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 A Phase II Study of Continuous Hepatic Arterial Infusion With Floxuridine (FUDR) and Dexamethasone (DEX) in Patients With Unresectable Primary Hepatic Malignancy (NCT00587067), a na phase 2 study. The primary outcome is Treatment Response.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00587067 |
| Phase | Phase 2 |
| Condition | Hepatic Cancer |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 34 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Memorial Sloan Kettering Cancer Center |
| Results posted | Yes |
2. Clinical Question
The study asks whether continuous hepatic arterial infusion of floxuridine (FUDR) and dexamethasone (DEX) can produce treatment responses in patients with unresectable primary hepatic malignancy. The registry description focuses on hepatocellular carcinoma and intrahepatic cholangiocarcinoma, including patients whose disease was considered unresectable after review by the Hepatobiliary Surgery service. The primary outcome is titled Treatment Response, measured as a count of participants, with a timeframe of up to 5 years.
The question is evaluated in a single-group Phase II study. There is no randomized control group or other comparator described in the record, so the study is not designed to make a like-for-like comparison between treatments. The relevant statistical question is therefore about the number of participants meeting the response definition, not about a difference between treatment groups. The response criteria themselves are not reported in the registry record.
Population
Patients with unresectable primary hepatic malignancy. The study description identifies hepatocellular carcinoma and intrahepatic cholangiocarcinoma; unresectability was reviewed by the Hepatobiliary Surgery service.
Intervention
Continuous hepatic arterial infusion of floxuridine (FUDR) and dexamethasone (DEX), with hepatic artery pump placement described in the study summary.
Comparator
No comparator is listed. The registry record identifies a single-group study and does not describe random allocation.
Primary question
How many participants meet the study's definition of treatment response during a timeframe of up to 5 years? The response definition and the observed result are not reported in the registry record.
3. Trial Design
The registry record classifies this as a Phase II study with a single-group model and no masking. Allocation is listed as not applicable. The study is marked completed, with enrollment of 34 participants. Its recorded start is 2003-06, and its primary completion date is 2016-05-19. Memorial Sloan Kettering Cancer Center is listed as the sponsor.
The intervention is floxuridine, while the study description specifies continuous hepatic arterial infusion of FUDR and DEX. It describes hepatic artery pump placement for eligible patients with hepatocellular carcinoma and peripheral cholangiocarcinoma considered unresectable after review by the Hepatobiliary Surgery service. The primary outcome description also refers to hepatocellular carcinoma and intrahepatic cholangiocarcinoma. The registry record does not explain the difference in wording between these descriptions.
A single-group design can describe outcomes observed among participants receiving the intervention, but it does not provide a concurrent control group for estimating a treatment contrast. General statistical interpretation: without a comparator, observed responses cannot by themselves separate an intervention effect from other explanations, such as the underlying course of disease or features of the participants selected for the study.
The registry lists Disease Progression and Median Survival as secondary outcomes. It records four outcome measures overall, but does not include analysis entries. Details such as eligibility criteria beyond the summary, treatment duration, assessment schedule, response criteria, and the statistical analysis plan are not reported in the registry record. The design classification and enrollment describe the study structure; they do not establish the size or precision of any treatment-response result.
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 | Floxuridine + Dexamethasone |
|---|---|
| STARTED | 34 |
| COMPLETED | 34 |
| NOT COMPLETED | 0 |
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 | Floxuridine + Dexamethasone |
|---|---|---|
| Participants analysed | 34 | |
| Age, Continuous | years | 56.5 (30 to 85) |
| Sex: Female, Male: Female | Participants | 22 |
| Sex: Female, Male: Male | Participants | 12 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 1 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 31 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 2 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 |
| Race (NIH/OMB): Asian | Participants | 2 |
| 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 | 30 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 2 |
| Region of Enrollment: United States | participants | 34 |
6. Primary Endpoint
The registry lists the primary outcome as Treatment Response, measured over Up to 5 years.
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary outcome is Treatment Response, recorded as a count of participants, with an assessment timeframe of up to 5 years. A participant count is a direct way to describe how many people meet an outcome definition in the study group. For a response endpoint, that count can convey whether responses were observed, but it is interpretable only when the response criteria and the number assessed are clear.
The registry record does not report the response criteria, the observed count, or a corresponding estimate of uncertainty. It also does not state how participants with incomplete assessments or missing response data were handled. As a result, the endpoint's name and measurement unit are available, but the operational definition and the result are not.
General statistical context: a binary response outcome classifies each participant according to whether the prespecified response condition was met. A count then reports the number classified as responders. A confidence interval, when reported, expresses the uncertainty around an estimated response frequency; it does not correct for a missing definition, missing assessments, or the absence of a comparison group. No response estimate or confidence interval is reported in the registry record, so the extent of statistical uncertainty cannot be assessed from the endpoint entry.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record establishes that this was a completed, single-group Phase II study with enrollment of 34 participants. It identifies Treatment Response as the primary outcome and gives a timeframe of up to 5 years. It also lists Disease Progression and Median Survival as secondary outcomes. These entries describe what the study intended to assess, but they do not give the observed efficacy results.
No primary response count, response proportion, confidence interval, or test result is reported in the registry record. The record also lists no analysis entries. Therefore, the posted information does not show how many participants responded or how precisely any response estimate was measured. The outcome title alone is not evidence that responses occurred, and the completed status does not supply a missing result.
The single-group design limits the type of inference possible even if outcome counts are available elsewhere. General statistical context: a treatment-group outcome can be described, but a treatment effect relative to usual care or another intervention requires a suitable comparison. With no comparator listed, a causal contrast cannot be estimated from this registry information. The absence of a comparator is a design feature, not a flaw that can be repaired by interpreting a participant count as a comparative result.
The safety entry reports 5 affected participants among 34 at risk for serious adverse events in the floxuridine plus dexamethasone group. It also lists serious-event terms with percentages and reports 33 deaths. Those figures are safety information, not a substitute for the missing primary efficacy result. The registry record does not state the relationship of the reported events or deaths to the intervention, their timing, or whether event terms overlap.
Consequently, interpretation should remain narrow: the record documents the study question, design, enrollment, selected outcome labels, and some safety counts. It does not provide enough outcome information to estimate treatment response, compare treatments, or judge the precision of an efficacy finding. Any fuller interpretation would require results and methods not reported in the registry record.
9. Statistical Methods Explained
The registry record identifies a single-group study with no masking and allocation listed as not applicable. Those design details determine which statistical comparisons can be supported. There is no listed randomized allocation or control group. General statistical context: an outcome measured in one group can be summarized descriptively, while a treatment contrast usually relies on comparing outcomes across groups that provide a meaningful counterfactual. A single-group response count does not itself establish what would have happened to similar participants under another treatment or without the intervention.
What does a participant count tell us?
The primary outcome is measured as a count of participants. For a response endpoint, the count would represent participants meeting the response definition, if that definition and result were reported. A count alone does not reveal the proportion of all participants who responded unless its denominator is known and relevant. The registry record lists enrollment of 34, but does not report how many participants were assessed for response or the response count. It therefore does not support calculation of a response proportion.
General statistical context: estimates from a finite group are subject to sampling uncertainty. A confidence interval communicates a range of values compatible with the data and a stated method, under its assumptions. Its width depends on factors including the amount of information and the variability of the outcome. No confidence interval is reported for treatment response in the registry record. It would not be appropriate to manufacture one from enrollment alone because the response count and analysis details are absent.
Why does the study design matter?
In a single-group study, observed outcomes can describe the participants who received the intervention. They cannot, without a suitable comparison, isolate an intervention effect from other explanations. General statistical context: randomization can help balance measured and unmeasured factors across groups on average, while a nonrandomized or single-group design does not create that protection through allocation. The registry record lists no comparator and provides no allocation procedure. The study's design should therefore guide claims about what the data could establish, even if fuller results are available outside this record.
The record lists Disease Progression and Median Survival as secondary outcomes, but does not report their results or detailed analysis methods. It does not describe how progression was defined, how survival time was handled, or how censoring was addressed. General statistical context: time-to-event analyses account for both when an event occurs and the fact that some participants may not have an observed event during follow-up. Without the event definitions, follow-up information, and analysis results, no time-to-event estimate can be evaluated from this entry.
What can the planning assumptions support?
The registry's planning assumptions describe an illustration based on an observed effect, with a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. They describe planning for a stated fraction of an observed effect. For time-to-event illustrations, the stated quantity is the total number of events; for other illustrations, it is participants per arm. These are planning assumptions, not a description of the actual single-group trial design. The record does not report numerical planning results or the observed effect used for such an illustration. Any sample-size illustration should be read as hypothetical planning information, not as observed power or as evidence that the trial was adequately powered.
General statistical context: over-dispersion occurs when variation in count data exceeds what a chosen probability model expects. If present, standard errors based on the simpler model may be too small, producing intervals that are too narrow or tests that are too optimistic. The registry record does not identify an over-dispersion assessment or a count-data model, and the primary endpoint is listed simply as a participant count. It is therefore not possible to say whether this issue was relevant to the sponsor's analysis.
The record lists zero analysis entries. It does not describe the statistical test, estimation method, confidence-interval method, covariate adjustment, or handling of repeated assessments. This means that method-specific assumptions cannot be checked and no sponsor analysis can be compared with a simple re-calculation. General statistical context: a recalculation from summary statistics, when possible, is unadjusted and may not reproduce an analysis that accounts for design or participant-level information. There are no primary outcome summary statistics in this record from which to make such a recalculation.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists four outcome measures overall, identifies Treatment Response as the primary outcome, and names Disease Progression and Median Survival as secondary outcomes. It does not provide results for these outcomes or a detailed list of their measurement definitions. No analysis entries are listed, and the statistical analysis plan is not reported in the registry record.
When a study evaluates multiple outcomes or makes multiple statistical tests, the chance of at least one apparently notable finding can increase. General statistical context: multiplicity procedures can control that risk, but whether they are appropriate depends on the prespecified questions and analysis plan. The record does not state whether multiplicity was addressed, so no adjustment method or interpretation of multiple testing can be established.
Missing-data handling is also not reported in the registry record. The entry does not state how many participants completed response assessments, whether follow-up was incomplete, or how missing outcome observations were treated. The enrollment count is not enough to establish the analysis population for each outcome. The record does not identify an intention-to-treat, per-protocol, or other analysis population.
These omissions matter because the number assessed and the handling of missing observations can affect how an outcome count is interpreted. They also limit reproducibility: readers cannot determine which participants contributed to an outcome analysis or whether the reported results, if available elsewhere, follow prespecified procedures. Interim analyses and stopping rules are not reported in the registry record.
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 |
|---|---|---|---|
| Floxuridine + Dexamethasone | 5 | 34 | 14.7% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Floxuridine + Dexamethasone | 33 of 34 |
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 |
|---|---|
| Floxuridine + Dexamethasone | 5 of 34 |
Most frequent serious adverse event terms
| Event term | Organ system | Floxuridine + Dexamethasone |
|---|---|---|
| Bilirubin Increased | Investigations | 2 of 34 |
| GI, other | Gastrointestinal disorders | 1 of 34 |
| Hepatic, other | Hepatobiliary disorders | 1 of 34 |
| Melena | Gastrointestinal disorders | 1 of 34 |
| Pain, other | General disorders | 1 of 34 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Floxuridine + Dexamethasone |
|---|---|---|
| Increased bilirubin | Investigations | 3 of 34 |
| Wound infection | Infections and infestations | 3 of 34 |
| Headache/Migraine | Nervous system disorders | 1 of 34 |
| Diarrhea | Gastrointestinal disorders | 1 of 34 |
| Delerium | Psychiatric disorders | 1 of 34 |
| Supraventricular tachycardia | Cardiac disorders | 1 of 34 |
Serious events with the highest arm-level rate: bilirubin increased (5.9%), gi, other (2.9%), hepatic, other (2.9%), melena (2.9%), pain, other (2.9%).
The safety entry lists a floxuridine plus dexamethasone group with 34 participants at risk for serious adverse events and 5 affected participants. The top serious-event terms include Bilirubin Increased at 5.9%, and GI, other; Hepatic, other; Melena; and Pain, other, each at 2.9%. The record also reports 33 deaths in the group.
These are safety counts and listed percentages, not evidence by themselves that the intervention caused an event. The registry record does not state the timing of the events, their relationship to treatment, whether participants could contribute more than one event term, or the basis for the reported percentages. It also does not provide detail about the deaths, including their timing or attribution. The death count should not be interpreted as a treatment-related death count unless that relationship is reported.
General statistical context: adverse-event summaries depend on how events are defined, collected, grouped, and attributed. A count of affected participants differs from a count of total event episodes, since one participant may experience more than one event. The record provides the affected-participant count for serious adverse events and selected term percentages, but does not provide enough detail to reconstruct event-level data or assess comparative safety. No comparator group is listed.
12. What the Result Does, and Does Not, Mean
The registry information supports a description of the study's purpose and structure: a completed Phase II, single-group evaluation of continuous hepatic arterial infusion of FUDR and DEX in patients with unresectable primary hepatic malignancy. It records enrollment of 34 participants, a primary outcome titled Treatment Response, a timeframe of up to 5 years, and secondary outcomes of Disease Progression and Median Survival. It also contains serious adverse-event counts and selected event-term percentages.
It does not report the primary response count or the results for the secondary outcomes. Without those results, it is not possible to describe how often response occurred, estimate the precision of a response measure, or assess what the progression and survival outcomes showed. The record also does not report response criteria, analysis populations, missing-data methods, or a statistical analysis plan.
The single-group design means the registry entry does not offer a concurrent comparison. General statistical context: observing an outcome in a treated group is not the same as estimating a difference from an untreated or differently treated group. For that reason, the record alone cannot establish a comparative treatment effect. This is a limit on inference from the design and available information, not a conclusion about whether the intervention is beneficial or harmful.
The serious adverse-event figures should likewise be read descriptively. The registry lists 5 affected participants among 34 at risk and 33 deaths, but does not report attribution or timing. Those entries do not by themselves establish that events or deaths were caused by treatment. Overall, the record documents a study and some safety information, but does not contain the efficacy results needed to answer its primary treatment-response question.
13. Why This Trial Matters Statistically
This trial is statistically notable as a single-group Phase II study with an explicit treatment-response question and a registry record that does not report the response result. The distinction between a study's planned outcome and its posted result is central to evidence interpretation: an endpoint label describes what was to be measured, while an observed estimate is needed to describe what the study found.
The design also illustrates why the comparison structure matters. Enrollment of 34 participants can describe the scale of the study, but enrollment alone does not quantify the response rate or its precision. With no comparator listed, the record cannot support a direct estimate of a treatment difference. General statistical context: a small or single-group dataset may be useful for describing outcomes and informing future questions, but its claims must match the information its design can support.
The registry record additionally highlights the importance of complete reporting. Response criteria, results, confidence intervals, analysis populations, missing-data handling, and a detailed statistical plan are not reported. Without these details, readers cannot reproduce the analysis, understand which observations contributed, or judge the uncertainty attached to an efficacy estimate. The listed safety counts contribute descriptive information, while the missing attribution and timing limit their interpretation.
The statistical lesson is not a verdict on the intervention. It is that clinical interpretation depends on both design and transparent outcome reporting. A completed study record can still leave its primary statistical question unanswered when outcome results and analysis details are absent.
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 main limitation specific to this record is that the primary outcome result is not reported. Treatment Response is identified as a participant count with a timeframe of up to 5 years, but the response definition, observed count, and uncertainty measure are absent. Results for the listed secondary outcomes, Disease Progression and Median Survival, are also not reported.
The single-group model and lack of a listed comparator restrict comparative interpretation. Allocation is recorded as not applicable, and masking is listed as none. These design details mean the registry record does not describe a randomized, masked comparison. No treatment contrast can be estimated from the information in the entry.
Several methods needed to assess the results are not reported in the registry record: eligibility details beyond the summary, response assessment procedures, the analysis population, missing-data handling, statistical tests, confidence-interval methods, multiplicity procedures, interim analyses, and the statistical analysis plan. The record also does not explain the relation between the population wording in the summary and primary outcome description.
Safety interpretation has additional limits. The registry lists 5 participants affected by serious adverse events, selected event-term percentages, and 33 deaths, but does not report timing, attribution, overlap among terms, or the details needed to reconstruct event counts. These omissions prevent assessment of causal relationships or comparative safety from this record alone. The listed enrollment is not a substitute for outcome-specific denominators or follow-up information.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: A Phase II Study of Continuous Hepatic Arterial Infusion With Floxuridine (FUDR) and Dexamethasone (DEX) in Patients With Unresectable Primary Hepatic Malignancy — NCT00587067.
- Registry record last updated 2023-04-04; status last verified 2023-03.
- Results first posted 2017-08-04.
- 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.
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18. Record Summary
NCT00587067 is a completed Phase II, single-group study sponsored by Memorial Sloan Kettering Cancer Center. The recorded start is 2003-06, primary completion date is 2016-05-19, and enrollment is 34. The study evaluates continuous hepatic arterial infusion of floxuridine (FUDR) and dexamethasone (DEX) in patients with unresectable primary hepatic malignancy.
The primary outcome is Treatment Response, measured as a count of participants over a timeframe of up to 5 years. Disease Progression and Median Survival are listed as secondary outcomes. No analysis entries or primary response results are reported in the registry record.
The safety entry lists 5 participants affected by serious adverse events among 34 at risk and reports 33 deaths. It includes selected serious-event terms and percentages, but does not report event attribution or timing. The record does not list a comparator, response criteria, or detailed analysis methods.