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 Two Stage Study Of Single Dose PEGPH20 And Cetuximab In Patients With Pancreatic Adenocarcinoma Prior To Surgical Resection (NCT02241187), a na na study. The primary outcome is Effects of PEGPH20.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02241187 |
| Phase | NA |
| Condition | Pancreatic Cancer |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 2 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Memorial Sloan Kettering Cancer Center |
| Results posted | Yes |
2. Clinical Question
Population
The intended population was people with resectable pancreatic adenocarcinoma before surgical resection. The registry reports a crucial difference between that intended population and actual accrual: the two participants accrued were healthy participants, and no participants were accrued for treatment.
Intervention
The study description concerns cetuximab before surgery and, in its second stage, cetuximab with PEGPH20. It also describes DW- and DCE-MRI, blood draws, and examination of resected tumors. The registry does not report that either drug was administered to participants in the study.
Comparator
This was a single-group study, with allocation recorded as not applicable and no masking. It therefore does not describe a randomized or like-for-like control comparison. The record does not report a separate comparator group.
Primary question
The stated primary question concerned effects of PEGPH20 on resectable pancreatic adenocarcinoma tumors. The planned assessment involved MRI measures of tumor permeability, cetuximab distribution, and examination of resected tumors for evidence of stromal degradation. The registry reports no treatment accrual and no collected data, so it contains no observed answer to that question.
3. Trial Design
The registry describes a two-stage study of cetuximab, with PEGPH20 included in the second stage, in patients with pancreatic adenocarcinoma before surgical resection. Its aims included assessing safety, cetuximab delivery to tumors, and tumor characteristics using imaging and examination of resected tissue. The primary outcome description gives a timeframe of 1 year. These entries describe the study’s intended questions; they do not establish that the planned treatment or assessments were carried out.
The design is recorded as single group, with allocation and phase listed as not applicable and masking listed as none. No randomized control group or masked comparison is described. The arm description refers to MRI sequence optimization and repeatability work, and mentions five participants, while the enrollment field reports two participants. The primary outcome population note clarifies that the two accrued participants were healthy and that no participants were accrued for treatment. No data were collected.
The registry status is completed. The listed start date is 2014-09-12 and the primary completion date is 2016-09-13. Those dates describe the record’s timeline; they do not change the distinction between the intended study and the reported accrual and data collection. The registry record does not report a sample-size justification, a detailed statistical analysis plan, or a rationale for the actual enrollment. Without treatment participants and collected outcome data, the proposed single-group design cannot yield an estimate of the effects of treatment on pancreatic tumors.
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 | PEGPH20 And Cetuximab |
|---|---|
| STARTED | 2 |
| COMPLETED | 0 |
| NOT COMPLETED | 2 |
Reasons for not completing this period:
| Reason | PEGPH20 And Cetuximab |
|---|---|
| Protocol closed due to lack of accrual. | 2 |
Analysis population for the primary outcome, as defined in the registry: The two participants accrued to this study were healthy participants. No participants were accrued on study for treatment. No data were collected.
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 | PEGPH20 And Cetuximab |
|---|---|---|
| Participants analysed | 2 | |
| Age, Continuous | years | 64 (61 to 67) |
| Sex: Female, Male: Female | Participants | 1 |
| Sex: Female, Male: Male | Participants | 1 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 1 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 1 |
| 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 | 0 |
| Race (NIH/OMB): White | Participants | 2 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Region of Enrollment: United States | Participants | 2 |
6. Primary Endpoint
The registry lists the primary outcome as Effects of PEGPH20, measured over 1 year.
| Element | Registry entry |
|---|---|
| Result type | N/A |
| Measure of spread | not applicable |
| Unit | not stated |
| Endpoint classification | not reduced to a single comparison |
The primary outcome is titled “Effects of PEGPH20.” Its description concerns PEGPH20 administration and resectable pancreatic adenocarcinoma tumors. The intended assessments include DW- and DCE-MRI to study tumor permeability to small and larger molecules, respectively, and cetuximab distribution. Resected tumors were to be examined for evidence of stromal degradation. The stated timeframe is 1 year.
These measures were intended to characterize biological and imaging changes in tumor tissue, rather than to report a clinical comparison between treatment groups. In general, imaging measures can be used to investigate properties such as permeability, while tissue examination can assess structural features. Such measures are meaningful only when the planned assessments are performed and their results collected and analyzed.
The registry record reports that no participants were accrued for treatment and no data were collected. It therefore provides no endpoint values, estimates, or uncertainty measures. The outcome description establishes what the study intended to examine, but it cannot be interpreted as evidence that PEGPH20 changed permeability, cetuximab distribution, or tumor stroma.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The central interpretive fact is that the registry reports two participants accrued, identifies them as healthy participants, and states that no participants were accrued on study for treatment. It also states that no data were collected. Consequently, the posted record does not contain an observed treatment outcome for PEGPH20, cetuximab, MRI, or tumor tissue assessment.
The primary outcome describes effects of PEGPH20 on resectable pancreatic adenocarcinoma tumors, assessed through imaging, cetuximab distribution, and examination of resected tumors. Those are intended measurements, not reported results. There is no reported estimate of tumor permeability, no reported distribution measure for cetuximab, and no reported tissue finding about stromal degradation. The record also provides no treatment outcome estimate or confidence interval from which to judge precision.
The listed secondary outcome concerns safety of administering PEGPH20 and cetuximab. The adverse-event record gives zero participants at risk and zero events in its listed categories. Because the record says no participants entered treatment, these zero counts should not be read as evidence that either drug was safe or that adverse events did not occur among treated participants. They reflect an absence of treated participants in the reported record, not an evaluable safety experience.
This is not a comparison of treatment with a control. The study is classified as single group, allocation is not applicable, and the record does not describe a comparator arm. Accordingly, no relative treatment effect can be estimated. Nor can the planned two-stage structure be treated as evidence that both stages enrolled or generated results: the actual accrual and data-collection note is explicit that there was no treatment accrual and no data collection.
In general, an estimate is informative only in relation to the data on which it is based. When no outcome data are collected, an effect estimate, its precision, and its clinical interpretation are unavailable. The registry’s completed status does not supply those missing results. The appropriate reading is that the record documents a proposed research question and design, alongside actual accrual information that leaves the primary and secondary questions unanswered.
9. Statistical Methods Explained
The record describes a single-group study, not a randomized comparison. Allocation is listed as not applicable, and masking is listed as none. This means the registry does not describe assignment between treatment and control groups or masking of participants or study personnel. The title and study description refer to treatment before surgery, but the population note reports that the two people accrued were healthy participants and that no one was accrued for treatment.
What does a single-group design estimate?
In general, a single-group study can describe outcomes among participants who receive an intervention, if those participants are treated and outcomes are measured. Without a concurrent comparator, it does not by itself separate an intervention’s effects from other influences, such as the course of the underlying condition or changes over time. Here, the registry reports no treatment accrual and no collected data, so even a descriptive treated-group outcome is unavailable. The record cannot support an estimate of treatment effect.
What were the planned measurements intended to do?
The primary outcome description links DW- and DCE-MRI to assessment of tumor permeability to small and larger molecules, respectively, and identifies cetuximab distribution as another subject of study. It also proposes careful study of resected tumors for evidence of stromal degradation. In general, imaging and tissue measures can provide different kinds of information: imaging can characterize features of a tumor in vivo, while tissue assessment examines material removed at surgery. These are general roles for such measurements, not findings from this study. The registry reports no measurements or collected results.
The arm description also mentions MRI sequence parameter optimization and repeatability investigation, including optional repeat imaging and T1 mapping. In general, sequence optimization concerns how an imaging acquisition is configured, while repeatability work examines whether a measure is consistent when an assessment is repeated. The description does not establish that these procedures were completed. The registry’s actual accrual and data-collection note remains that no treatment participants were accrued and no data were collected.
What would an estimate and confidence interval mean?
In general, a statistical estimate summarizes a feature of observed data, while a confidence interval expresses uncertainty around an estimate under the assumptions of the analysis. A narrow interval is generally more precise than a wide interval, but precision does not resolve limitations in study design or measurement. No effect estimate or confidence interval is reported for the primary outcome in this record. With no collected outcome data, there is no study-specific estimate whose precision can be assessed.
Why do sample-size calculations need careful interpretation?
In general, a sample-size calculation depends on assumptions about the outcome, the effect to detect, variability, significance level, allocation, and analysis method. A planning calculation is not evidence that a study enrolled the required number of participants, and it is not an observed measure of power. The registry record does not report the study’s own sample-size justification. Any sample-size illustration should therefore be understood as a hypothetical planning exercise, not as a result from the participants enrolled in this study. In particular, an illustration cannot replace missing outcome data or create an observed treatment effect.
What can the safety counts establish?
The secondary outcome concerns safety of PEGPH20 and cetuximab. The adverse-event entry lists zero participants at risk and zero events in its categories. In general, event counts are interpretable only in relation to participants who were exposed, observed, and included in the safety assessment. Since the record reports no treatment accrual, these entries do not provide an evaluable estimate of safety among treated participants. They should not be interpreted as confirming an absence of treatment-related events.
The registry reports zero analyses. It does not report model specifications, covariate adjustment, handling of repeated imaging, or inferential procedures for the intended tumor outcomes. Those details are not reported in the registry record. General statistical methods can explain how such analyses might be approached, but they cannot be attributed to this study without a reported analysis plan or results.
10. Multiplicity, Missing Data and Analysis Populations
The study description lists several aims: imaging the tumor, assessing safety before surgery, studying cetuximab delivery, and, in the second stage, examining safety and tumor effects of PEGPH20 with cetuximab. The primary outcome description includes multiple measurement domains, including MRI, drug distribution, and examination of resected tumors. The registry also lists a secondary safety outcome. These aims raise the general issue of multiplicity: when several outcomes or analyses are considered, the chance of at least one apparently notable result can increase unless the analysis strategy addresses that issue.
The registry record does not report a multiplicity adjustment, a hierarchy among outcome measures, or a detailed plan for selecting which measurements would be treated as confirmatory. It also reports zero analyses and no collected data, so there are no reported outcome tests whose multiplicity can be evaluated. No significance results or confidence intervals are given for the intended outcomes.
The record does not report how missing outcome observations would have been handled, or define analysis populations for an efficacy or safety analysis. More fundamentally, it states that no participants were accrued for treatment and no data were collected. This is not a case where a reported treatment analysis can be assessed for incomplete measurements: the record contains no treatment outcome dataset to analyze. The two healthy participants reported as accrued do not constitute an observed treatment population for the tumor question.
In general, analysis-population definitions and missing-data methods matter because they clarify who contributes to each result and how incomplete observations are addressed. Those methods are not reported in the registry record. Their absence should be distinguished from a claim that a particular method was or was not used outside the record.
11. What the Result Does, and Does Not, Mean
The record is meaningful as documentation of an intended study question and its actual accrual status. It describes a proposed investigation of PEGPH20 and cetuximab before surgery for pancreatic adenocarcinoma, with imaging, safety assessment, drug-distribution measures, and examination of resected tumors. It also records that two healthy participants were accrued, no participants were accrued for treatment, and no data were collected.
Those details do not establish an effect on tumors, a change in MRI measures, altered cetuximab distribution, or evidence of stromal degradation. No primary outcome estimate is reported. The safety outcome likewise cannot establish whether treatment caused adverse events or whether treatment was safe, because the registry reports no treated participants. Zero adverse-event counts alongside zero participants at risk are not a treated safety result.
The single-group classification and absence of a reported comparator mean that the study description does not support a randomized treatment comparison. More importantly, the lack of treatment accrual and data means no treatment-group estimate is available at all. The completed registry status is not itself a result and should not be mistaken for completion of the intended treatment assessments.
In general, a study’s scientific question, its actual conduct, and its reported results are distinct. The study description states what investigators intended to learn; the accrual and outcome record indicates what can be interpreted. In this case, the record can inform readers about the gap between the intended pancreatic-cancer treatment study and the reported actual experience. It cannot answer whether either drug affected the intended tumor or safety outcomes.
12. Why This Trial Matters Statistically
This record matters statistically because it illustrates why planned outcomes and observed evidence must be kept separate. The study description sets out a biologically focused question involving tumor permeability, cetuximab distribution, MRI, and tissue findings. Yet the registry’s population note says that the two accrued participants were healthy, that no participants entered treatment, and that no data were collected. The planned scientific question therefore has no reported outcome estimate in this record.
It also shows why a zero event count must be read alongside its denominator. The adverse-event entry lists zero participants at risk as well as zero events. In general, counts without an exposed and observed population do not quantify treatment safety. The record should not be summarized as showing an absence of adverse events among treated participants.
Finally, the single-group classification is important when considering what a study could establish even if outcome data were available. Without a control comparison, causal interpretation would require caution. Here, however, the more basic limitation is that treatment outcomes were not collected. Statistical methods cannot recover an effect estimate, precision, or comparison when the underlying data are absent. Clear reporting of this distinction helps prevent intended measurements, administrative completion, and actual evidence from being conflated.
13. 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 the registry’s statement that no participants were accrued for treatment and no data were collected. The two participants recorded as accrued were healthy participants, not the intended population of patients with pancreatic adenocarcinoma receiving pre-surgical treatment. Thus, the primary tumor question and the secondary safety question have no reported treatment outcomes.
The record describes a single-group study with no allocation to a comparator and no masking. It does not report a control group, an inferential analysis plan, a sample-size justification, or methods for handling missing data. It lists zero analyses. These omissions prevent assessment of how the intended outcomes would have been analyzed, and no outcome estimates are available for independent interpretation.
The arm description refers to MRI optimization and repeatability activities and mentions five participants, whereas the enrollment field reports two and the outcome population note specifies two healthy participants. The registry does not explain this difference. The actual enrollment and outcome note should not be overridden by the arm description when characterizing the reported treatment experience.
The adverse-event counts are also limited by zero participants at risk. They do not provide a usable safety denominator for treated participants. The completed status and dates recorded for the study do not resolve the absence of treatment data or explain why the intended treatment population was not accrued. Further reasons or conduct details are not reported in the registry record.
14. Related Tutorials
15. Related Statistical Calculators
16. Sources
- ClinicalTrials.gov: Two Stage Study Of Single Dose PEGPH20 And Cetuximab In Patients With Pancreatic Adenocarcinoma Prior To Surgical Resection — NCT02241187.
- Registry record last updated 2017-09-27; status last verified 2017-02.
- Results first posted 2017-09-27.
- 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.
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17. Record Summary
NCT02241187 is a completed, single-group study record concerning PEGPH20 and cetuximab before surgical resection for pancreatic adenocarcinoma. Allocation and phase are listed as not applicable, and masking is listed as none. The study description includes MRI, safety, cetuximab distribution, and tumor-tissue aims.
The registry reports enrollment of two healthy participants, no participants accrued for treatment, and no data collected. The primary outcome concerns effects of PEGPH20 on tumors, with a stated timeframe of 1 year; the secondary outcome concerns safety of PEGPH20 and cetuximab. No analyses are reported, and the record contains no interpretable treatment-effect or treated-safety result.