Combination Chemoembolization and Stereotactic Body Radiation Therapy in Unresectable Hepatocellular Carcinoma: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Combination Chemoembolization and Stereotactic Body Radiation Therapy in Unresectable Hepatocellular Carcinoma (NCT02513199), a na phase 2 study. The primary outcome is Number of Participants With Objective Response Rate.

2. Clinical Question

3. Trial Design

This is a phase 2, single-group study of TACE combined with SBRT in unresectable HCC. The registry lists 32 participants as the enrollment and describes the treated group as participants with HCC and a lesion greater than 3 cm. It records the study as completed. The start is listed as 2014-11 and primary completion as 2022-01-01.

Allocation is marked not applicable, the model is single-group, and masking is listed as none. The registry therefore does not describe a randomized, masked comparison. With no concurrent control group, the study can describe outcomes observed among participants receiving the combination, but cannot by itself isolate the combination’s effects from the natural course of disease, other care, or differences in patient characteristics.

The treatments are listed as SBRT, a radiation intervention, and TACE, a drug intervention. The brief description states that the treatments are combined in an organized sequence, but the sequence details are not reported in the registry record. The primary outcome is the number of participants with an objective response, assessed by mRECIST and diffusion-weighted imaging via MRI surveillance, up to 72 months.

Secondary outcomes include time to complete response, time to progression, overall survival, progression-free survival, and change in Child-Turcotte-Pugh score. The record lists these outcome types but does not provide their observed results in the registry record for this summary. It also does not report a protocol-level sample-size justification, a detailed statistical analysis plan, or how follow-up and censoring were handled.

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.

Treatment

Reasons for not completing this period:

Follow-Up

Reasons for not completing this period:

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 Number of Participants With Objective Response Rate, measured over up to 72 months.

The primary endpoint is the number of participants with an objective response. It combines complete and partial response under the mRECIST definitions described in the registry. Complete response means disappearance of all target lesions. Partial response means at least a 30% decrease in the sum of the longest diameters of target lesions, using the baseline sum as reference. Stable disease and progressive disease are defined separately and do not meet the stated objective-response criteria.

Tumor response is a way to characterize change in measurable disease following treatment. The registry specifies assessment using mRECIST criteria and diffusion-weighted imaging (DWI) through MRI surveillance. These criteria describe how response categories are assigned; they do not, by themselves, indicate how many participants responded.

The timeframe is up to 72 months. A count is readily interpretable as the number of participants meeting the response definition, but the count alone does not show the precision of an estimated response rate or establish what would have occurred under a different treatment. The observed primary endpoint count and a corresponding estimate of uncertainty are not reported in the registry record.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The registry describes a phase 2 study with a single-group model and no masking. This design provides a framework for reporting outcomes among participants receiving the TACE/SBRT combination. It does not include a randomized control group. Thus, even if response results were available, the analysis would be descriptive of this treated group rather than a direct estimate of the difference between two assigned treatments.

What does the primary outcome count?

The primary outcome is the number of participants with objective response. The registry defines response using mRECIST criteria and identifies DWI via MRI surveillance as part of assessment. The response categories distinguish complete response, partial response, stable disease, and progressive disease. Complete response is disappearance of all target lesions. Partial response requires at least a 30% decrease in the sum of the longest diameters of target lesions relative to the baseline sum. Stable disease falls short of both the stated partial-response shrinkage and progressive-disease increase thresholds. Progressive disease is at least a 20% increase relative to the smallest sum recorded since treatment started, or the appearance of one or more new lesions.

These definitions set clinical measurement rules; they do not supply the observed classifications. The registry record does not report how many participants were classified in each category or the objective-response count. Nor does the record state whether the primary endpoint was analyzed as a simple count, as a proportion with an interval estimate, or using another procedure. The count and the uncertainty around an estimated response frequency are different pieces of information: a confidence interval, in general, conveys the range of values compatible with the data and a stated statistical procedure.

Why does the single-group design matter?

In general, a treatment comparison requires a reference group to estimate how outcomes differ under alternative care. A single-group study instead observes outcomes under the treatment approach being studied. Responses can be reported, but the design cannot separate treatment effects from other explanations as reliably as a randomized comparison. Participants may differ in disease characteristics or other factors that also relate to outcomes, and there is no concurrent randomized group in this record to address that contrast.

Allocation is recorded as not applicable and masking as none. The record does not describe random assignment or masked outcome assessment. Lack of masking can matter when outcome assessment involves interpretation, even when response categories are defined in advance. The registry entries do not state whether imaging was reviewed independently or whether any procedures were used to reduce assessment differences, so no such procedures should be assumed.

How should precision and planning be understood?

For a response count, precision depends on the number of observations and on the amount of information in the outcome. In general, a confidence interval communicates uncertainty around an estimate; a point estimate alone does not show that uncertainty. No primary response estimate or confidence interval is reported in the registry record, so neither the magnitude nor precision of the response outcome can be evaluated.

The sample-size assumptions describe planning illustrations based on an observed effect, with a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. The illustrations specify that time-to-event calculations express the total number of events, while other calculations express participants per arm. Those assumptions are not the design described for this study, which is single-group. A planning illustration based on a hypothetical two-arm allocation is not an observed feature of the trial and is not observed power. The registry record does not report the trial’s own sample-size justification, nor does it explain the effect size used in any sponsor planning.

What analyses are not described?

The record does not report a detailed statistical analysis plan, a prespecified method for handling missing response assessments, or definitions of analysis populations. Those omissions limit interpretation: the analysis population determines which participants contribute to an outcome, while missing-data rules can affect which observations are available and how conclusions are framed. The record also does not report interim analyses or a plan for multiplicity. These methods should not be inferred from the endpoint list or from the study’s completed status.

Secondary outcomes include time to CR, time to progression, overall survival, progression-free survival, and change in CTP score. In general, time-to-event outcomes require rules for defining events, follow-up, and censoring; the record does not report those analytical details or results. Multiple outcomes may also raise questions about interpretation of chance findings when many hypotheses are considered. Whether and how that issue was addressed is not reported in the registry record. The listed outcome names alone do not establish the statistical procedures used.

10. Multiplicity, Missing Data and Analysis Populations

The registry lists a primary endpoint and several secondary outcomes, including time to complete response, time to progression, overall survival, progression-free survival, and change in Child-Turcotte-Pugh score. Considering multiple outcomes can raise multiplicity concerns: in general, testing many hypotheses can increase the chance that at least one result appears noteworthy by chance. The registry record does not report whether multiplicity was addressed, whether outcomes were prioritized beyond the primary designation, or whether any adjustment was planned.

The record also does not report methods for missing data, the analysis populations used, or how participants with incomplete imaging or follow-up were handled. These details can affect the number of participants contributing to an endpoint and the interpretation of an estimate. No assumptions about imputation, exclusion, or censoring can be made from the information available in the registry record.

Because the primary response count and results for the listed secondary outcomes are not reported in the registry record, missing-data handling and multiplicity cannot be assessed against actual analyses. The registry record does not report interim analyses or a detailed statistical analysis plan. The endpoint listing should therefore be distinguished from evidence that a specific analysis procedure was conducted.

11. Safety Results

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

Deaths

Other (non-serious) adverse events

Participants with at least one non-serious event at or above the registry's 0% reporting threshold.

The safety entry records 32 participants at risk for serious adverse events and 0 affected. It also records 32 at risk for other adverse events and 0 affected. The entry separately reports 12 deaths. These are counts as recorded; they do not establish when events occurred, their causes, or whether investigators considered them related to treatment. The registry record does not report those details.

The zero affected counts should not be interpreted as proof that no adverse events occurred outside the categories or reporting period represented by this registry entry. The record gives no detailed serious adverse-event terms, and the list of top serious terms is not reported in the registry record. It also does not describe event-collection procedures, severity grading, or attribution rules in the registry record.

Deaths are a distinct reported count and should not be added to, or assumed to overlap with, the affected counts. Without timing, cause, attribution, and follow-up details, the counts are descriptive rather than a treatment comparison. The single-group design also offers no control-group safety rate for context.

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

This study record is relevant as an evaluation of a combination approach for unresectable HCC, specifically participants with a lesion greater than 3 cm. It defines an objective-response endpoint, identifies imaging methods, and lists additional outcomes related to response, progression, survival, and liver function. These details show what the study set out to assess and the basis for its primary response classification.

However, the information available in the registry record does not include the primary response count or results for the secondary outcomes. It therefore does not establish how frequently objective response occurred, how long responses took, how progression or survival outcomes unfolded, or how CTP scores changed. The study’s completed status is not a substitute for reporting these results.

Even if outcome counts were available, the single-group design would limit causal interpretation. It could describe outcomes among participants treated with TACE/SBRT, but without a control group it would not directly estimate how the combination compares with another treatment or with outcomes in otherwise similar participants receiving different care. The registry entries do not report an adjusted comparative analysis.

The safety record presents serious adverse-event and other adverse-event counts, together with deaths, but does not provide attribution, timing, causes, or detailed terms. Those counts should not be interpreted as establishing the combination’s safety profile in isolation. More complete outcome reporting and analysis details would be needed to assess the size, uncertainty, and clinical context of the observed outcomes.

13. Why This Trial Matters Statistically

Statistically, this study illustrates the distinction between defining an endpoint and reporting its result. The registry gives a detailed objective-response definition, including the imaging approach and mRECIST thresholds. That information makes clear what the primary outcome is intended to measure. But the observed response count is not reported in the registry record, so an endpoint definition alone cannot support a quantitative conclusion.

The single-group structure is another central feature. It can describe outcomes under a treatment combination, but it does not provide a randomized comparator for estimating differences between treatments. This is important when reading any observed response or safety count: an outcome among treated participants is not, by itself, evidence of what would have happened under an alternative approach.

The study also lists outcomes spanning tumor response, progression, survival, and CTP score. Different outcome types need different analytical details. In general, time-to-event outcomes depend on definitions of events and follow-up, while a response count depends on classification rules and assessment availability. The registry entries do not report these analysis details or the results, so they cannot be evaluated.

Finally, planning calculations should not be confused with the trial’s actual design or observed findings. The stated illustration assumptions describe equal allocation between two arms, whereas the registry records a single-group study. Keeping those ideas separate is essential: planning calculations are not observed power and do not fill the gap left by unreported trial outcomes.

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 primary limitation for interpreting efficacy is that the registry record does not report the objective-response count or results for the listed secondary outcomes. Without these, response frequency, outcome precision, and the pattern of follow-up outcomes cannot be assessed. The primary endpoint timeframe is up to 72 months, but the registry entries do not report the timing or completeness of individual assessments.

The single-group design has no concurrent control arm. Therefore, the study cannot provide a randomized estimate of how the combination compares with TACE alone, SBRT alone, or another treatment. The absence of masking is also recorded, but the registry entries do not describe whether imaging assessment was independently reviewed or how assessment differences were managed.

The registry does not report detailed treatment sequencing, analysis populations, missing-data procedures, censoring rules, interim analyses, or a statistical analysis plan. The listed secondary outcomes do not include their results or analysis methods. The registry entries also do not provide the study’s sample-size justification, so the enrollment figure should not be treated as evidence that a particular precision or statistical power was achieved.

Safety interpretation is limited by the lack of event terms, timing, attribution, and causes of death. The recorded counts alone cannot establish causal relationships or provide a comparison with another treatment group. These limitations are specific to the available record details and should not be filled in by assumptions about the protocol or conduct.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: Combination Chemoembolization and Stereotactic Body Radiation Therapy in Unresectable Hepatocellular Carcinoma — NCT02513199.
• Registry record last updated 2023-06-18; status last verified 2023-05.
• Results first posted 2023-06-18.
• Data retrieved from ClinicalTrials.gov on 2026-11-09.
• Group sizes, means, event counts and sponsor-reported p-values are reproduced as posted. Effect estimates, confidence intervals and p-values not attributed to the sponsor are independently calculated from the posted summary statistics.

18. Record Summary

The registry records NCT02513199 as a completed phase 2, single-group study of TACE combined with SBRT in unresectable HCC. Enrollment is listed as 32. The treated group is described as participants with HCC and a lesion greater than 3 cm. The primary endpoint is the number of participants with objective response, assessed by mRECIST and DWI via MRI surveillance, up to 72 months.

The registry lists secondary outcomes covering time to complete response, progression, survival, and change in CTP score. The observed primary response count and secondary outcome results are not reported in the registry record. Safety counts list 0 affected by serious adverse events, 0 affected by other adverse events, and 12 deaths; event details and attribution are not reported.