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 Trial of IRE in Cholangiocarcinoma (NCT03769753), a na phase 2 study. The primary outcome is Number of Patients Experiencing a Clinically Relevant Complications.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT03769753 |
| Phase | Phase 2 |
| Condition | Cholangiocarcinoma |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 8 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Memorial Sloan Kettering Cancer Center |
| Results posted | Yes |
2. Clinical Question
Population
The registry describes cholangiocarcinoma and an arm of patients with intraoperatively determined advanced unresectable PHC. The acronym PHC is not expanded in the registry record. The overall enrollment is listed as 8, while the arm description gives N=20; the record does not explain this inconsistency.
Intervention
The intervention is an IRE device used during surgical exploration. The description says electrodes are placed with ultrasound guidance by hepatopancreatobiliary surgeons experienced in IRE, during the same exploration session.
Comparator
No comparator arm is reported. The study is listed as single-group, with allocation marked NA and masking listed as NONE. The registry therefore does not provide a like-for-like control comparison.
Primary question
The primary endpoint asks how many participants experience a clinically relevant complication, defined as a CTCAE version 5.0 grade 3 or higher complication within 30 days post-IRE. The numerical result for that endpoint is not reported in the registry record.
In practical terms, the stated primary question is about complications after IRE in this described surgical setting, rather than a comparative estimate of benefit against another treatment. The record lists one outcome measure and no analyses. It does not report an efficacy endpoint, a comparison group, or an estimated treatment effect. The posted adverse-event counts are separate registry safety figures and should not be treated as the missing result for the primary complication endpoint.
3. Trial Design
The registry identifies NCT03769753 as a phase II study and lists its status as COMPLETED. Its design is single-group, allocation is NA, and masking is NONE. These features describe a nonrandomized study without a reported control arm. The intervention is an IRE device, used intraoperatively during surgical exploration for patients described as having advanced unresectable PHC. The record does not expand PHC.
The arm description says that IRE is delivered during the same surgical exploration session in which unresectability is determined. It specifies ultrasound guidance for electrode placement and says that hepatopancreatobiliary surgeons experienced in IRE place the electrodes. These details describe the intervention in the registry; additional procedural details are not reported in the registry record.
Enrollment is listed as 8, but the arm description states N=20. This is an internal discrepancy that the registry record does not resolve. The two figures should not be reconciled by assumption, and neither should be used to infer an analysis population. The record lists a start date of 2018-12-05 and a primary completion date of 2021-08-16.
The registry lists one outcome measure and zero analyses. A statistical analysis plan, methods for handling missing observations, and a participant flow accounting are not reported in the registry record. The design supports a descriptive account of outcomes in the studied group, but the absence of a control group means it does not offer a direct between-group treatment comparison.
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 | Intraoperative Use of IRE |
|---|---|
| STARTED | 8 |
| COMPLETED | 1 |
| NOT COMPLETED | 7 |
Reasons for not completing this period:
| Reason | Intraoperative Use of IRE |
|---|---|
| Not Treated | 6 |
| Inevaluable - No Evidence of Disease | 1 |
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 | Intraoperative Use of IRE |
|---|---|---|
| Participants analysed | 8 | |
| Age, Continuous | years | 72 (64 to 78) |
| Sex: Female, Male: Female | Participants | 2 |
| Sex: Female, Male: Male | Participants | 6 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 0 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 8 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 |
| Race (NIH/OMB): Asian | Participants | 1 |
| 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 | 6 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Region of Enrollment: United States | Participants | 8 |
6. Primary Endpoint
The registry lists the primary outcome as Number of Patients Experiencing a Clinically Relevant Complications, measured over within 30 days post-IRE.
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is the number of participants experiencing clinically relevant complications within 30 days post-IRE. The registry defines a clinically relevant complication as a CTCAE version 5.0 grade 3 or higher complication. It is a count of participants with an event, rather than a continuous measure such as a laboratory value or a time-to-event estimate.
In general, a threshold based on event severity can focus attention on complications considered more serious than minor symptoms or findings. A participant count also gives a direct description of how many people in the study met the defined endpoint. That count is most interpretable when the eligible population, follow-up, event ascertainment, and handling of incomplete observations are clear.
The registry record does not report the observed count for this primary endpoint, its denominator as analyzed, or an uncertainty interval. The separate adverse-event summary lists serious and other adverse-event counts, but the registry does not establish that those counts are equivalent to the primary endpoint definition. They should therefore be read as safety figures, not substituted for the primary endpoint result.
The endpoint definition specifies a follow-up window and a severity threshold, but the record does not report further details about adjudication, attribution to IRE, or how complications were counted if a participant experienced more than one. Those details matter when interpreting event summaries and are not reported in the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The record describes a phase II, single-group study with an intraoperative IRE intervention. Its primary outcome is a count of participants with a CTCAE version 5.0 grade 3 or higher complication within 30 days post-IRE. However, the numerical result for that endpoint is not reported in the registry record. Without that result, the primary question cannot be answered from the posted information.
The registry does post safety figures for the Intraoperative Use of IRE group: serious adverse events affected 0 participants among 8 at risk, other adverse events affected 1 participant among 8 at risk, and the death count is 2. These are descriptive counts. The record does not report causes, timing, attribution, event terms for serious events, or whether the deaths were related to the procedure or intervention. It also does not clarify how these safety categories relate to the primary complication definition. No event rates or comparisons should be inferred from the counts alone.
Because no comparator is reported, the study cannot estimate how outcomes differ from those under another treatment or no IRE. A single-group observation can describe outcomes among participants treated in the study, but it cannot isolate the effect of IRE from other clinical factors, selection into treatment, or the surgical setting. The posted record does not report a causal analysis.
The discrepancy between enrollment of 8 and the arm description's N=20 further limits interpretation of the population represented by the posted figures. The record does not explain whether these refer to different stages, populations, or reporting conventions. It would be inappropriate to choose one as the definitive analyzed sample size.
No confidence interval, p-value, adjusted estimate, or analysis method is reported; the record lists zero analyses. A confidence interval would ordinarily communicate the precision of an estimate, while a p-value would address compatibility with a specified statistical model and null hypothesis. Neither can be interpreted for the primary endpoint from the registry information. The planning assumptions described in the record are illustrations and should not be mistaken for an observed power calculation or a trial-design justification.
Overall, the posted material documents the design and limited safety counts but does not report the primary endpoint result or a comparative effect. The absence of these results is a limitation of the registry information, not evidence that the endpoint did or did not occur.
9. Statistical Methods Explained
The registry describes the study as single-group, with allocation marked NA and masking listed as NONE. In general, a single-group design records outcomes in participants receiving the intervention without a concurrent control arm. It can describe what was observed in that group, but it does not by itself distinguish effects of the intervention from background risk, the underlying disease, or other aspects of care. Without a comparator, a direct treatment contrast is unavailable.
What does the endpoint count?
The primary endpoint is a binary participant-level outcome summarized as a count: whether a participant experienced at least one clinically relevant complication under the stated definition. The registry defines that event as a CTCAE version 5.0 grade 3 or higher complication within 30 days post-IRE. For a binary endpoint, each participant is classified according to whether the event occurred under the definition. A count describes the number classified with the event, while an event proportion would also require a clear analyzed denominator. The registry record does not report the primary endpoint count or its analyzed denominator.
For this kind of outcome, statistical uncertainty depends on both the observed count and the population represented by the analysis. A confidence interval is a range generated by a statistical procedure to express uncertainty around an estimate. It does not guarantee that the true value lies within the particular interval, and its interpretation depends on the method and assumptions used. The registry record does not report an interval or the method used to calculate one.
Why does the design matter?
In general, a randomized comparison can help balance prognostic characteristics between treatment groups, while a single-group study does not have that design feature. Here, allocation is NA and the model is SINGLE GROUP, so the registry does not describe random assignment or a control group. Consequently, a difference between this group's outcome and an external benchmark would not automatically represent an intervention effect. Differences in participant selection, disease status, surgery, follow-up, and outcome recording could also matter.
The record identifies the intervention as an IRE device used during surgical exploration and describes electrode placement under ultrasound guidance by experienced hepatopancreatobiliary surgeons. This describes the intended setting, but it does not supply a statistical method for adjusting for differences in participants or care. No adjustment strategy is reported in the registry record.
What is known about analysis?
The registry lists one outcome measure and zero analyses. It does not report a model, an estimation procedure, a hypothesis test, a confidence interval method, or a prespecified analysis population. It also does not report how missing observations were handled. These omissions mean that statistical methods cannot be reconstructed from the record, and no unreported analysis should be inferred from the safety counts.
The sample-size assumptions in the record describe a planning illustration based on an observed effect: a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation, with an effect defined as a stated fraction of the observed effect. The record distinguishes participants per arm for ordinary outcomes from total events for time-to-event outcomes. These assumptions are not a description of the actual single-group trial design and do not establish that the trial used those methods. They are not observed power. The record does not report the trial's own sample-size justification, and the planning illustration cannot resolve the enrollment inconsistency.
Why should over-dispersion be considered only when relevant?
In general, over-dispersion means that observed variation is greater than a particular statistical model expects. It can matter for outcomes modeled as counts, especially when a model assumes a specific relationship between the mean and variability. The primary endpoint is defined as a participant count for a binary event, and no count model or variance assumption is reported. There is no basis in the registry record to claim that over-dispersion was assessed or that a particular method was needed to address it.
For a binary outcome, methods may differ in how they estimate an event probability and its uncertainty, particularly when observations are sparse. The appropriate method depends on the analysis question and assumptions. The registry does not report a chosen method, so it is not possible to say whether an exact, approximate, or model-based approach was used. No estimate or uncertainty interval for the primary endpoint is posted.
How should planning calculations be read?
In general, sample-size calculations are conditional on assumptions about the effect to detect, variability, significance level, power, allocation, and analysis method. A planning illustration describes what might be required under its stated assumptions; it does not establish what was observed in a completed study. The record explicitly labels its assumptions as a planning illustration, not the trial's own design. Because no calculated sample-size values or observed primary endpoint effect are included in the registry record, no further planning interpretation is warranted.
Finally, the recorded safety counts are descriptive and do not replace an analysis of the defined primary endpoint. The serious-event count, other-event count, and death count have different labels, and the record does not give enough detail to map them onto the primary endpoint's severity and follow-up criteria. Statistical interpretation should preserve those distinctions rather than treating all reported counts as interchangeable.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists one outcome measure, but it does not report a multiplicity strategy. In general, multiplicity arises when multiple outcomes, treatment contrasts, subgroups, or repeated tests are examined; the chance of at least one false-positive result can increase when many hypotheses are tested without an appropriate plan. The record lists zero analyses, so there is no reported set of inferential tests or adjustment procedure to evaluate. It is not possible to determine from the registry whether additional analyses were conducted elsewhere.
Missing-data methods are not reported in the registry record. The posted information does not state how participants with incomplete follow-up, unavailable complication assessments, or other missing endpoint information were handled. In general, the handling of missing observations can affect both the denominator and interpretation of an event estimate, particularly if missingness is related to outcome status.
The analysis population is also not reported. The enrollment figure is 8, while the arm description gives N=20, and the record does not reconcile these values or state which population was used for outcome analysis. The safety figures give 8 at risk for each listed adverse-event category, but they do not identify an analysis population for the primary endpoint. These differences should remain unresolved rather than being combined or interpreted as a participant flow.
No protocol deviations, exclusions, or participant disposition details are reported in the registry record. Accordingly, the posted information does not establish whether the primary endpoint was analyzed in all enrolled participants or in another defined group.
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 |
|---|---|---|---|
| Intraoperative Use of IRE | 0 | 8 | 0.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Intraoperative Use of IRE | 2 of 8 |
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 |
|---|---|
| Intraoperative Use of IRE | 1 of 8 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Intraoperative Use of IRE |
|---|---|---|
| Alkaline phosphatase increased | Investigations | 1 of 8 |
| Anemia | Blood and lymphatic system disorders | 1 of 8 |
| Aspartate aminotransferase increased | Investigations | 1 of 8 |
| Blood bilirubin increased | Investigations | 1 of 8 |
| Lymphocyte count decreased | Investigations | 1 of 8 |
The registry lists the Intraoperative Use of IRE group with serious adverse events affecting 0 participants among 8 at risk and other adverse events affecting 1 participant among 8 at risk. The listed death count is 2. These are the posted safety counts and should be read as descriptive entries, not as comparative estimates.
The record does not identify serious adverse-event terms; no top serious terms are listed. It also does not report the timing, causes, severity details beyond the serious/other categories, or relationship of the deaths to IRE or surgery. The death count should not be assumed to represent treatment-related deaths. The record does not explain whether deaths overlap with the adverse-event counts.
These categories cannot be substituted for the primary endpoint. That endpoint is specifically a count of participants with a CTCAE version 5.0 grade 3 or higher complication within 30 days post-IRE, while the posted safety summary uses serious and other adverse-event categories. The registry does not establish that the definitions, follow-up, or participant sets are the same. No percentage or event rate is needed to interpret the raw counts, and none can resolve these definition and reporting limitations.
12. What the Result Does, and Does Not, Mean
The registry record establishes that the trial was a phase II, single-group study of intraoperative IRE in cholangiocarcinoma, with an endpoint defined around clinically relevant complications within 30 days post-IRE. It also provides a limited adverse-event summary. Those details describe the design and some safety reporting, but they do not provide the numerical result for the primary endpoint.
The safety entries report 0 participants with serious adverse events among 8 at risk, 1 participant with other adverse events among 8 at risk, and 2 deaths. These counts do not establish why events or deaths occurred, whether they were related to the intervention, or how they correspond to the primary endpoint. They should not be interpreted as proof of safety or as evidence about efficacy.
The study has no reported comparator. Therefore, the registry cannot support a direct comparison with another treatment or a causal estimate of the effect of IRE. A single-group result, even if complete, would describe outcomes in the observed group under the reported conditions; it would not show what would have happened to comparable participants under a different approach.
The record also contains an unresolved enrollment discrepancy: enrollment is 8, whereas the arm description states N=20. The record does not explain the difference. It reports zero analyses and no primary endpoint result, confidence interval, or p-value. The stated planning assumptions are illustrations, not evidence of observed power and not a substitute for results.
Accordingly, the posted information is insufficient to determine the frequency of the defined primary complication endpoint or to make a comparative claim. This is a statement about what the registry record reports, not a conclusion that the outcome did or did not occur.
13. Why This Trial Matters Statistically
This trial is statistically notable because its record combines a clearly defined safety-oriented primary endpoint with a single-group design and incomplete outcome reporting. A precise endpoint definition matters: the registry specifies a participant count, a CTCAE severity threshold, and follow-up within 30 days post-IRE. Those elements identify what the primary outcome is intended to capture. Yet the numerical result for that endpoint is not reported in the registry record, so the key descriptive quantity cannot be assessed.
The absence of a comparator is also central. Single-group studies can document outcomes in a treated population, but they do not provide a direct estimate of how outcomes differ under an alternative. This distinction is important when readers encounter safety counts or planning illustrations: neither supplies a comparative treatment effect.
The record's internal enrollment inconsistency—8 listed as enrollment and N=20 in the arm description—adds uncertainty about the population represented. The posted safety counts give 8 at risk, but the record does not explain how these numbers relate to enrollment or to the primary endpoint analysis. That makes transparent participant accounting especially relevant.
Finally, the record lists zero analyses and does not report confidence intervals, missing-data handling, or a statistical analysis plan. This illustrates why registry interpretation depends not only on endpoint definitions, but also on complete results, denominators, and analytic context. The study's statistical contribution cannot be judged from design labels and isolated adverse-event counts alone.
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 registry record does not report the result for the primary endpoint: the number of participants with a CTCAE version 5.0 grade 3 or higher complication within 30 days post-IRE. Without that outcome, the central question specified by the trial cannot be evaluated from the posted record. No uncertainty interval or inferential result is reported either.
The enrollment figures are inconsistent. The record lists enrollment as 8, but the arm description states N=20. No explanation is reported, and the difference should not be resolved by assumption. Although the adverse-event summary lists 8 at risk for each of its event categories, that does not establish which population was intended for the primary endpoint analysis.
The single-group design has no reported control arm, so it cannot provide a direct comparative estimate. The record also does not report a statistical analysis plan, analysis population, missing-data procedures, endpoint adjudication process, or details of event attribution. The posted safety counts do not include serious-event terms, and causes or timing for the listed deaths are not reported.
The registry describes the population as advanced unresectable PHC but does not expand the acronym. It offers procedural details about electrode guidance and operator experience, but further eligibility, follow-up, and outcome-assessment details are not reported in the registry record. These limitations constrain interpretation of both the intended study population and the meaning of the posted safety entries.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Trial of IRE in Cholangiocarcinoma — NCT03769753.
- Registry record last updated 2022-09-10; status last verified 2022-08.
- Results first posted 2022-09-10.
- 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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18. Record Summary
NCT03769753 is listed as a completed phase II, single-group trial of an IRE device for cholangiocarcinoma. The record lists enrollment as 8 and describes intraoperative use during surgical exploration for advanced unresectable PHC, with ultrasound-guided electrode placement by experienced hepatopancreatobiliary surgeons. The arm description states N=20, an inconsistency the record does not explain.
The primary endpoint is the count of participants with a CTCAE version 5.0 grade 3 or higher complication within 30 days post-IRE. Its numerical result is not reported in the registry record. The posted safety summary lists 0 serious adverse events among 8 at risk, 1 other adverse event among 8 at risk, and 2 deaths. The record lists one outcome measure and zero analyses, with no comparator or reported statistical analysis plan.