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 Cediranib Maleate and Combination Chemotherapy in Treating Patients With Advanced Biliary Cancers (NCT01229111), a na phase 2 study. The primary outcome is The Response Rate of Patients Evaluated Using the Response Evaluation Criteria in Solid Tumors (RECIST) v1.1.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01229111 |
| Phase | Phase 2 |
| Condition | Adult Primary Cholangiocellular Carcinoma, Advanced Adult Primary Liver Cancer, Cholangiocarcinoma of the Extrahepatic Bile Duct |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 14 |
| Arms in the primary outcome | n/a |
| Lead sponsor | National Cancer Institute (NCI) |
| Results posted | Yes |
2. Clinical Question
Population
The study enrolled patients with advanced biliary cancers, including biliary, gallbladder, liver, periampullary, and extrahepatic bile duct cancer diagnoses listed in the registry record. The primary endpoint applies to patients considered evaluable, but the number evaluable and the criteria for being evaluable are not reported in the registry record.
Intervention
The treatment was cediranib maleate together with modified FOLFOX6. The record describes cediranib maleate by mouth once daily on days 1-14. Modified FOLFOX6 consisted of oxaliplatin and leucovorin calcium given intravenously over 2 hours, and fluorouracil given intravenously over 46 hours, on day 1. Drug doses are not reported in the registry record.
Comparator
The study used a single-group model, with allocation recorded as not applicable and no masking. The record does not describe a concurrent control group or a randomized comparison. Consequently, the posted results cannot establish how outcomes under this regimen compare with outcomes under another treatment or with no treatment.
Primary question
The primary endpoint is titled the response rate assessed using RECIST v1.1. Its registered measure is the number of evaluable participants classified as having a response, with complete response, partial response, progressive disease, and stable disease defined in the endpoint description. The stated timeframe is up to 3 years. The outcome count and response estimate are not reported in the registry record.
3. Trial Design
This was a Phase 2, single-group study sponsored by the National Cancer Institute. The registry record lists enrollment of 14 and marks the study as terminated. It records the start as 2010-10 and the primary completion as 2014-03. The reason for termination is not reported in the registry record.
The treatment group received cediranib maleate with modified FOLFOX6. Cediranib maleate was administered by mouth once daily on days 1-14. On day 1, the modified FOLFOX6 components were oxaliplatin intravenously over 2 hours, leucovorin calcium intravenously over 2 hours, and fluorouracil intravenously over 46 hours. The record does not give drug doses or report treatment adherence.
Allocation is recorded as not applicable, masking as none, and the model as single group. Thus, the design does not include a concurrent treatment arm for estimating a between-group contrast. Its primary endpoint concerns response under the study regimen; it cannot by itself distinguish a treatment effect from other influences on outcomes.
The primary outcome is the number of evaluable patients classified according to RECIST v1.1, with a timeframe of up to 3 years. Listed secondary outcomes concern toxicity, progression-free survival, overall survival, and factors that predict survival. The registry record identifies 5 outcome measures and 0 analyses. Results for these outcomes and details of the analysis methods are not reported in the registry record.
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 | Treatment (Cediranib Maleate and Modified FOLFOX) |
|---|---|
| STARTED | 14 |
| COMPLETED | 13 |
| NOT COMPLETED | 1 |
Reasons for not completing this period:
| Reason | Treatment (Cediranib Maleate and Modified FOLFOX) |
|---|---|
| Death | 1 |
Analysis population for the primary outcome, as defined in the registry: Patients that were evaluable
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: All patients who signed consent.
| Characteristic | Unit | Treatment (Cediranib Maleate and Modified FOLFOX) |
|---|---|---|
| Participants analysed | 14 | |
| Age, Customized: 30-39 years | participants | 1 |
| Age, Customized: 40-49 years | participants | 1 |
| Age, Customized: 50-59 years | participants | 3 |
| Age, Customized: 60-69 years | participants | 8 |
| Age, Customized: 70-79 years | participants | 1 |
| Sex: Female, Male: Female | Participants | 11 |
| Sex: Female, Male: Male | Participants | 3 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 0 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 14 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 |
| Race (NIH/OMB): Asian | Participants | 1 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 |
| Race (NIH/OMB): Black or African American | Participants | 2 |
| Race (NIH/OMB): White | Participants | 11 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Region of Enrollment: United States | participants | 14 |
6. Primary Endpoint
The registry lists the primary outcome as The Response Rate of Patients Evaluated Using the Response Evaluation Criteria in Solid Tumors (RECIST) v1.1, measured over Up to 3 years.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is titled the response rate assessed using Response Evaluation Criteria in Solid Tumors (RECIST) v1.1. The registered measure is a number of participants, among those considered evaluable, and the stated timeframe is up to 3 years. The registry record does not report how many participants were evaluable or how many had each outcome category.
The endpoint description defines complete response as disappearance of all target lesions. Partial response requires at least a 30% decrease in the sum of target-lesion diameters relative to the baseline sum. Progressive disease requires at least a 20% increase relative to the smallest sum on study, along with an absolute increase of at least 5 mm; the appearance of new lesions also qualifies as progression. Stable disease is neither enough shrinkage for partial response nor enough increase for progressive disease, using the smallest on-study sum as the reference.
As a general statistical concept, standardized response criteria make tumor measurements interpretable using prespecified categories rather than an informal judgment of change. A response endpoint can help describe whether target lesions changed during observation. It does not, by itself, show that a treatment caused the change. The single-group design and the absence of a posted response result limit what can be concluded from this endpoint in this record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record describes the study design and primary outcome definition, but it does not report the primary response count, a response estimate, or an analysis of the endpoint. The number of participants evaluable for response is also not reported in the registry record. Therefore, the posted information does not establish how often complete response, partial response, stable disease, or progressive disease occurred in this study group.
Enrollment is listed as 14, while the adverse-event group lists 13 participants at risk for serious events and 13 at risk for other events. Those safety figures are not a substitute for response data. The record does not explain the relation between enrollment and the denominators used for adverse-event reporting, so no reason for that difference can be inferred.
The study has a single-group model and no concurrent control arm. Even if an outcome estimate were available, a single-group result would describe outcomes among participants receiving this regimen; it would not provide a randomized estimate of the difference between the regimen and another treatment. Changes over time, patient characteristics, and other influences cannot be separated from treatment effects using a concurrent comparison that is not part of this design.
Precision also cannot be judged from the primary outcome because neither its observed count nor an uncertainty interval is reported in the registry record. In general, a confidence interval expresses the range of values compatible with the observed data under a statistical model. Without the underlying response results and an analysis, it is not possible to assess the stability of an estimate or how much uncertainty surrounds it.
The record marks the study as terminated, but does not report why. Termination alone does not establish that the regimen was ineffective, unsafe, or infeasible. The listed secondary outcomes include toxicity, progression-free survival, overall survival, and factors that predict survival, but their results are not reported in the registry record. The record also lists 0 analyses, so it does not provide a statistical account with which to interpret those outcomes.
Planning illustrations based on an observed effect, if considered, must be read as hypothetical design calculations rather than as evidence about the trial’s observed power. The assumptions describe a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. Those assumptions do not describe the actual single-group structure. The planning illustration is not the trial’s own design, and it cannot replace the missing observed endpoint results.
9. Statistical Methods Explained
The registry record identifies a binary primary outcome in which participants are classified using RECIST v1.1. The endpoint is titled a response rate, while the registered measure is a number of participants. These are related but not identical summaries: a count describes how many participants fall into a category, while a rate expresses a count relative to an eligible denominator. The number of evaluable participants and the observed response count are not reported in the registry record, so a rate cannot be interpreted from the posted outcome information.
What does the endpoint classify?
RECIST v1.1 assigns tumor response categories using changes in measurements of target lesions and the appearance of new lesions. The registry description specifies thresholds for partial response and progressive disease, and defines stable disease as falling between those categories. Complete response requires disappearance of all target lesions. These definitions establish how the categories are intended to be distinguished; they do not tell readers how many participants met each definition in this study.
For a binary analysis, investigators typically specify which category or categories count as the event of interest and which participants belong in the denominator. That distinction matters because a response rate depends on both the response definition and the population included. The registry record’s endpoint title uses “response rate,” but the description lists all four RECIST categories and does not report an observed summary or analysis population beyond “patients that were evaluable.” The detailed operational rule used to form any response numerator is not reported in the registry record.
Why does the study design matter?
The study is listed as single group, with allocation not applicable and no masking. A single-group study can describe observed outcomes in participants treated under the study regimen. It does not create a concurrent estimate of what would have happened to comparable participants under another treatment. In general, random allocation helps balance known and unknown prognostic factors across treatment groups; it is not part of the design described in this record. As a result, the design does not support a randomized treatment-versus-control contrast.
Masking is recorded as none. In general, masking can reduce the influence of knowledge about treatment assignment on outcome assessment or other trial processes. For a measurement-based endpoint, standardized criteria may help structure assessment, but the registry record does not report the assessment procedures, assessor masking, or how potential assessment differences were handled.
How should precision be assessed?
A point estimate, such as a response proportion, is only part of the statistical result. A confidence interval is a general way to express uncertainty around an estimate: it shows a range of values that are compatible with the data under the chosen model and method. Its width depends on the amount of information and the analysis assumptions. The primary outcome count, an estimate, and a confidence interval are not reported in the registry record, so neither the magnitude nor the precision of response can be assessed.
For a binary endpoint, the number of observed events and the denominator are central to estimating uncertainty. The denominator is especially important when the endpoint applies only to evaluable participants, because participants without an evaluable assessment may be handled differently under different analysis rules. The record does not report how many participants were evaluable, what happened to missing assessments, or whether participants were analyzed according to any broader analysis population.
What do the planning assumptions describe?
The planning assumptions describe a hypothetical calculation using a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. The assumed effect is a stated fraction of an observed effect. These are planning assumptions, not a description of the actual single-group trial design. In general, sample-size calculations estimate how much information a proposed study might need to detect a specified effect under stated assumptions. They are not evidence of the probability that this completed study produced a statistically significant result, and they are never observed power.
The record does not report the study’s own statistical plan, sample-size justification, interim analyses, or the methods used for any endpoint analysis. It also lists 0 analyses. Consequently, there is no posted analysis method to evaluate for model choice, covariate adjustment, handling of repeated assessments, or uncertainty estimation. None of those methods should be assumed from the endpoint definition alone.
Why does over-dispersion matter in general?
For count outcomes, over-dispersion means that observed variability is greater than a simple model assumes. It can make standard errors too small if ignored, and thus make statistical uncertainty appear narrower than it is. This is a general consideration for count-data models; it is not evidence that over-dispersion occurred in this study. The primary outcome is registered as a participant count with response categories, and no count-model analysis or dispersion assessment is reported in the registry record.
10. Multiplicity, Missing Data and Analysis Populations
The record lists the primary RECIST v1.1 response outcome and secondary outcomes concerning toxicity, progression-free survival, overall survival, and factors that predict survival. It also identifies 5 outcome measures and 0 analyses. Results and analysis methods for the outcomes are not reported in the registry record.
Multiplicity arises in general when several outcomes, time points, or statistical comparisons are examined. The more questions assessed, the greater the chance that at least one result may appear notable by chance if each is interpreted separately. A statistical plan may address this through a prespecified primary outcome, testing hierarchy, or adjustment procedure. The registry record does not report a multiplicity strategy, a testing hierarchy, or whether any adjustment was used. It is not possible to determine from the posted information how the listed outcomes were prioritized or interpreted together.
The primary outcome is defined for patients considered evaluable, but the number evaluable and the rules used to determine evaluability are not reported in the registry record. Missing-data methods are also not reported. In general, missing outcome assessments can affect both the denominator and the result, and different assumptions about unobserved outcomes can lead to different estimates. The record does not state whether missing assessments were excluded, counted in a particular category, or addressed through another method.
The analysis population is not described beyond the endpoint’s reference to evaluable patients. The registry record does not report whether a broader treated population was considered or how protocol deviations, treatment discontinuation, or incomplete follow-up were handled. These omissions limit assessment of the primary result even apart from the absence of the result itself.
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 |
|---|---|---|---|
| Treatment (Cediranib Maleate and Modified FOLFOX) | 11 | 13 | 84.6% |
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 |
|---|---|
| Treatment (Cediranib Maleate and Modified FOLFOX) | 13 of 13 |
Most frequent serious adverse event terms
| Event term | Organ system | Treatment (Cediranib Maleate and Modified FOLFOX) |
|---|---|---|
| Neutrophil count decreased | Investigations | 3 of 13 |
| Hypokalemia | Metabolism and nutrition disorders | 3 of 13 |
| Nausea | Gastrointestinal disorders | 2 of 13 |
| Vomiting | Gastrointestinal disorders | 2 of 13 |
| Death | General disorders | 2 of 13 |
| Catheter related infection | Injury, poisoning and procedural complications | 2 of 13 |
| Platelet count decreased | Investigations | 2 of 13 |
| Generalized muscle weakness | Musculoskeletal and connective tissue disorders | 2 of 13 |
| Anemia | Blood and lymphatic system disorders | 1 of 13 |
| Abdominal Pain | Gastrointestinal disorders | 1 of 13 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Treatment (Cediranib Maleate and Modified FOLFOX) |
|---|---|---|
| Alkaline phosphatase increased | Investigations | 13 of 13 |
| Platelet count decreased | Investigations | 12 of 13 |
| Abdominal pain | Gastrointestinal disorders | 11 of 13 |
| Anorexia | Metabolism and nutrition disorders | 11 of 13 |
| Fatigue | General disorders | 11 of 13 |
| Hyponatremia | Metabolism and nutrition disorders | 11 of 13 |
| Nausea | Gastrointestinal disorders | 11 of 13 |
| Peripheral sensory neuropathy | Nervous system disorders | 11 of 13 |
| Aspartate aminotransferase increased | Investigations | 10 of 13 |
| Insomnia | Psychiatric disorders | 10 of 13 |
Serious events with the highest arm-level rate: neutrophil count decreased (23.1%), hypokalemia (23.1%), nausea (15.4%), vomiting (15.4%), death (15.4%).
The registry record lists one treatment group for adverse events. It records 13 participants at risk for serious events, with 11 affected, and 13 at risk for other events, with 13 affected. These are group-level safety counts; they do not establish that every listed event was caused by the treatment. The record does not describe event attribution in the information.
The serious-event terms with listed percentages are neutrophil count decreased and hypokalemia, each at 23.1%; nausea, vomiting, death, and catheter related infection, each at 15.4%. These percentages should be read as the registry’s reported figures for the terms, not as a comparison between treatments. The registry record does not provide a control group for such a comparison.
The separate deaths field is not reported in the registry record. The listed serious-event term “Death” and the missing separate deaths figure should therefore not be treated as interchangeable summaries. Details such as event timing, severity grading, recurrence, relationship to treatment, and handling of multiple events per participant are not reported in the registry record. The posted safety information is not enough to characterize the full safety profile or compare risks across treatments.
12. What the Result Does, and Does Not, Mean
The record establishes that a Phase 2, single-group study of cediranib maleate with modified FOLFOX6 was conducted in patients with advanced biliary cancers and is marked terminated. It also establishes that the primary outcome was defined using RECIST v1.1 and was intended to be assessed among evaluable patients over a timeframe of up to 3 years.
The record does not report the primary response count, response estimate, number evaluable, or an analysis of the endpoint. It therefore does not show what response outcomes occurred in the study group or how precisely those outcomes were estimated. The absence of a posted result is not evidence that there were no responses or that a particular response category predominated.
The single-group design can describe outcomes among participants who received the regimen, but it cannot supply a randomized comparison with another treatment. Any observed outcomes would also need to be interpreted in light of the broad set of biliary cancer diagnoses listed and the study’s eligibility and assessment procedures, which are not fully described in the registry record.
The record lists serious and other adverse-event counts and selected serious-event terms with percentages. Those figures are descriptive. They do not establish causation, and the record does not report sufficient detail to compare harms with another treatment. The terminated status does not explain why the study ended; the reason is not reported in the registry record.
Overall, the registry record conveys the trial’s treatment structure, endpoint definitions, and selected safety information, but it does not provide the outcome analyses needed to judge efficacy or precision. It neither establishes that the regimen works nor establishes that it does not work.
13. Why This Trial Matters Statistically
This trial is statistically informative as an example of the distinction between an endpoint definition and an endpoint result. The registry record gives detailed RECIST v1.1 criteria, including the thresholds that distinguish partial response and progressive disease. Those criteria clarify what investigators intended to measure, but without the observed count and evaluable denominator they do not answer how participants fared.
Its single-group design also illustrates why the type of comparison matters. A response estimate from a treatment group can describe what happened during the study, but a causal comparison with another regimen requires a suitable comparator and a design that supports that contrast. Neither a concurrent comparator nor a randomized allocation is recorded. Any later interpretation of an observed outcome would need to be correspondingly cautious.
The record also highlights how outcome reporting supports interpretation. It lists secondary outcomes for toxicity, progression-free survival, overall survival, and survival-predictive factors, yet does not report their results or analyses. Without analysis populations, missing-data methods, and uncertainty estimates, readers cannot assess the robustness or precision of findings. These are important elements of statistical reporting, not merely technical additions.
Finally, the safety figures demonstrate why denominators and definitions matter. Counts for participants at risk and affected are available, alongside selected percentages for serious-event terms, but details about event attribution, timing, and analysis are not reported. The figures are useful descriptive information while remaining insufficient for comparative or causal conclusions. Together, these features make the record a case study in reading design, outcome definitions, and reporting completeness separately.
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 principal limitation is that the registry record does not report the primary response outcome or any endpoint analysis. The number of evaluable participants, response counts by category, estimate, and uncertainty interval are not reported. This prevents assessment of both the observed outcome and its precision.
The study’s single-group model and absence of a concurrent comparator limit interpretation to description of outcomes under the regimen; they do not support a randomized between-treatment comparison. The record marks allocation as not applicable and masking as none. It does not report assessment procedures or describe how potential bias in outcome measurement was addressed.
Enrollment is listed as 14, and the status is terminated. The reason for termination is not reported in the registry record. The record does not provide details about recruitment, follow-up completeness, treatment exposure, or discontinuations, so their influence on the available outcomes cannot be assessed.
The population includes a range of advanced biliary cancer diagnoses. The record does not report subgroup results or enough information to determine how outcomes varied across these diagnoses. The primary endpoint applies to evaluable patients, but the evaluability rules and missing-data methods are not reported. The adverse-event group lists 13 participants at risk, while the enrollment figure is 14; the registry record does not explain the difference, and it should not be interpreted as evidence about why any participant is absent from that safety denominator.
Finally, the registry record lists 0 analyses and does not report a statistical analysis plan, sample-size justification, interim analyses, or multiplicity strategy. These missing details constrain evaluation of the endpoint definitions and selected safety summaries. The posted record alone cannot resolve these limitations.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Cediranib Maleate and Combination Chemotherapy in Treating Patients With Advanced Biliary Cancers — NCT01229111.
- Registry record last updated 2017-03-29; status last verified 2017-02.
- Results first posted 2017-03-29.
- 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
This National Cancer Institute-sponsored Phase 2 study examined cediranib maleate with modified FOLFOX6 in advanced biliary cancers. It used a single-group design, with allocation not applicable and no masking. Enrollment was 14; the study is marked terminated. The recorded start is 2010-10 and primary completion is 2014-03.
The primary endpoint was the number of evaluable participants assessed for response using RECIST v1.1, with a timeframe of up to 3 years. Listed secondary outcomes concern toxicity, progression-free survival, overall survival, and factors that predict survival. The registry record lists 0 analyses and does not report the primary outcome result, the number evaluable, or a reason for termination.
Safety information includes counts for serious and other adverse events and selected serious-event terms with percentages. These are descriptive figures, not a treatment comparison. The registry record does not provide the analyses or outcome results needed to assess response, precision, or comparative effects.