QUILT-3.010: A Study of Gemcitabine and Nab-paclitaxel With or Without NPC-1C to Treat Patients With Pancreatic Cancer: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for QUILT-3.010: A Study of Gemcitabine and Nab-paclitaxel With or Without NPC-1C to Treat Patients With Pancreatic Cancer (NCT01834235), a randomized phase 1, phase 2 study. The primary outcome is Safety and Tolerability.

2. Clinical Question

This record covers a combined Phase I/II study, so its portions addressed different questions. Phase 1 evaluated safety of NPC-1C with gemcitabine using dose de-escalation. Phase 2 randomized patients between a three-drug regimen and gemcitabine plus nab-paclitaxel. The title emphasizes the addition of NPC-1C to the chemotherapy combination, while the primary-outcome description refers specifically to NPC-1C with gemcitabine.

The registry reports 81 participants enrolled. It posts Phase 1 outcome counts of 0.0 among 3.0 participants at dose level 1, and no participants at dose level -1. The two Phase 2 arms have posted counts of 3.0 among 38.0 participants for NPC-1C plus chemotherapy and 7.0 among 40.0 for chemotherapy alone. These are counts attached to the registered safety and tolerability outcome; the record does not define a specific event or threshold underlying the counts. They should therefore be read as registry-reported outcome counts, not as a complete description of all safety events.

3. Trial Design

The registry identifies this as a multicenter Phase I/II study with randomized allocation in its Phase 2 portion. Its design is listed as sequential and its masking as none. The record describes the study as prospective and open label. It is registered as NCT01834235 and has status terminated. The listed start is 2013-04, and primary completion is recorded as 2017-03-13.

Phase 1 was a dose-finding portion evaluating NEO-102, also called NPC-1C, with gemcitabine. The summary describes a dose de-escalation plan beginning at 1.5 mg/kg/dose and says the lower dose would be evaluated if 2 of 6 patients experienced dose-limiting toxicity. The record states that dose level -1 did not enroll any patients because dose level 1 was found to be safe with NEO-102 and gemcitabine, so dose de-escalation was not needed. The Phase 1 outcome data list 3.0 participants and a count of 0.0 at dose level 1.

In Phase 2, patients were randomized between NPC-1C with gemcitabine and nab-paclitaxel, and gemcitabine with nab-paclitaxel. The posted group sizes are 38.0 and 40.0, respectively. The chemotherapy descriptions specify nab-paclitaxel at 125 mg/m2 followed by gemcitabine at 1000 mg/m2, on 3 consecutive weeks followed by a week of rest. The description for the experimental arm says patients also received NPC-1C infusion, but it does not give a complete Phase 2 NPC-1C dosing schedule.

The study population had metastatic, locally advanced unresectable, or recurrent pancreatic cancer and prior FOLFIRINOX or a FOLFIRINOX-like regimen. The registry does not report additional eligibility detail, stratification factors, concealment procedures, or a separate analysis plan for the Phase 1 and Phase 2 portions.

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: Dose level -1 did not enroll any patients because dose level 1 was found to be safe when NEO-102 was given in combination with Gemcitabine. Hence there was no need to dose de-escalate.

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.

Population: NEO-102 Dose level 1 in combination with Gemcitabine was found to be safe and hence the NEO-102 dose did not need to be de-escalated to Dose level -1.

6. Primary Endpoint

The registry lists the primary outcome as Safety and Tolerability, measured over 28 days.

The primary outcome is named safety and tolerability, with a unit of participants and a timeframe of 28 days. Its stated purpose was to assess NPC-1C monoclonal antibody therapy combined with gemcitabine among patients with pancreatic cancer whose tumors bind NPC-1C and who failed or did not tolerate first-line FOLFIRINOX chemotherapy.

Safety outcomes are used in early clinical development to describe whether treatment-related harms or tolerability concerns occur, and to inform decisions about further evaluation and dose selection. In general, a count of participants can communicate how many people met an outcome definition, but its meaning depends on a clear definition of the qualifying event, the assessment period, and the population included. The registry record does not specify the event definition for the posted counts, nor does the primary-outcome description say whether these counts refer to dose-limiting toxicity, a particular adverse event, or another safety criterion. The 28-day timeframe is reported for the primary outcome, but the record does not explain how that period was applied to each Phase 2 arm.

The registered outcome lists a count of 0.0 among 3.0 participants at Phase 1 dose level 1. No participants were enrolled at dose level -1. The randomized-arm entries list counts of 3.0 among 38.0 participants for NPC-1C plus chemotherapy and 7.0 among 40.0 for chemotherapy alone. These figures are the posted outcome counts, not a detailed event-level safety analysis. They should not be treated as evidence about a specific adverse event unless the registry record defines that event, which it does not.

The registry also posts serious and other adverse-event summaries separately. Those data can describe safety reporting, but the record does not explicitly state that they are the same outcome as the primary safety and tolerability measure. Keeping the outcome count distinct from the adverse-event summary avoids assigning a meaning that the record does not establish.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The registry describes randomization in the Phase 2 portion, but posts no formal analysis results. The distinctions below are general statistical explanations, not claims about methods actually used in the trial. The registry record does not report a statistical analysis plan, so the intended models, testing procedures, and handling of missing information are not known.

What do the participant counts mean?

A participant count is a descriptive summary. It indicates how many participants were recorded as meeting a stated outcome, but interpretation depends on the outcome definition and the group in which it was assessed. The primary outcome is expressed as a count of participants over 28 days. Because the record does not define the event that qualifies a participant for that count, the reported 3.0 and 7.0 cannot be attached to a particular toxicity or threshold. Counts can be useful for an initial overview, but without definition and context they do not describe event severity, duration, or relationship to treatment.

Group sizes matter when interpreting counts because the same count can have different implications in groups of different sizes. A formal comparison would generally estimate a treatment contrast and its uncertainty using an approach suited to the outcome and study design. No such estimate or uncertainty interval is posted for this outcome. No new effect estimate should be inferred from the counts alone.

What does a confidence interval add?

In general, a confidence interval describes the range of effect estimates compatible with the observed data under a specified statistical procedure. It communicates precision as well as the estimated effect. A narrow interval indicates greater precision than a wide interval, although interpretation also depends on the chosen method and the clinical question. The registry does not post a confidence interval for the primary outcome or the secondary progression-free survival outcome. It is therefore not possible to assess comparative precision from the registered results.

Why does randomization matter?

In general, random assignment aims to make treatment groups comparable with respect to measured and unmeasured characteristics, on average. It supports a fair comparison when allocation is implemented as intended, outcomes are measured consistently, and analyses preserve the randomized comparison. The record identifies Phase 2 as randomized, but does not describe sequence generation, allocation concealment, stratification, or the population included in the analysis. It also identifies the study as open label, meaning masking was not used. Lack of masking can affect outcome assessment or reporting, particularly when outcome definitions involve judgment; the record does not describe procedures to mitigate that possibility.

The Phase 1 dose-finding portion is separate from the randomized Phase 2 comparison. It used a dose de-escalation approach, according to the summary, and dose level -1 did not enroll. Phase 1 observations should not be combined conceptually with the randomized-arm comparison: the treatment structure, purpose, and number of participants differ. The registry does not post a joint analysis across the phases.

What can a planning illustration tell a reader?

In general, sample-size planning asks how much information a future study might need to detect a specified effect under stated assumptions. Such a calculation depends on assumptions about the outcome, effect size, allocation, statistical test, significance level, and desired power. The record’s planning assumptions describe a two-sided significance level of 0.05, equal allocation, a normal approximation, and effects expressed as fractions of the observed effect. They do not establish that these assumptions were used to design this trial. Planning calculations are illustrations, not observed power and not evidence that a result is statistically significant. The registry does not report a sample-size justification or its own planned target for a formal comparative analysis.

For time-to-event outcomes, a general planning approach may focus on the number of events rather than only the number of participants, because event information drives precision. Progression-free survival is listed as a secondary outcome, but the registry does not provide its event count, analysis method, or results. No time-to-event conclusion can be drawn from the presence of that outcome alone.

Why can over-dispersion matter?

In general, a count outcome can vary more than a simple statistical model expects. This is called over-dispersion. If a model assumes less variability than is present, uncertainty estimates can be too small and tests can appear more decisive than warranted. Model choice should reflect the outcome’s distribution and how observations were collected. The registry record does not report a model for the participant counts, and the primary outcome is described as a count of participants rather than a count of recurrent events. There is therefore no basis to say that over-dispersion was assessed or that a particular count model was used.

Finally, posted safety summaries are descriptive unless accompanied by a stated comparative analysis. Differences in counts do not by themselves account for exposure time, event recurrence, censoring, or differences in assessment. The record does not report these details for the adverse-event summaries. A reader can describe the posted counts, but should not infer a statistical test, adjusted result, or causal effect from them.

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("Phase 1-Dose Finding Dose Level 1", "Arm A: Abraxane, Gemcitabine, NPC-1C", "Arm B: Abraxane, Gemcitabine")
n      <- c(3, 38, 40)
events <- c(0, 3, 7)
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  Phase 1-Dose Finding Dose Level 1  3  0  0.0  0.0  70.8  0.0  56.1  0.6  60.2
ARM  Arm A: Abraxane, Gemcitabine, NPC-1C  38  3  7.9  1.7  21.4  2.7  20.8  2.9  20.9
ARM  Arm B: Abraxane, Gemcitabine  40  7  17.5  7.3  32.8  8.7  31.9  8.8  32.1

11. Multiplicity, Missing Data and Analysis Populations

The registry record does not report how multiplicity was handled. Multiplicity arises when a study evaluates more than one outcome, comparison, subgroup, or analysis and the results are interpreted together. In general, repeated testing can increase the chance of finding an apparently unusual result by chance. Researchers may address this through a prespecified hierarchy, adjustment, or a clearly defined distinction between primary and exploratory analyses. The record names safety and tolerability as the primary outcome and lists progression-free survival as a secondary outcome, but does not describe a testing hierarchy or adjustment approach.

The record does not report how missing outcome data were handled. It does not describe whether participants who stopped treatment, withdrew, or missed assessments remained in an analysis, or how incomplete follow-up was treated. The analysis population is not reported in the registry record. Although Phase 2 is described as randomized, the record does not state whether the posted counts include all randomized participants or a different population.

For safety outcomes, missingness can matter if follow-up duration or event ascertainment differs between participants. The registry does not provide individual follow-up, exposure duration, or details about completeness of adverse-event reporting. For progression-free survival, it does not report censoring rules, assessment schedules, or a statistical analysis. These omissions prevent assessment of whether missingness or follow-up could have influenced results.

The registry lists no formal analyses. It therefore does not report an intention-to-treat analysis, a per-protocol analysis, or sensitivity analyses. These methods cannot be presumed from the randomized design. In general, transparent reporting of populations, missing-data methods, and multiplicity decisions helps readers distinguish planned confirmatory analyses from exploratory summaries; those details are not reported in 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: abdominal pain (13.2%), neutrophil count decreased (5.3%), deep vein thrombosis (5.0%), dehydration (2.6%), anemia (2.6%).

The adverse-event summary reports serious adverse events for 18 of 38 participants at risk in Arm A and 12 of 40 at risk in Arm B. Other adverse events are reported for 32 of 38 in Arm A and 33 of 40 in Arm B. These are participant counts in the registry’s adverse-event summary. The record does not provide a formal comparison, confidence intervals, or information about severity grades, duration, timing, recurrence, or attribution to study treatment. The counts should be read descriptively, not as proof that one regimen caused more or fewer events.

The record lists serious-event terms with percentages: Abdominal Pain, 13.2%; Neutrophil Count Decreased, 5.3%; Deep Vein Thrombosis, 5.0%; dehydration, 2.6%; Anemia, 2.6%; and Alanine aminotransferase increased, 2.6%. The registry does not identify the arm-specific denominators or explain the basis for these percentages. They should not be compared across arms without that context.

A separate deaths entry lists 38 for Arm A and 40 for Arm B, alongside at-risk counts of 38 and 40. The registry record does not clarify whether those entries denote actual deaths or another recorded value, so they cannot safely be interpreted as mortality outcomes. Serious adverse events are not synonymous with deaths, and the registry does not provide enough detail to resolve this ambiguity.

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

The posted results describe a terminated Phase I/II study that examined NPC-1C with chemotherapy in pancreatic cancer after prior FOLFIRINOX or a FOLFIRINOX-like regimen. They report a Phase 1 count of 0.0 at dose level 1 among 3.0 participants, no enrollment at dose level -1, and Phase 2 safety-and-tolerability counts of 3.0 and 7.0 in the two randomized arms. They also provide adverse-event participant counts. This is a descriptive account of the registry record, not a complete report of the trial’s clinical findings.

The record does not establish that NPC-1C improves outcomes or does not improve outcomes. Progression-free survival appears as a secondary outcome, but no analysis result is posted. The safety and tolerability outcome lacks a detailed event definition, and no comparative estimate, confidence interval, or p-value is reported. The adverse-event summaries do not include enough detail to assess treatment attribution, duration, exposure, or statistical uncertainty. The count differences should not be interpreted as evidence of treatment benefit or harm.

The Phase 1 statement that dose level 1 was found to be safe is recorded in the study description, but the posted Phase 1 outcome count is based on 3.0 participants. The record does not provide the dose-limiting-toxicity criteria or a detailed account of observed events to support broader conclusions about safety. The absence of participants at dose level -1 means the registry provides no participant outcome data at that dose level.

The status is terminated, but the reason for termination is not reported in the registry record. It is not possible to infer the reason from the status, the posted counts, or the lack of analysis results. The enrollment figure and group counts describe the registered record but do not establish how many participants were included in each analysis or how completely outcomes were assessed. More detailed results, analysis methods, and follow-up information would be needed to evaluate the comparative findings and their precision.

14. Why This Trial Matters Statistically

This trial is statistically notable for combining early dose finding with a randomized Phase 2 comparison. Those portions answer different questions: dose finding evaluates a regimen and dose in an early study context, whereas randomization is intended to support a comparison between treatment strategies. Separating the phases is important when interpreting the reported counts. The record identifies the Phase 1 dose level that enrolled participants and explains why the lower dose level did not proceed, while Phase 2 compares chemotherapy with and without NPC-1C.

The study also illustrates why outcome definitions are essential. A registered primary outcome of safety and tolerability, recorded as a participant count over 28 days, is not enough by itself to identify what event the count represents. Without a definition, detailed assessment rules, and an analysis method, even accurately posted counts may have limited interpretability. Safety summaries become more informative when they distinguish serious from non-serious events and describe severity, attribution, timing, and exposure.

The registry makes the open-label design and randomized allocation visible, but does not report key analysis details. Randomization supports a comparative design, yet the credibility and precision of an analysis also depend on how participants are followed, which population is analyzed, how outcomes are measured, and how missing data are handled. Those items are not reported in the registry record.

Finally, the record’s terminated status and lack of posted analyses highlight a distinction between trial registration and a complete results report. The registered design and available counts can help readers understand what was planned and what limited results were posted. They do not replace a full analysis with outcome definitions, uncertainty estimates, and transparent explanations of study completion and analysis decisions.

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.

Several record-specific limitations constrain interpretation. The primary outcome is labeled safety and tolerability, but the registry does not define the event counted in the posted participant totals. Although a 28-day timeframe is listed, the record does not explain how it was applied to the randomized arms or how participants with incomplete observation were handled. The listed outcome counts therefore cannot be mapped confidently to particular safety events.

The registry posts no formal analyses. It does not provide a comparative effect estimate, confidence interval, p-value, or adjusted analysis for the primary outcome. It lists progression-free survival as a secondary outcome but gives no analysis result. There is no reported statistical analysis plan, sample-size justification, multiplicity strategy, analysis population, or missing-data method.

The record identifies randomization and open-label masking, but does not report sequence generation, concealment, stratification, or safeguards for outcome assessment. It does not report eligibility criteria in full, baseline characteristics, treatment exposure, follow-up duration, or participant disposition by analysis population. These omissions prevent a thorough assessment of comparability, precision, and potential bias.

The adverse-event summary lacks event-level detail such as grades, duration, attribution, and timing. The percentages attached to serious-event terms have no stated arm-specific denominators. The deaths entry is not clearly explained relative to the at-risk counts. In addition, the registry does not report the reason for termination. These gaps mean that the posted record supports cautious description but not a full evaluation of comparative efficacy, safety, or why the study stopped.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: QUILT-3.010: A Study of Gemcitabine and Nab-paclitaxel With or Without NPC-1C to Treat Patients With Pancreatic Cancer — NCT01834235.
• Registry record last updated 2025-05-07; status last verified 2025-02.
• Results first posted 2025-05-07.
• 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

NCT01834235 is a terminated, open-label, multicenter Phase I/II study of NPC-1C with chemotherapy in pancreatic cancer after FOLFIRINOX or a FOLFIRINOX-like regimen. The Phase 1 portion evaluated NPC-1C with gemcitabine using dose de-escalation; dose level -1 did not enroll. The Phase 2 portion randomized participants to NPC-1C, gemcitabine, and nab-paclitaxel or gemcitabine and nab-paclitaxel.

The primary outcome is safety and tolerability, measured as participants over 28 days. The record posts participant counts for Phase 1 and both Phase 2 arms, along with separate adverse-event summaries. It does not define the event underlying the primary counts or post formal analysis results. Progression-free survival is listed as a secondary outcome without a posted analysis. The record does not report the reason for termination, analysis populations, missing-data methods, or a statistical analysis plan.