored by Barbara Ann Karmanos Cancer Institute. The start is recorded as 2015-12 and the primary completion as 2021-06-21. Enrollment was 2. Allocation is listed as not applicable, and masking as none. Those features mean the record does not describe a randomized, masked comparison between treatment groups.

The treatment arm is named “Treatment (anti-CD3 x anti-EGFR BATs).” Its description says patients received one of several standard chemotherapy regimens at the treating physician’s discretion, alongside the study treatment. The description in the registry record is incomplete, ending during a regimen description, so the full regimen details are not reported in the registry record. The registry lists aldesleukin, antibody therapy, fluorouracil, gemcitabine hydrochloride, irinotecan hydrochloride, leucovorin calcium, and oxaliplatin among the interventions; it also lists laboratory biomarker analysis.

The title and narrative use different wording for the immune-stimulating combination: the short title refers to low-dose IL-1 and GM-CSF, while the official title refers to low-dose IL-2 and GM-CSF. The brief summary names aldesleukin and sargramostim. These differences are part of the record and should not be resolved by inference.

With enrollment of 2 and no control group, the trial provides a limited basis for describing outcomes in the enrolled group. The registry’s phase Ib/II designation indicates the stated development phase, but a detailed protocol, dose-escalation account, and statistical analysis plan are not reported in the registry record. The record lists the study as completed; that status alone does not provide the missing endpoint results.

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 Median Overall Survival (OS), measured over Up to 18 months.

Overall survival is the time from a defined starting point until death from any cause. It is commonly used in oncology because it captures a clinically consequential event and does not depend on how progression is defined or assessed. As a time-to-event outcome, it can also account for participants whose survival time is not fully observed by the end of follow-up, provided the analysis handles censoring appropriately.

The primary endpoint was median overall survival, expressed in years, with a 90% confidence interval. The registry specifies a Kaplan–Meier estimate, calculated on an intention-to-treat basis using all registered patients and on a response-evaluable basis using patients who completed all BAT infusions. The timeframe is listed as up to 18 months. However, the numerical median, confidence limits, event count for the endpoint, and detailed follow-up results are not reported in the registry record.

General statistical explanation: the median is the time at which the estimated survival curve reaches one-half. A confidence interval conveys uncertainty around an estimate; it is not a range containing a fixed proportion of individual survival times. With enrollment of 2, the estimate would be highly dependent on the observed follow-up and event pattern, and its precision cannot be assessed without the reported estimate and interval. The stated endpoint therefore describes what the trial planned to estimate, but does not by itself show the observed survival result.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The registry describes a Kaplan–Meier approach for the primary endpoint, median overall survival. General statistical explanation: Kaplan–Meier estimation builds a survival curve over follow-up, updating the estimated proportion surviving when deaths occur and accounting for participants whose observation ends before a death is recorded. Such participants are censored at their last known observation time. Censoring is not itself a death; the method relies on assumptions about how loss of observation relates to future survival. The registry does not report censoring details or the numerical curve estimates.

What does the median describe?

The median survival is the time associated with the point where the estimated survival curve reaches one-half. It is a summary of the curve, not a prediction for any one participant. If follow-up does not allow the curve to reach that point, a median may be difficult or impossible to estimate. The registry says the median would be estimated, but does not report the resulting value, whether it was estimable, or its confidence limits. The stated timeframe is up to 18 months; that timeframe does not establish that every participant was observed for the full period.

Why report a confidence interval?

The planned interval is a 90% confidence interval. General statistical explanation: a confidence interval expresses uncertainty in an estimate under the assumptions of the method. It does not describe the spread of individual outcomes, and it does not mean there is a 90% chance that a particular participant’s survival time falls inside it. With enrollment of 2, the estimate can be strongly affected by the observed event and follow-up pattern. Since the interval endpoints are not reported in the registry record, the precision of the survival estimate cannot be assessed.

Why are there two analysis populations?

The registry states that overall survival would be estimated on an intention-to-treat basis using all registered patients, and on a response-evaluable basis using patients who completed all BAT infusions. These populations answer different descriptive questions. An analysis including all registered patients preserves the initial enrolled group, regardless of treatment completion. An analysis restricted to treatment completers describes a selected group defined by what happened after enrollment. General statistical explanation: completion can be related to health status, tolerability, or other factors, so a completer-only estimate may not represent the experience of everyone who started the study. The record does not report results for either population or explain how missing follow-up was handled.

What can a single-group design estimate?

This study is recorded as single-group, with allocation not applicable and no masking. General statistical explanation: an uncontrolled study can describe outcomes observed among participants receiving the study approach. It cannot, by itself, separate the effect of the intervention from other influences, such as concurrent treatment, patient characteristics, or the natural course of illness. The regimen description includes chemotherapy selected at the treating physician’s discretion, which further means treatment exposure was not a single fixed regimen as described in the available arm text. A direct treatment contrast is not reported.

How should the sample-size planning material be understood?

Any sample-size figures associated with the posted material are planning illustrations, not observed power. The record’s stated illustration assumptions refer to a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation, with the target effect expressed as a fraction of the observed effect. For time-to-event outcomes, the stated planning quantity is the total number of events; other planning quantities are participants per arm. This trial itself is single-group, so those two-arm planning assumptions do not describe its design. No sample-size justification or trial-specific power calculation is reported in the registry record. Such planning illustrations do not establish that a study had a particular probability of detecting an effect.

What does the safety analysis report?

The safety record gives counts for serious events considered related to treatment, other adverse events, and deaths. General statistical explanation: counts describe the number recorded under the registry’s categories; they do not convey event timing, severity distribution, duration, or comparative risk unless those details are also reported. The registry lists CTCAE Version 4.0 for its toxicity outcome, but the displayed safety information does not include detailed event terms. The categories should therefore be read as limited reporting, not as a complete characterization of safety.

The record lists no analyses in its analysis count and does not report a detailed statistical analysis plan. Specific decisions about censoring, confidence-interval construction, missing observations, interim analyses, or adjustment for other factors are not reported in the registry record. Those omissions limit reproducibility and prevent a fuller assessment of how the planned methods were implemented.

10. Multiplicity, Missing Data and Analysis Populations

The registry lists several secondary outcomes in addition to median overall survival: biomarker analysis, toxicity, progression-free survival, quantitative immune response, and time to progression. It does not report a multiplicity strategy or explain whether any secondary outcomes were designated as confirmatory. General statistical explanation: when multiple outcomes are examined, the chance of finding at least one apparently notable result can increase unless the analysis plan accounts for the number and relationship of those tests. Without reported results and a stated strategy, statistical significance across multiple outcomes cannot be interpreted or compared.

Outcome reporting is incomplete for interpretation of the primary endpoint. The registry describes the planned Kaplan–Meier estimate and two analysis populations, but the numerical medians and 90% confidence intervals are not reported in the registry record. Event counts, censoring information, and detailed follow-up are also not reported. The registry does not provide numerical findings for the listed secondary outcomes.

The record does not explain how missing outcome data were handled, whether follow-up was incomplete, or how participants who did not complete all BAT infusions were treated in each analysis. General statistical explanation: missingness can affect estimates when the availability of an outcome is related to prognosis or treatment experience. An analysis of all registered patients and one restricted to treatment completers can also differ because completion is determined after enrollment.

The registry’s stated analysis populations are useful to identify, but they do not supply the absent results or missing-data methods. A detailed statistical plan, interim-analysis information, and sample-size justification are not reported in the registry record. No assumptions about those procedures should be inferred from the phase designation or the completed status.

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 0% reporting threshold.

Most frequent non-serious adverse event terms

The registry’s safety group is titled “Treatment (Anti-CD3 x Anti-EGFR BATs).” It records 2 participants at risk for serious adverse events, with 0 serious events listed as related to treatment. For other adverse events, 2 participants were at risk and 2 were affected. The record also lists 1 death. These are the posted counts, not rates or comparisons with a control group.

The serious-event and other-event fields are separate categories. The reported count of 0 treatment-related serious events does not mean that no serious events occurred under every possible definition, nor does it establish that the regimen has no serious risks. The record does not list the serious-event terms, the non-serious event terms, severity details, timing, or duration. It also does not state whether the death was related to treatment. Those details are not reported in the registry record.

General statistical explanation: safety counts in a group of this size provide little precision for estimating how frequently an event might occur in a broader population. They are best read as a record of what was reported for this trial, with the categories and attribution shown, rather than as a complete safety profile. The registry lists toxicity assessed using CTCAE Version 4.0 as a secondary outcome, but numerical toxicity results beyond these limited counts are not reported in the registry record.

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

The record establishes that a completed phase Ib/II, single-group study of anti-CD3 × anti-EGFR BATs in advanced pancreatic cancer enrolled 2 patients. It specifies median overall survival as the primary endpoint and describes a Kaplan–Meier method, including an intention-to-treat analysis and an analysis of patients who completed all BAT infusions. It also records limited adverse-event and death counts.

It does not report the numerical median overall survival, the 90% confidence interval, or numerical results for the secondary outcomes. Consequently, the posted record does not show how long participants survived, how precise a survival estimate was, or what the results were for progression-free survival, time to progression, immune response, or biomarker outcomes. The completed status does not fill those gaps.

Because the study had a single group and no randomized control, it cannot provide a direct estimate of how outcomes compare with a different treatment. The record’s chemotherapy description also indicates that standard regimens were selected at the treating physician’s discretion. Any outcomes observed in such a design would be descriptive of the treated group rather than a clean estimate of the contribution of BATs alone.

General statistical explanation: a small uncontrolled study can generate observations that may inform further study, but estimates from it are uncertain and vulnerable to the experience of individual participants. Without reported endpoint estimates and detailed follow-up, even a descriptive assessment of survival is not possible from the registry record. These limitations do not establish that the treatment is effective or ineffective; they define what can and cannot be concluded from the information recorded.

13. Why This Trial Matters Statistically

This trial illustrates why the design and the result must be read together. Median overall survival is a meaningful time-to-event endpoint, and the registry identifies Kaplan–Meier estimation and a 90% confidence interval as the planned approach. Yet the record does not include the numerical estimate or interval. Naming an endpoint and method is not equivalent to reporting an outcome.

The single-group structure matters because it sets a limit on causal interpretation. Without a randomized comparator, outcomes cannot be attributed to the study treatment through a direct between-group contrast. The physician’s discretion in selecting standard chemotherapy further emphasizes that the treatment context may vary. This is important when interpreting any descriptive observation from the trial.

Enrollment of 2 also highlights the role of precision. General statistical explanation: estimates based on very few participants can change substantially with the experience of an individual participant, and safety counts cannot establish a stable event frequency. Confidence intervals and complete event reporting are therefore central to interpreting small clinical studies. Here, the planned interval is described, but its values are not reported.

The record additionally demonstrates that analysis populations can change the question being answered. The intention-to-treat population includes all registered patients, while the response-evaluable population is limited to those completing all BAT infusions. Comparing such estimates would require the actual results and a careful account of completion and follow-up.

For clinical researchers and biostatisticians, the main statistical lesson is not a conclusion about treatment benefit. It is the need to distinguish design, planned methods, observed estimates, and uncertainty. The registry documents the first two more clearly than it documents the outcome results needed to judge the latter.

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 most direct limitation is that the primary endpoint result is not reported in the registry record. The median overall survival and its 90% confidence interval are absent, as are the endpoint event count and detailed follow-up. This prevents assessment of the estimate, its precision, and whether the stated median was estimable.

Enrollment was 2, and the design was single-group with no randomized control. The study therefore cannot support a comparative estimate of treatment effect. Its small enrollment also limits how much can be learned from the recorded safety counts. The registry does not report detailed adverse-event terms, severity, timing, or the relationship of the listed death to treatment.

The intervention description is not fully specified in the available arm text, which ends partway through a chemotherapy regimen description. The registry wording also differs between the short title, official title, and narrative regarding low-dose IL-1 versus IL-2 and the immune-stimulating agents. Those discrepancies should remain visible rather than be settled by assumption.

The record does not report detailed missing-data procedures, censoring information, a statistical analysis plan, a sample-size justification, or interim-analysis procedures. It also does not provide numerical results for the listed secondary endpoints. These omissions constrain both reproducibility and interpretation. The registry’s completed status and stated analysis methods do not substitute for these missing details.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: BATS With in Combination With Low Dose IL-1 and GM-CSF for Advanced Pancreatic Cancer — NCT02620865.
• Registry record last updated 2023-05-15; status last verified 2023-04.
• Results first posted 2023-05-15.
• 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.

18. Record Summary

NCT02620865 is recorded as a completed phase Ib/II, single-group study sponsored by Barbara Ann Karmanos Cancer Institute. The registry lists enrollment of 2 and advanced pancreatic cancer conditions. The treatment approach involved anti-CD3 × anti-EGFR bispecific antibody-armed activated T cells, with immune-stimulating agents and physician-selected standard chemotherapy described in the record.

The primary endpoint was median overall survival, with a planned 90% confidence interval estimated using Kaplan–Meier methods and an up-to-18-month timeframe. Numerical primary and secondary outcome results are not reported in the registry record. The safety fields list 0 treatment-related serious adverse events, 2 participants with other adverse events, and 1 death; details of event terms and death attribution are not reported.

The record describes study design and planned analysis populations, but it does not provide the outcome estimates needed to assess survival or comparative benefit. Its single-group design and enrollment of 2 are central limits on interpretation.