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 Intra-arterial Hepatic Beads Loaded With Irinotecan With Concomitant Chemotherapy With FOLFOX in Patients With Colorectal Cancer With Unresectable Liver Metastases: a Phase II Multicenter Study (NCT01839877), a na phase 2 study. The primary outcome is Progression-free Survival Rate at 9 Months.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01839877 |
| Phase | Phase 2 |
| Condition | Colorectal Cancer With Non Resectable Hepatic Metastasis |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 58 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Federation Francophone de Cancerologie Digestive |
| Results posted | Yes |
2. Clinical Question
Population
Patients with colorectal cancer and non-resectable liver metastases. The primary endpoint population was the mITT population: patients included in the study who had at least one radiological assessment within 9 months following inclusion.
Intervention
Intra-arterial hepatic beads loaded with irinotecan, described as DEBIRI, alongside systemic FOLFOX. The registry describes the intended approach as combining hepatic intra-arterial irinotecan with systemic 5FU and oxaliplatin.
Comparator
There was no concurrent comparison group in the registry description. The study is classified as single-group, and allocation is listed as not applicable. The registry therefore does not describe a randomized comparison of this regimen against another treatment.
Primary question
Among the defined mITT population, what proportion of patients were alive without progression at 9 months after starting treatment? Progression was based on investigator-assessed radiological progression under RECIST V1.1; death from any cause also counted as failure.
The question focuses on disease status at a specified follow-up point rather than a direct comparison between treatment strategies. The endpoint combines radiological progression and death: either event within the specified period means the patient does not meet the endpoint. The registry states that the first event was to be considered, but the numerical endpoint result and its precision are not reported in the registry record.
The registry background describes systemic chemotherapy as the basis of treatment and notes concerns about toxicity and the practical demands of hepatic intra-arterial chemotherapy. It presents the study regimen as a way to combine systemic FOLFOX with irinotecan delivered through hepatic beads. Those statements explain the rationale for evaluating the approach; they do not establish a comparative treatment effect.
3. Trial Design
This was a completed phase II, multicenter study with a single-group model. The registry lists enrollment as 58 and identifies the intervention as HIA DEBIRI with systemic FOLFOX. Allocation is recorded as not applicable, and masking is listed as none. No randomized or masked control group is described.
The treatment combined intra-arterial hepatic beads loaded with irinotecan and systemic FOLFOX. The registry summary says the procedure was limited to 2 sessions and describes that restriction as a feature intended to make the approach easier to generalize. It does not report a comparison of different treatment schedules or components.
The primary endpoint was progression-free survival rate at 9 months after inclusion. The registry defines the primary endpoint population as the mITT population, consisting of patients included in the study with at least one radiological assessment within 9 months following inclusion. Its endpoint description counts radiological progression assessed by the investigator, or death from any cause, as failure when occurring within the endpoint period.
Overall survival is listed as a secondary outcome. Numerical results for the primary endpoint and secondary outcome are not reported in the registry record. The record lists 0 analyses, so it does not provide a statistical analysis result that would support an estimate of the treatment effect or its precision.
The registry gives a start date of 2013-05 and a primary completion date of 2017-12, and the study status is completed. It does not report the randomization procedures, a statistical analysis plan, a sample-size justification, or details of any interim analyses. Those omissions prevent a fuller assessment of how the study was planned and how its final endpoint analysis was conducted.
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 | HIA DEBIRI + Systemic FOLFOX |
|---|---|
| STARTED | 58 |
| COMPLETED | 57 |
| NOT COMPLETED | 1 |
Reasons for not completing this period:
| Reason | HIA DEBIRI + Systemic FOLFOX |
|---|---|
| Protocol Violation | 1 |
Analysis population for the primary outcome, as defined in the registry: Primary endpoint was on mITT population meaning all the patients included in the study with at least one radiological assessments within the 9 months following the inclusion
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.
Population: Baseline population is on ITT population meaning all patients included excepted the patient wrongly included (beacuase of pancreatic metastases and not colorectal metastases)
| Characteristic | Unit | HIA DEBIRI + Systemic FOLFOX |
|---|---|---|
| Participants analysed | 57 | |
| Age, Continuous | years | 63.14 (SD 8.57) |
| Sex: Female, Male: Female | Participants | 25 |
| Sex: Female, Male: Male | Participants | 32 |
| Race and Ethnicity Not Collected | Participants | — |
| Region of Enrollment: France | participants | 57 |
6. Primary Endpoint
The registry lists the primary outcome as Progression-free Survival Rate at 9 Months, measured over at 9 months after inclusion.
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint asks whether patients were alive without progression at 9 months after starting treatment. It is a fixed-time measure: the patient must meet both parts of the definition, being alive and having no qualifying progression by the specified assessment point.
The registry defines progression as radiological progression according to RECIST V1.1, assessed by the investigator. Death from any cause within 9 months also counts as failure. If progression and death both occur, the endpoint description says the first event is considered. Thus, this endpoint combines evidence of disease progression with survival rather than measuring either component alone.
In general, a fixed-time progression-free outcome can be useful because it gives a clinically interpretable summary at a stated follow-up point. Combining progression and death also avoids treating a patient who dies before assessment as though their progression status were simply unknown. The interpretation still depends on complete and consistently timed assessment, and the registry does not report details of assessment schedules or handling of incomplete observations.
The stated analysis population is the mITT population: all patients included in the study who had at least one radiological assessment within 9 months following inclusion. The number meeting the endpoint, the estimated rate, and any confidence interval are not reported in the registry record. The endpoint definition therefore identifies what was intended to be measured, but does not by itself show the observed outcome.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry describes a single-group phase II study of HIA DEBIRI with systemic FOLFOX. Its primary outcome is a 9-month progression-free survival rate, but the numerical rate is not reported in the registry record. No confidence interval, p-value, or analysis result for this endpoint is reported either. Consequently, the posted information does not establish how many patients met the endpoint or how precisely the endpoint was estimated.
The endpoint definition is specific about the events counted as failure: investigator-assessed radiological progression under RECIST V1.1, or death from any cause, within 9 months after treatment began. This definition supports a clear description of the intended outcome. It does not supply the observed event data needed to judge the outcome against any benchmark or to compare it with another treatment.
The single-group design is important to interpretation. Without a concurrent control group, the study does not estimate a randomized between-group difference. Any observed outcome, if reported, would describe patients treated in this study; it would not by itself distinguish the effect of the regimen from differences in patient characteristics, disease course, assessment, or other care. A comparison with external experience would require additional assumptions and information that are not reported in the registry record.
Overall survival is listed as a secondary outcome, but its numerical result is not reported in the registry record. The safety summary lists 27 patients affected by serious adverse events among 57 at risk, and 31 deaths. The record does not specify in this summary the timing or cause of those deaths, and it does not establish that a listed event was caused by treatment. These figures should be read as registry safety reporting, not as a comparative estimate of harm.
The registry's enrollment is 58, while the safety summary lists 57 at risk. The relationship between these populations is not explained in the registry record. That difference should not be resolved by assuming that the same eligibility, follow-up, or analysis rules applied to both summaries.
Planning illustrations, where available, are not observed power and do not substitute for endpoint results. The assumptions describe hypothetical two-arm calculations despite the single-group design. No numerical endpoint effect is reported in the registry record, so those assumptions alone cannot establish what a future study would detect.
9. Statistical Methods Explained
The primary outcome is a binary participant-level result: each patient either meets the definition of being alive without progression at 9 months or does not. The registry describes the endpoint unit as participants and the measure as a count of participants. A rate would ordinarily relate the number meeting the definition to the applicable analysis population, but the registry does not report the endpoint count or rate. No new rate can be derived from the information recorded.
What does a confidence interval communicate?
In general, a confidence interval expresses the uncertainty around an estimated quantity under a specified statistical procedure. A narrower interval indicates greater precision under that procedure, while a wider interval indicates less precision. It does not show the range of outcomes for individual patients, nor does it remove limitations in the study design or data. No confidence interval for the primary endpoint is reported in the registry record, so its precision cannot be assessed from the posted information.
Why does the single-group design matter?
In general, a single-group study can describe outcomes among treated participants, but it does not provide a randomized estimate of the difference between treatment strategies. Random allocation helps balance measured and unmeasured factors on average across groups; without it, a difference from an external benchmark could reflect differences in populations, follow-up, assessment, or care. This study's registry record identifies a single group and no comparator, so a comparative causal effect cannot be obtained from the design description alone.
How should statistical significance be read?
In general, a p-value assesses how compatible observed data are with a specified null hypothesis under the assumptions of a statistical test. It is not the probability that a treatment works, and it does not measure the size or clinical importance of an effect. The registry does not report a p-value or a primary endpoint analysis. It also does not report the test, model, or statistical analysis plan used for that endpoint.
What do the planning assumptions mean?
The registry describes sample-size planning illustrations using a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. It says the effect to detect is a stated fraction of the observed effect. For time-to-event calculations, the stated output is the total number of events; for other calculations, it is participants per arm. These are planning conventions, not features of the observed single-group design. Such illustrations are hypothetical design aids, not observed power. The numerical observed effect and numerical planning results are not reported in the registry record, so the illustrations' outputs and inputs cannot be assessed from the record.
Why can over-dispersion matter in general?
For count outcomes, over-dispersion means that variability is greater than a simple statistical model expects. If it is ignored, standard errors can be too small and intervals or tests can be misleading. This is a general consideration, not a description of the analysis in this trial. The primary endpoint is specified as a participant count for a binary outcome, and the registry does not report a dispersion estimate, model, or adjustment for over-dispersion.
The registry lists 0 analyses. It therefore offers endpoint definitions and safety summaries, but no reported inferential analysis from which to evaluate an estimated effect, its uncertainty, or the assumptions of a fitted model. Any fuller statistical interpretation would require the analysis methods and results, which are not reported in the registry record.
10. Multiplicity, Missing Data and Analysis Populations
Multiplicity arises when several outcomes, comparisons, or repeated tests are examined: the chance of at least one apparently notable result can increase if no adjustment or prespecified strategy is used. In general, an analysis plan can identify a primary outcome, define secondary outcomes, and state how multiple tests will be handled. The registry identifies progression-free survival rate at 9 months as the primary endpoint and overall survival as a secondary outcome, but multiplicity procedures are not reported in the registry record.
The registry does not report an analysis plan, interim analyses, or whether any additional outcomes or subgroup analyses were prespecified. It also lists 0 analyses. It is therefore not possible to determine from the registry record which analyses were conducted, whether results were selected for reporting, or how any multiplicity was handled.
The primary analysis population is described as mITT: patients included in the study with at least one radiological assessment within 9 months following inclusion. The registry does not explain how patients without such an assessment were handled, why an assessment might be absent, or whether missing outcome information was imputed or otherwise addressed. In general, missingness can affect an estimate when patients with incomplete assessments differ from those with observed assessments. The direction and size of any such effect cannot be inferred without further information.
The safety summary lists 57 at risk, while the study enrollment is 58. The registry record does not explain the population definitions or reasons for this difference. It also does not describe the handling of missing safety follow-up. These are reporting gaps rather than grounds for assuming a particular analysis rule.
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 |
|---|---|---|---|
| HIA DEBIRI + Systemic FOLFOX | 27 | 57 | 47.4% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| HIA DEBIRI + Systemic FOLFOX | 31 of 57 |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 5% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| HIA DEBIRI + Systemic FOLFOX | 57 of 57 |
Most frequent serious adverse event terms
| Event term | Organ system | HIA DEBIRI + Systemic FOLFOX |
|---|---|---|
| Diarrhoea | Gastrointestinal disorders | 4 of 57 |
| Pancreatitis acute | Gastrointestinal disorders | 4 of 57 |
| Pyrexia | General disorders | 4 of 57 |
| Pancreatitis | Gastrointestinal disorders | 3 of 57 |
| Clostridium difficile colitis | Infections and infestations | 3 of 57 |
| Enteritis | Gastrointestinal disorders | 2 of 57 |
| Large intestinal obstruction | Gastrointestinal disorders | 2 of 57 |
| Asthenia | General disorders | 2 of 57 |
| Chest pain | General disorders | 2 of 57 |
| Cholecystitis | Hepatobiliary disorders | 2 of 57 |
Most frequent non-serious adverse event terms
| Event term | Organ system | HIA DEBIRI + Systemic FOLFOX |
|---|---|---|
| Anemia | Blood and lymphatic system disorders | 56 of 57 |
| Neuropathy | Nervous system disorders | 49 of 57 |
| Alanine aminotransferase increased | Investigations | 49 of 57 |
| Aspartate aminotransferase increased | Investigations | 49 of 57 |
| Phosphatases alcalines increased | Investigations | 47 of 57 |
| Fatigue | General disorders | 43 of 57 |
| Platelets decreased | Investigations | 36 of 57 |
| Neutropenia | Investigations | 36 of 57 |
| Nausea | Gastrointestinal disorders | 32 of 57 |
| Diarrhoea | Gastrointestinal disorders | 31 of 57 |
Serious events with the highest arm-level rate: diarrhoea (7.0%), pancreatitis acute (7.0%), pyrexia (7.0%), pancreatitis (5.3%), clostridium difficile colitis (5.3%).
The safety summary lists 57 patients at risk for serious adverse events and 27 affected. It also lists 57 at risk for other adverse events and 57 affected. The registry record does not provide further detail in this summary about event severity grading, observation period, recurrence, or how multiple events in the same patient were handled. The figures should therefore be read as the registry's participant-level summaries, not as a complete account of event counts or timing.
The listed serious adverse event terms and percentages are diarrhoea, 7.0%; acute pancreatitis, 7.0%; pyrexia, 7.0%; pancreatitis, 5.3%; Clostridium difficile colitis, 5.3%; and enteritis, 3.5%. These are presented as separate terms in the registry. The record does not clarify whether related terms were grouped or whether patients could appear under more than one term.
The summary also reports 31 deaths. It does not state in this summary when the deaths occurred, their causes, or whether they were considered treatment-related. A serious adverse event summary alone cannot establish causation or support a comparison of safety with another regimen. The safety population listed as at risk is 57, while enrollment is 58; the registry does not explain the relationship between these counts.
12. What the Result Does, and Does Not, Mean
The study addresses a defined clinical question: the proportion of patients alive without investigator-assessed radiological progression at 9 months after starting HIA DEBIRI with systemic FOLFOX. The registry provides the endpoint definition, including the use of RECIST V1.1 and the rule that death from any cause within the period counts as failure. That information clarifies what the primary endpoint was intended to capture.
The registry record does not report the numerical primary endpoint result, its uncertainty, or a statistical test. It therefore does not show the observed 9-month progression-free survival rate or how precisely it was estimated. The absence of these results means the endpoint's observed performance cannot be evaluated from the posted information.
The study is single-group and does not describe a concurrent control. Even if an outcome estimate were available, it would describe the studied participants rather than provide a randomized comparison against another strategy. The registry background gives reasons for examining the combined approach, including the stated practical considerations around hepatic intra-arterial chemotherapy, but rationale is not evidence of a comparative effect.
The safety summary gives participant counts and selected serious-event terms, as well as a death count. These data warrant careful reading alongside their limited contextual detail: timing, causes, attribution, and the relationship between the safety population and enrollment are not explained in the registry summary. They cannot establish that an event was caused by treatment or quantify comparative risk.
Overall, the record documents a completed phase II study and defines an endpoint that could be clinically interpretable if its result and analysis were available. It does not provide enough numerical efficacy or inferential information to conclude how the endpoint turned out, nor does it establish that the regimen is better or worse than another approach.
13. Why This Trial Matters Statistically
This trial is statistically notable because its primary question is expressed as a fixed-time binary endpoint in a single-group design. The endpoint asks whether participants are alive and progression-free at 9 months, with progression defined using investigator-assessed radiological criteria and death from any cause counted as failure. Clear event definitions are central to interpreting such an endpoint, even when the result itself is unavailable.
The design also illustrates the difference between describing an outcome and estimating a treatment contrast. A single-group study can document what happened in its enrolled population, but without a concurrent randomized comparator it cannot isolate the contribution of the treatment regimen. This distinction matters when interpreting a rate against historical experience or when considering a later comparative study.
The registry includes a stated mITT population definition requiring at least one radiological assessment within 9 months after inclusion. That definition makes the analysis population part of the endpoint interpretation. The record does not describe how patients without an assessment were handled, so the reader cannot judge whether the analysis population and missing observations affected the result.
The listed planning assumptions further show why design calculations should not be mistaken for trial results. They refer to a hypothetical two-arm allocation, whereas the registered study is single-group. Planning illustrations may inform future research design, but they are not evidence of observed power or of an effect in the study population.
Finally, the distinction between endpoint definition and endpoint reporting is important. Here, the registry contains a detailed primary endpoint description and safety summaries, but no numerical primary efficacy result or inferential analysis. Statistical transparency requires both a prespecified question and enough results and methods for readers to assess the estimate and its uncertainty.
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 for interpretation is that the numerical primary endpoint result is not reported in the registry record. The record also lacks a confidence interval, p-value, and reported analysis method, so neither the size nor precision of the endpoint estimate can be assessed. Overall survival is listed as a secondary outcome, but its numerical result is also not reported.
The single-group design has no concurrent comparator. It can describe outcomes in the treated population but cannot, by itself, separate treatment effects from other influences on outcome. The record does not describe an external comparison strategy or provide information that would support a valid comparative analysis.
The mITT definition requires at least one radiological assessment within 9 months following inclusion. The registry does not report the number of patients who met this definition, the handling of those without a qualifying assessment, or any missing-data method. It also does not describe the assessment schedule beyond the endpoint timeframe or report further details about investigator assessment.
The safety summary lists 57 at risk, compared with enrollment of 58, without explaining the relationship between those populations. It reports 31 deaths but does not give their timing, causes, or attribution in the summary. Serious-event terms and percentages are reported, but the registry does not clarify recurrence, overlap among terms, or the observation period.
The record does not report the sample-size justification, statistical analysis plan, multiplicity approach, or interim-analysis procedures. These omissions limit assessment of whether the final analysis followed a prespecified plan. They do not prove that such procedures were absent; their details are not reported in the registry record.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Intra-arterial Hepatic Beads Loaded With Irinotecan With Concomitant Chemotherapy With FOLFOX in Patients With Colorectal Cancer With Unresectable Liver Metastases: a Phase II Multicenter Study — NCT01839877.
- Registry record last updated 2024-10-08; status last verified 2024-07.
- Results first posted 2024-10-08.
- 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
NCT01839877 is a completed, multicenter phase II study of intra-arterial hepatic beads loaded with irinotecan alongside systemic FOLFOX in patients with colorectal cancer and non-resectable liver metastases. The registry lists enrollment as 58, a single-group model, allocation as not applicable, and masking as none.
The primary endpoint is the progression-free survival rate at 9 months after inclusion. Progression is defined as investigator-assessed radiological progression under RECIST V1.1; death from any cause within the period also counts as failure. The mITT population is defined as included patients with at least one radiological assessment within 9 months following inclusion.
Overall survival is listed as a secondary outcome. Safety summaries include serious and other adverse-event counts, selected serious-event terms, and 31 deaths. Numerical efficacy results, inferential analyses, and further details about missing data, multiplicity, and safety follow-up are not reported in the registry record.