Pancreaze (Pancrelipase) for Patients With Pancreatic Adenocarcinoma With Cachexia and Exocrine Pancreatic Insufficiency: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Pancreaze (Pancrelipase) for Patients With Pancreatic Adenocarcinoma With Cachexia and Exocrine Pancreatic Insufficiency (NCT04098237), a na phase 2 study. The primary outcome is Feasibility of Completing Pancreatic Enzyme Replacement Therapy During the First 8 Weeks of the Study: Daily Compliance Diary.

2. Clinical Question

3. Trial Design

NCT04098237 is registered as a phase 2, single-group, open-label study. Allocation is listed as not applicable, and masking is listed as none. The intervention is Pancreaze (pancrelipase) added to standard of care for pancreatic adenocarcinoma with cachexia and exocrine pancreatic insufficiency. The specified regimen is 84,000 IU lipase units per main meal and 42,000 IU lipase units per snack for 24 weeks.

The registry record lists an enrollment of 36. Its brief summary says the study will treat 40 consecutive patients; these are different figures in the record, and it does not explain the difference. The listed start date is 2020-12-17, the primary completion date is 2024-01-12, and the status is active, not recruiting.

The primary outcome addresses diary-recorded adherence during the first 8 weeks. Secondary outcomes include changes in weight and calories consumed through the end-of-study visit, changes in stool frequency and consistency through Cycle 3 Day 1, and changes in serum levels of vitamins A, D, E, and K. The record lists 18 outcome measures overall. It does not describe the additional outcome measures in the information available for this study.

Because this is a single-group design, the study does not make a randomized comparison with a separate control group. Its results can describe outcomes among participants receiving the regimen, but the design alone cannot distinguish changes associated with the regimen from changes that could occur for other reasons.

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 Feasibility of Completing Pancreatic Enzyme Replacement Therapy During the First 8 Weeks of the Study: Daily Compliance Diary, measured over 8 weeks.

The primary endpoint is a count of participants meeting a diary-based adherence criterion during the first 8 weeks of the study. A participant meets the stated criterion by completing pancreatic enzyme replacement therapy at a level of at least 50% of the needed total lipase units. The daily compliance diary is the specified recording method.

This endpoint is used to assess feasibility: whether participants can follow the prescribed enzyme regimen sufficiently to meet the predefined adherence threshold over the specified period. Feasibility is distinct from a clinical outcome such as weight change or quality of life. A count meeting the adherence criterion would describe participation in the regimen, not establish that the regimen caused a change in symptoms or other outcomes.

The registry record gives the endpoint definition and timeframe but does not report the observed count or the number of participants included in its analysis. It also does not report how diary entries were handled when they were incomplete or absent, or how adherence was calculated when recorded use was unavailable. Without those details and the result, the endpoint cannot be interpreted as an observed feasibility estimate.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

General statistical context: A study endpoint translates a research question into a defined measurement. Here, the primary endpoint is a participant count based on a diary-recorded adherence threshold during the first 8 weeks. Counts answer how many participants met a criterion; they do not, on their own, describe the size of a change in weight, intake, or another continuous measure. The registry record does not report the primary count or an analysis of it.

What does the single-group design allow?

General statistical context: In a single-group study, outcomes can be described among participants who receive the intervention. If measurements are collected at baseline and later visits, a within-group change can also be described. That change is not the same as a comparison with an untreated or differently treated group. Without a concurrent comparison, changes over time cannot be separated from other influences with the same confidence as a randomized treatment contrast. This is a design limitation, not a calculation problem.

The registry lists allocation as not applicable, masking as none, and the model as single group. It does not report a separate control arm. The record therefore does not support interpreting any unreported within-group change as an estimate of the effect relative to an alternative treatment.

How should outcome estimates and uncertainty be read?

General statistical context: An estimate describes the observed value or change in a study population. A confidence interval, when reported, expresses uncertainty around an estimate under the assumptions of the statistical method. A narrow interval indicates greater precision than a wide interval, while neither interval width nor a point estimate resolves limitations in study design. A p-value addresses compatibility between data and a specified statistical model or null hypothesis; it does not measure clinical importance or the probability that a treatment works.

The registry record does not report confidence intervals, p-values, or analysis estimates for the primary endpoint or the listed secondary outcomes. No inference about statistical significance or precision can therefore be made from the outcome descriptions alone.

What do the secondary outcomes represent?

The listed secondary measures include mean changes in weight and calories consumed from baseline through the end-of-study visit; mean changes in stool frequency and stool consistency from baseline through Cycle 3 Day 1; and mean changes in serum levels of vitamins A, D, E, and K from baseline. These measures have different units and timeframes. A mean change summarizes participant-level changes, but interpretation also depends on how many participants contributed measurements and how missing observations were handled. Those analysis details and results are not reported in the registry record.

General statistical context: Continuous outcomes such as weight or calorie intake are often summarized by a mean and a measure of variability. Count outcomes, such as the primary adherence endpoint, are summarized as counts and may also be expressed as proportions if the denominator is clear. The type of outcome influences which statistical model is suitable. The registry record does not identify a model or provide analysis details for these outcomes.

What do the planning illustrations mean?

General statistical context: A sample-size illustration asks how many participants a hypothetical study might need under specified assumptions about an effect, significance level, allocation, and statistical method. Such an illustration depends on those assumptions; it is not a description of the number actually enrolled and does not show the probability that the completed trial detected an effect. The registry record’s stated illustration assumptions are a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation, with the effect to detect set as a stated fraction of the observed effect. Time-to-event calculations use a total number of events; other calculations are expressed as participants per arm.

These assumptions describe a planning illustration, not the design of this single-group study. The record does not report a trial-specific sample-size justification, the observed effect used for the illustration, or the resulting sample-size estimates. The illustration should not be interpreted as observed power or as evidence that the study had a particular ability to detect an effect.

General statistical context: Over-dispersion occurs when count data vary more than a chosen model expects. If a model assumes less variability than the data contain, uncertainty may be understated. Whether over-dispersion is relevant depends on the outcome and its distribution. The registry record does not report a model for the adherence count or information that would establish whether over-dispersion was assessed.

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("Standard of Care Treatment With Pancreaze (Pancrel")
n      <- c(30)
events <- c(29)
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  Standard of Care Treatment With Pancreaze (Pancrel  30  29  96.7  82.8  99.9  83.3  99.4  83.3  99.2

11. Multiplicity, Missing Data and Analysis Populations

The registry record lists 18 outcome measures and identifies a primary endpoint alongside secondary outcomes. Multiple outcomes create a multiplicity concern: when many hypotheses are tested, the chance of at least one apparently notable result can increase if each is assessed separately without an appropriate strategy. General approaches include identifying a primary outcome in advance, adjusting inference across a family of tests, or treating secondary findings as exploratory. Which approach was planned for this study is not reported in the registry record.

The record lists 0 analyses. It does not report estimates, hypothesis tests, confidence intervals, or a multiplicity procedure for the primary or secondary outcomes. The listed outcomes should therefore not be treated as evidence of positive or negative findings merely because they were specified.

Missing-data handling is also not reported in the registry record. For the diary-based primary endpoint, the record does not say how incomplete diaries, missed entries, or participants without sufficient follow-up were handled. For the secondary measures, it does not report the number of observations available at each visit, how missing measurements were addressed, or whether the analysis used all enrolled participants or a defined analysis population.

Those details matter because the participants with recorded outcomes may differ from those without them. The registry record does not describe assumptions about missingness, any imputation method, or sensitivity analyses. It also does not report analysis populations or whether participants were included according to a prespecified rule. Consequently, the completeness and representativeness of the outcome data cannot be assessed from the registry record.

12. Safety Results

The registry reports serious adverse events as affected participants divided by participants at risk.

Deaths

Other (non-serious) adverse events

Participants with at least one non-serious event at or above the registry's 5% reporting threshold.

Most frequent serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: other (6.7%), ascites (3.3%), atrial fibrillation (3.3%), biliary tract infection (3.3%), blood bilirubin increased (3.3%).

The registry record lists 30 participants at risk for both serious and other adverse events in the treatment group. It reports serious adverse events for 12 participants and other adverse events for 7 participants. The listed death count is 21. These are counts, not comparative estimates, because the record describes a single-group study without a separate control group.

The serious-event terms listed include Other at 6.7%, and Ascites, Atrial Fibrillation, Biliary Tract Infection, Blood Bilirubin Increased, and Colotis, each at 3.3%. These percentages are reported category values; the record does not supply enough detail to interpret them as mutually exclusive categories or to infer a causal relationship with Pancreaze. The spelling “Colotis” is retained as recorded.

The death count should be read as a reported safety figure, not as evidence that the intervention caused those deaths. The registry record does not report attribution, timing, or a comparison that would clarify the relationship between deaths and treatment. Counts of participants with events also do not indicate event severity beyond the serious-versus-other classification or describe recurrent events.

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

The registry record defines a feasibility question about diary-recorded adherence to pancreatic enzyme replacement therapy during the first 8 weeks. It also specifies a 24-week Pancreaze regimen added to standard of care and identifies nutritional, gastrointestinal, and vitamin measures as secondary outcomes. These details clarify what the study set out to assess.

However, the registry record does not report the observed primary endpoint count or results for the listed secondary outcomes. It therefore does not show how many participants met the adherence threshold, whether mean weight or calorie intake changed, or what happened to stool measures or vitamin levels. No estimates or uncertainty intervals are reported for those outcomes.

The single-group design also limits what any future within-group change could establish. Without a separate comparison group, change over time cannot be attributed to the intervention alone. The registry record does not report an inferential analysis that addresses this limitation.

The safety record lists event counts, including serious adverse events and deaths, but does not establish causation or provide a comparative safety estimate. The figures are important to describe what was recorded in the study group, while remaining insufficient by themselves to determine whether the intervention changed the risk of an event.

Accordingly, the posted information describes the trial’s question, design, outcomes, and selected safety counts; it does not establish a treatment benefit, lack of benefit, or causal safety effect. The missing outcome estimates and analysis details prevent assessment of the magnitude and precision of clinical changes.

14. Why This Trial Matters Statistically

This study is statistically notable because its primary question is feasibility rather than a direct treatment comparison. A diary-based adherence threshold can describe whether participants recorded use at a specified level, but it answers a different question from whether treatment changes weight, function, or quality of life. Keeping those questions distinct helps prevent an adherence result from being mistaken for a clinical outcome.

The study also illustrates the interpretive limits of a single-group design in a complex clinical setting. The listed population includes patients with borderline resectable, locally advanced, and advanced pancreatic adenocarcinoma, weight loss, and exocrine pancreatic insufficiency. Changes in measures over time could be relevant descriptions, but without a concurrent comparator their causes cannot be isolated by the design alone.

The outcomes span counts, mean changes, stool measures, and serum vitamin levels. Each measure has its own definition and timeframe, so the interpretation depends on having outcome-specific estimates, denominators, and uncertainty information. The record lists 18 outcome measures but does not report their analysis results. That makes transparent reporting of the planned analysis, missing-data approach, and multiplicity strategy especially important; these details are not reported in the registry record.

Finally, the record distinguishes planned treatment details from enrollment and safety counts. It lists enrollment of 36 while the brief summary states that 40 patients will be treated, and it reports safety counts for 30 participants at risk. Those quantities describe different aspects of the record and should not be treated as interchangeable. The registry record does not explain the differences.

15. Limitations

• The posted summary statistics are the only data these results rest on; patient-level data, full protocols and statistical analysis plans are not available.
• Recalculated results are simple, unadjusted calculations from posted summaries. They do not replace the sponsor's prespecified analysis.

The most immediate limitation is that the registry record does not report a result for the primary adherence endpoint. It also does not report results for the listed secondary outcomes, despite identifying 18 outcome measures overall. Without estimates and uncertainty information, the magnitude and precision of any outcome cannot be evaluated.

The single-group design has no separate concurrent comparison arm. Consequently, the study design cannot isolate intervention-associated change from other influences over time. The record lists masking as none and does not report a randomized comparison.

Enrollment is listed as 36, while the brief summary describes treating 40 consecutive patients. The registry record does not explain the discrepancy. Safety counts are listed for 30 participants at risk; the record does not explain how that denominator relates to enrollment or outcome populations.

Important analytic details are not reported in the registry record, including a trial-specific sample-size justification, statistical analysis plan, analysis populations, handling of missing diary entries or outcome measurements, and a strategy for multiple outcomes. The registry record also does not report how adverse events were attributed, their timing, or whether the listed category percentages refer to mutually exclusive events. These gaps constrain interpretation of both efficacy-related outcomes and safety counts.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Pancreaze (Pancrelipase) for Patients With Pancreatic Adenocarcinoma With Cachexia and Exocrine Pancreatic Insufficiency — NCT04098237.
• Registry record last updated 2026-01-23; status last verified 2026-01.
• Results first posted 2025-03-07.
• 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

NCT04098237 is a phase 2, open-label, single-group study of Pancreaze added to standard of care for pancreatic adenocarcinoma with cachexia and exocrine pancreatic insufficiency. The listed regimen is 84,000 IU lipase units per main meal and 42,000 IU lipase units per snack for 24 weeks. The primary outcome is a diary-based adherence count during the first 8 weeks.

The registry lists enrollment of 36, while the brief summary says 40 patients will be treated. Status is active, not recruiting. The record lists 18 outcome measures and 0 analyses. It reports safety counts among 30 participants at risk, including 12 with serious adverse events, 7 with other adverse events, and 21 deaths. The primary outcome result and secondary outcome results are not reported in the registry record.