Sunitinib and Chemoembolization in Treating Patients With Liver Cancer That Cannot Be Removed by Surgery: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Sunitinib and Chemoembolization in Treating Patients With Liver Cancer That Cannot Be Removed by Surgery (NCT00524316), a na phase 2 study. The primary outcome is Progression-free Survival.

2. Clinical Question

3. Trial Design

This was a phase II, single-group study of sunitinib and chemoembolization in patients with unresectable hepatocellular cancer. The registry lists Roswell Park Cancer Institute as the sponsor, an enrollment of 16, and a terminated status. The recorded start is 2007-04 and the primary completion date is 2010-12. The reason for termination is not reported in the registry record.

The study is classified as having no allocation and no masking. In a single-group design, participants receive the intervention under study without a concurrent randomized control arm. This can describe outcomes observed in the treated group, but it cannot by itself separate the treatment’s contribution from other explanations, such as the natural course of disease or differences between participants. The registry’s skip note also identifies that the arms are not a like-for-like control comparison.

The listed regimen specifies oral sunitinib malate once daily on days 1-7 and 15-35 in course 1, and on days 1-28 in subsequent courses. Doxorubicin hydrochloride and hepatic artery embolization are also listed as interventions. The record does not report the sunitinib dose, a detailed schedule for chemoembolization, or a protocol description explaining how treatment was adjusted or stopped.

The primary endpoint is progression-free survival. Secondary outcomes include overall survival; DCE-MRI measures of tissue perfusion, Ktrans, IAUC and percent viable tumor; safety and tolerability; quality of life measured with FACTHep version 4; and AFP tumor-marker response. The record lists six outcome measures and zero analyses. It does not give participant flow, the number included in each outcome analysis, or numerical estimates for the primary or secondary outcomes.

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

Reasons for not completing this period:

Analysis population for the primary outcome, as defined in the registry: All treated and eligible patients.

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 Progression-free Survival, measured over From date of randomization until the date of first documented progression or date of death from any cause, whichever came first, assessed up to 100 months.

Progression-free survival is a time-to-event measure. In this study, the event is the first documented progression or death from any cause, whichever comes first. The endpoint description defines the time origin as the date of randomization and gives a timeframe of up to 100 months. Because the trial is classified as single-group with allocation not applicable, the reference to randomization is not explained in the registry record.

The median is the point in the estimated time-to-event distribution at which half of the relevant population has experienced the event and half has not yet experienced it. It is one way to summarize how long participants remain alive without documented progression. It does not describe every participant’s experience, and it is not the same as a mean duration or a comparison between treatments.

A confidence interval expresses uncertainty around an estimate. The registry identifies a 95% confidence interval as the measure of dispersion for the primary endpoint, but it does not report the median value or the interval limits. Without those values, the magnitude and precision of the endpoint estimate cannot be assessed from the registry record. The stated analysis population is all treated and eligible patients; the number meeting that definition is not reported in the registry record.

Progression-free survival is commonly used in cancer trials because it combines progression and death into a time-to-event outcome. That general rationale does not make it a direct measure of quality of life or overall survival. Those are listed separately among the study outcomes.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

Progression-free survival is a time-to-event outcome: each participant contributes a duration from a defined starting point until an event or the end of observed follow-up. Here, the registry defines the event as documented progression or death from any cause, whichever occurs first. It describes the time origin as the date of randomization, while also classifying the study as single-group with allocation not applicable. The record does not explain that wording, so the exact operational time origin cannot be clarified from the registry alone.

What does a median time-to-event estimate mean?

The median is a summary of the distribution of event times. It marks the point at which the estimated proportion without an event reaches one half. Unlike an arithmetic average, a median time-to-event estimate can be used when some participants have not experienced the event by the end of their observed follow-up. The registry identifies the primary parameter as a median, but gives no numerical estimate.

Time-to-event methods generally account for incomplete observation through censoring. Censoring means that a participant’s event time is not observed during the available follow-up, even though the participant was observed up to some point. These methods can use the observed follow-up without treating every participant who has not had an event as if they will never have one. This is a general explanation of time-to-event analysis; the registry record does not describe censoring rules, follow-up completeness, or the method used to estimate progression-free survival.

How should the confidence interval be read?

A confidence interval gives a range of values compatible with the data under the statistical method and its assumptions. A narrower interval generally indicates greater precision, while a wider interval indicates more uncertainty. The record specifies a 95% confidence interval for the primary endpoint’s dispersion but reports neither the interval nor the median. Consequently, readers cannot assess the estimate’s precision or determine whether the interval includes particular clinically meaningful values.

A confidence interval is not a guarantee that the true value lies within a particular interval, and it does not by itself establish a treatment effect. In this single-group study, an interval around a median would describe uncertainty about the outcome estimate for the analyzed group. It would not provide a randomized comparison with another treatment.

Why does the single-group design matter?

In a randomized comparative study, allocation creates groups intended to be comparable, allowing a treatment contrast to be estimated under the design’s assumptions. This study has no randomized comparator. The enrolled group’s outcomes can be described, but the design does not estimate the difference that would have been observed against a concurrent control. Comparisons with outcomes from other settings would require information and assumptions not reported in the registry record.

The study is also listed as unmasked. This describes the masking classification, but the registry does not specify who knew the assigned treatment, how outcome assessment was conducted, or whether any outcome assessors were masked. It would be inappropriate to infer those details from the classification alone.

What is and is not reported about the analysis?

The registry names all treated and eligible patients as the primary population. It does not state how eligibility for analysis was determined, how many participants met that definition, or how missing outcome information was handled. The record lists zero analyses and does not give a statistical analysis plan. It therefore does not establish which estimation procedure was used, whether any adjustment was planned, or how secondary endpoints were analyzed.

The registry lists 16 enrolled participants. This is not necessarily the same as the number analyzed for every endpoint. Outcome-specific denominators and participant disposition are not reported. The primary completion date and the timeframe of up to 100 months do not establish how much follow-up was observed for each participant.

Planning calculations are sometimes used to illustrate how many participants might be needed under stated assumptions. They are planning illustrations, not observed results or observed power. The registry does not report a sample-size justification for this study. Any planning illustration based on an effect size should not be read as evidence that the trial was designed to detect that effect, or that the study had a particular probability of finding it.

10. Multiplicity, Missing Data and Analysis Populations

The registry lists progression-free survival as the primary endpoint and also lists secondary outcomes covering overall survival, DCE-MRI measurements, safety and tolerability, quality of life, and AFP tumor-marker response. When a study examines multiple outcomes, the chance of at least one apparently unusual result can increase if many statistical tests are conducted. Statistical plans may address this issue by specifying which outcomes are primary, how secondary outcomes will be interpreted, or whether adjustments will be used. These are general considerations; a multiplicity strategy is not reported in the registry record.

The record lists zero analyses and does not provide p-values, confidence intervals, or other outcome estimates for the listed measures. It is therefore not possible to determine which analyses were conducted, whether any multiplicity adjustment was used, or how secondary findings were intended to support conclusions.

Missing-data methods are also not reported in the registry record. There is no description of incomplete follow-up, missing endpoint assessments, censoring rules, imputation, or sensitivity analyses. These details matter because the handling of incomplete observations can affect time-to-event estimates and other outcomes. The primary analysis population is stated as all treated and eligible patients, but the number meeting that definition and the treatment of participants with missing data are not given.

Without an analysis plan, outcome estimates, and participant-level accounting, the record does not establish whether the reported endpoint population matches the enrolled population or how robust any result would be to missing observations. These are reporting gaps, not proof that particular analyses or procedures were or were not performed.

11. Safety Results

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

Other (non-serious) adverse events

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

Most frequent serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: anaemia (12.5%), abdominal pain (12.5%), gastrointestinal haemorrhage (12.5%), dehydration (12.5%), thrombocytopenia (6.2%).

The registry presents one treatment group, titled “Treatment: Sunitinib and Chemoembolization.” It lists 16 participants at risk and 16 affected for serious adverse events, and 16 at risk and 16 affected for other adverse events. These are the recorded group counts; the record does not explain the definitions or reporting procedures behind the affected counts. The counts alone do not describe the nature, severity, duration, or timing of all events.

Among the listed serious adverse event terms, anaemia, abdominal pain, gastrointestinal haemorrhage, and dehydration are each reported as 12.5%. Thrombocytopenia and constipation are each reported as 6.2%. These percentages describe the listed terms in the registry’s adverse-event summary. The record does not provide event-level detail, participant narratives, or a complete explanation of how terms were selected for display.

The registry also lists safety and tolerability as a secondary outcome. It does not provide a broader numerical safety analysis, exposure-adjusted rates, a comparison group, or the number of events per participant. Because this is a single-group study, the listed figures do not establish how event frequency compares with another treatment or background risk. They should be read as descriptive registry entries, not as a basis for a treatment recommendation.

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

The registry establishes that a phase II, single-group study of sunitinib and chemoembolization was conducted in patients with unresectable hepatocellular cancer, with 16 participants enrolled and the study marked terminated. It identifies progression-free survival as the primary endpoint and lists several other outcome areas. The record does not report the numerical result for the primary endpoint or the listed secondary outcomes.

That absence limits what can be concluded. Without a median progression-free survival estimate and its confidence interval, the size and precision of the observed outcome cannot be assessed. Without a comparator, the study cannot estimate a randomized difference between the combination and another treatment. Even a clearly reported outcome in the treated group would not, by itself, establish what would have occurred to similar participants under a different treatment.

The adverse-event summary records serious and other adverse-event counts for the treatment group and lists selected serious event terms with percentages. Those entries provide some descriptive safety information, but they do not supply a complete account of event timing, severity, recurrence, or comparative risk. The registry does not report a full safety analysis.

The status “terminated” does not explain why the study ended or what effect termination had on recruitment, follow-up, or analysis. Nor does the recorded enrollment establish the number included in each outcome analysis. These details are not reported in the registry record.

Overall, the record describes the study question, treatment schedule, design classification, endpoint definitions, and selected adverse-event entries. It does not report enough outcome statistics to quantify progression-free survival or assess the precision of the primary result, and its single-group design does not provide a direct treatment comparison. The findings should not be interpreted as proof that the combination does or does not change clinical outcomes.

13. Why This Trial Matters Statistically

This study illustrates why design and result reporting must be considered together. A time-to-event endpoint such as progression-free survival can summarize the interval until progression or death, but a named endpoint is not itself a result. The registry identifies a median and a 95% confidence interval as the intended summary, yet does not report their values. Without the estimate and its uncertainty, the endpoint cannot be quantitatively interpreted.

The single-group classification is equally important. An observed outcome in a treated group answers a descriptive question about that group; it does not create a concurrent counterfactual showing what would have happened under another treatment. This distinction remains important even when a study has a clearly defined endpoint and a detailed intervention description. The registry explicitly cautions that arms are not a like-for-like control comparison.

The record also shows why outcome populations and follow-up need clear reporting. Enrollment is listed as 16, while the primary population is all treated and eligible patients. The number in that analysis population is not reported. The endpoint timeframe extends up to 100 months, but the record does not give participant-level follow-up or outcome-specific denominators. Those omissions prevent readers from judging how the analyzed group relates to the enrolled group.

Finally, a list of secondary outcomes and adverse-event terms is not a substitute for statistical estimates and methods. Clear reporting of numerical results, uncertainty, missing-data handling, and the analysis plan would help readers distinguish descriptive observations from comparative evidence. This record makes the design and endpoint visible, but leaves those result-level questions unresolved.

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 interpretation is that the registry record does not report the numerical progression-free survival result, despite identifying it as the primary endpoint and specifying a 95% confidence interval. The reason the study was terminated is also not reported. The status alone does not explain whether the planned recruitment, treatment, or follow-up was completed.

The study is listed as single-group, with no allocation and no masking. There is no concurrent control group, so the design cannot establish a treatment contrast. The record lists enrollment as 16, but does not state how many participants were treated, met the primary analysis population definition, or contributed to each outcome.

The primary endpoint description refers to time from randomization, although allocation is listed as not applicable. The registry does not explain this wording. It also does not describe progression assessment procedures, censoring rules, follow-up completeness, or the statistical method used to estimate the median.

Other limitations in the available record include the absence of a sample-size justification, a statistical analysis plan, missing-data methods, multiplicity procedures, and numerical secondary outcome results. The intervention listing gives a sunitinib schedule but not the dose or a detailed chemoembolization schedule. The adverse-event summary supplies selected terms and percentages, without event-level details or a comparative safety analysis. These reporting gaps constrain interpretation; they do not establish that the underlying study materials contained no further information.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: Sunitinib and Chemoembolization in Treating Patients With Liver Cancer That Cannot Be Removed by Surgery — NCT00524316.
• Registry record last updated 2017-05-09; status last verified 2017-03.
• Results first posted 2014-06-04.
• 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

This is a terminated phase II, single-group study of sunitinib and chemoembolization for unresectable hepatocellular cancer. The registry lists 16 participants enrolled, no allocation, and no masking. The recorded study start is 2007-04, and primary completion is listed as 2010-12.

The primary endpoint is median progression-free survival, defined from the date of randomization to documented progression or death, whichever comes first, with a timeframe of up to 100 months. The analysis population is described as all treated and eligible patients. The registry does not report the endpoint estimate or its 95% confidence interval.

Listed secondary outcomes cover overall survival, DCE-MRI measures, safety and tolerability, quality of life, and AFP response. The adverse-event summary includes selected serious event terms. The registry does not report the reason for termination, the statistical analysis plan, or numerical results for the primary and secondary outcomes.