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 Randomized Phase II Adjuvant Chemotherapy ± FANG™ in Colorectal Carcinoma With Liver Metastases (NCT01505166), a randomized phase 2 study. The primary outcome is Percent of Patients Who Progressed After Treatment (Part 2).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01505166 |
| Phase | Phase 2 |
| Condition | Colon Cancer |
| Allocation | Randomized |
| Design model | Parallel |
| Masking | Single |
| Enrollment | 3 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Gradalis, Inc. |
| Results posted | Yes |
2. Clinical Question
Population
People with colorectal carcinoma and liver metastases after surgery were the intended study population. The registry describes a Part 1 vaccine run-in and a Part 2 randomized comparison. It records 3 participants enrolled and states that no subjects were randomized in Part 2.
Intervention
The intervention was Vigil™, an autologous tumor cell vaccine given by intradermal injection. The registry describes a minimum of 4 doses and a maximum of 12 doses for the randomized study arms. The run-in description specifies intradermal vaccine administration, with a maximum of 12 vaccinations.
Comparator
The planned comparator was placebo, also given by intradermal injection. The registry describes Vigil™ and placebo as the two Part 2 arms, but reports that no participants were randomized to those arms.
Primary question
The primary outcome was the percent of patients who progressed after treatment, assessed over 24 months in Part 2. Because no participants were randomized in Part 2 and no primary-outcome results are reported, the record does not establish a treatment-group comparison for progression.
The clinical question is therefore a planned comparison rather than a completed randomized efficacy comparison. The study record identifies a randomized, single-masked, parallel Phase II design. Those design features describe the intended structure; they do not mean that a randomized comparison was carried out. The registry specifically says that Part 2 had no randomized subjects and that the study was terminated.
The run-in and randomized portions should be kept distinct when interpreting the record. The run-in was intended to give Vigil™ to 6 patients. Safety data list 3 participants at risk in the run-in group. The total enrollment is also recorded as 3. These details do not provide comparative evidence about Vigil™ versus placebo, because the randomized comparison did not proceed to enrollment.
The registry’s primary outcome description also refers to tumor response using RECIST Version 1.1. It describes complete response, partial response, stable disease, and progressive disease, and states that a response requires confirmatory staging at least 4 weeks apart. These are outcome definitions, not reported response findings. No comparison of those response categories is given for the planned randomized arms.
3. Trial Design
This was a randomized, single-masked, parallel Phase II trial of post-operative adjuvant chemotherapy with or without Vigil™ vaccine in colorectal carcinoma with liver metastases. The registry identifies Vigil™ and placebo as the planned Part 2 groups. The record lists 3 participants enrolled and states that no subjects were randomized in Part 2. The study status is terminated.
The registry describes a two-part study. Part 1 was a vaccine run-in intended for 6 patients, who would receive intradermal autologous Vigil™ vaccine. The planned Part 2 was the randomized comparison between Vigil™ and placebo. In the Part 2 arm descriptions, treatment was to begin post-surgery Week 4-8, with a minimum of 4 doses and a maximum of 12 doses. The schedule is described as continuing at specified cycle visits and then every 28 days. The record does not report that the planned Part 2 treatment schedule was delivered to randomized participants.
The distinction between planned design and actual conduct is central. Random allocation, masking, and parallel groups are registered design features, but the absence of Part 2 randomization means the record contains no randomized comparison of outcomes. The reported enrollment of 3 should not be read as 3 participants in each arm, or as a completed comparison; the registry does not report such allocation.
The registry lists 5 outcome measures. The primary measure is the percent of patients who progressed after treatment in Part 2, with a 24 months timeframe. It also describes response assessment using RECIST Version 1.1. The registry record does not report the detailed statistical analysis plan, sample-size justification, interim analyses, or analysis populations. It records no analysis entries. The sponsor is Gradalis, Inc.; the recorded start is 2012-03, and primary completion is 2016-08. These dates describe the registry record, not evidence that the planned comparative endpoint was assessed.
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.
Before assignment: 3 subjects were enrolled in Part 1 and completed Vigil plus chemotherapy. No other subjects were enrolled in Part 1 and also Part 2.
Overall Study
| Stage | Vigil™ Vaccine (Part 1) - 6 Patient run-in | Vigil™ (Part 2) | Placebo (Part 2) |
|---|---|---|---|
| STARTED | 3 | 0 | 0 |
| COMPLETED | 3 | 0 | 0 |
| NOT COMPLETED | 0 | 0 | 0 |
Analysis population for the primary outcome, as defined in the registry: 3 subjects were enrolled in Part 1 and completed Vigil plus chemotherapy. However, TIL was not tested/collected so no correlation could be performed. Statistical analysis was not done. This study was terminated.
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: Study closed to enrollment and Part 2 was not initiated.
| Characteristic | Unit | Vigil™ Vaccine (Part 1) 6 Patient run-in | Vigil™ (Part 2) | Placebo (Part 2) | Total |
|---|---|---|---|---|---|
| Participants analysed | 3 | 0 | 0 | 3 | |
| Age, Customized: 0-15 Years | Participants | 0 | — | — | 0 |
| Age, Customized: 16-64 Years | Participants | 2 | — | — | 2 |
| Age, Customized: 65 Years and Older | Participants | 1 | — | — | 1 |
| Sex: Female, Male: Female | Participants | 2 | — | — | 2 |
| Sex: Female, Male: Male | Participants | 1 | — | — | 1 |
| Race/Ethnicity, Customized: White/Caucasian | Participants | 3 | — | — | 3 |
| Race/Ethnicity, Customized: Black/African American | Participants | 0 | — | — | 0 |
| Race/Ethnicity, Customized: Asian | Participants | 0 | — | — | 0 |
| Race/Ethnicity, Customized: Hispanic | Participants | 0 | — | — | 0 |
| Race/Ethnicity, Customized: Other | Participants | 0 | — | — | 0 |
6. Primary Endpoint
The registry lists the primary outcome as Percent of Patients Who Progressed After Treatment (Part 2), measured over 24 months.
| Element | Registry entry |
|---|---|
| Result type | N/A |
| Measure of spread | not applicable |
| Unit | not stated |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint is named as the percent of patients who progressed after treatment in Part 2, with assessment over 24 months. It is intended to describe the proportion of the relevant study population experiencing progression during the specified follow-up period. The registry does not give a numerical progression result, a denominator for an analyzed Part 2 population, or a treatment-group estimate.
The outcome description links tumor response assessment to RECIST Version 1.1. It defines response categories as complete response, partial response, stable disease, and progressive disease. For patients with measurable disease, any response, defined as complete or partial response, requires two confirmatory staging assessments at least 4 weeks apart. Patients are described as evaluable for tumor response if measurable disease is present.
In general, a progression endpoint is used to characterize whether disease has worsened according to prespecified assessment criteria over a follow-up period. Such an endpoint can be clinically interpretable when its assessment rules, follow-up, and analysis population are clear. It does not by itself describe every aspect of a patient’s experience or establish the cause of a change in disease status.
For this trial, the primary endpoint cannot support a treatment comparison in the registry record because no subjects were randomized in Part 2. No progression percentages, response-category counts, or estimates of uncertainty are reported. The endpoint definition clarifies what the study intended to measure, but it is not an outcome result.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The central statistical fact is that the planned randomized Part 2 comparison did not have randomized participants. The registry reports 3 participants enrolled overall, identifies a run-in cohort in the safety record, and explicitly states that no subjects were randomized in Part 2. Consequently, the record does not provide a randomized estimate comparing Vigil™ with placebo for progression or tumor response.
The primary outcome is defined as the percent of patients who progressed after treatment over 24 months. No value for that percentage is reported. There are also no reported group-specific outcome counts, treatment-effect estimate, confidence interval, or hypothesis-test result. Without those data, the direction and precision of a comparative effect cannot be assessed. The registered design alone does not supply evidence of an effect.
The safety entries are limited to the run-in cohort. They list 3 participants at risk for serious adverse events and 1 affected participant, as well as 3 at risk for other adverse events and 3 affected participants. The randomized Vigil™ and placebo entries list 0 at risk. These counts do not support a comparison between randomized arms: those arms had no reported randomized participants. Nor do the run-in counts establish a treatment contrast, because the registry provides no concurrent randomized comparator data for that group.
In general, estimates based on small cohorts are often imprecise, and a lack of observed comparative results is different from evidence that two interventions have the same outcome. Here, there is no Part 2 estimate whose precision can be evaluated. The absence of a reported estimate should not be interpreted as a zero effect, a null result, or a finding of equivalence.
The registry lists 5 outcome measures but no analysis entries. It does not report how outcome data were analyzed, how missing observations were handled, or which analysis population was used. It also does not report an interim-analysis plan or a multiplicity strategy. These omissions limit evaluation of how the intended analyses would have been conducted, even apart from the lack of randomized outcome data.
Any sample-size illustration based on an observed effect needs an effect estimate to serve as its starting point. The record does not report a Part 2 outcome estimate from which to assess the proposed clinical comparison. Planning illustrations are not observed power, and they cannot replace results from a randomized comparison. The registry’s stated planning assumptions include a two-sided significance level of 0.05, equal allocation, and a normal approximation; they do not constitute evidence that these assumptions were used to justify the trial’s original enrollment target.
Overall, the record describes a research question and a planned design, along with limited run-in safety information. It does not report the primary endpoint results needed to answer the randomized treatment question. Interpretation should remain focused on that distinction between trial intent and reported evidence.
9. Statistical Methods Explained
Statistical methods help translate trial observations into estimates and quantify how much uncertainty remains. In this trial record, the registered design and endpoint are described, but no Part 2 randomized outcome analysis is reported. The methods concepts below are general explanations; they should not be taken as a description of unreported analyses in this study.
What randomization is intended to do
In general, randomization assigns participants to study groups by chance. When implemented, it can reduce systematic differences between groups at the start of a study, so an observed outcome contrast is less likely to reflect pre-existing group differences. It does not guarantee that groups will be identical, especially in small studies. The registry calls this trial randomized, but also states that no subjects were randomized in Part 2. Thus randomization describes the planned design rather than a realized basis for comparing outcomes in Part 2.
What a parallel design means
In a parallel-group design, participants are assigned to a group and followed under that group’s planned intervention. The registry describes Vigil™ and placebo as the Part 2 groups. A treatment comparison would ordinarily use outcomes observed in those groups, with an analysis that matches the endpoint and follow-up. Because the record reports no Part 2 randomization and no group outcome data, it does not permit such a comparison.
What masking can and cannot establish
Masking is intended to limit the influence of knowledge about assigned treatment on aspects of trial conduct or outcome assessment. The registry identifies the study as single-masked, but does not report who was masked or how masking was maintained. Those details are not reported in the registry record. Masking also cannot compensate for the absence of randomized Part 2 participants or missing endpoint results.
How to interpret an endpoint percentage
In general, a percentage summarizes a count relative to a defined denominator. Interpreting it requires knowing which participants are included, how the outcome is assessed, and how missing observations are treated. The primary endpoint is described as the percent of patients who progressed after treatment over 24 months. The registry does not report a Part 2 numerator, denominator, or percentage. Therefore, no endpoint percentage can be interpreted from the record.
The record refers to RECIST Version 1.1 and describes complete response, partial response, stable disease, and progressive disease. It also specifies that a response requires two confirmatory staging assessments at least 4 weeks apart. In general, explicit outcome criteria help make assessments more consistent across participants. However, defining an outcome does not show how many participants met it, and no category results are reported for Part 2.
Confidence intervals and hypothesis tests
In general, a confidence interval expresses the range of values compatible with an estimate under a specified statistical procedure and its assumptions. A narrow interval indicates greater precision than a wide interval, while neither alone establishes clinical importance. A hypothesis test evaluates compatibility between data and a specified null hypothesis; its result depends on the data, model, and analysis choices. No treatment-effect estimate, confidence interval, or hypothesis-test result is reported for the primary endpoint in this registry record. There is consequently no basis for describing statistical significance or precision for the planned comparison.
What the planning assumptions mean
The registry describes planning illustrations based on an observed effect, using a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. In general, a significance level is a design parameter for a hypothesis test, and equal allocation is an assumption about how participants would be divided between groups. A normal approximation uses a mathematical approximation to derive planning quantities. For time-to-event planning, the record says the total number of events is used; for other outcomes, participants per arm are used.
The record also describes planning for a stated fraction of the observed effect. Such illustrations explore what sample size might be needed under stated assumptions, rather than reporting what happened in the trial. They are not observed power. The registry record does not report the original protocol’s sample-size justification, so the planning assumptions should not be attributed to that protocol. With no reported Part 2 outcome estimate, there is no comparative observed effect in the record to interpret as a trial finding.
Why analysis details matter
Statistical conclusions depend not only on endpoint definitions but also on decisions about populations, follow-up, missing observations, and the model used. The registry lists no analysis entries. The analysis method, covariate adjustment, handling of repeated assessments, and handling of missing data are not reported in the registry record. It is therefore not possible to verify whether a particular analysis would have aligned with the trial’s intended question.
In general, a prespecified analysis can reduce the risk that analytic decisions are driven by observed results. The registry record does not report a detailed statistical analysis plan or interim analyses. No assumptions about those procedures should be inferred from the study’s Phase II designation or randomized design label. For this record, the most important method-related distinction is straightforward: the protocol description specifies a comparative question, but the reported conduct and results do not supply a randomized comparison with which to answer it.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists 5 outcome measures and no analysis entries. It identifies the percent of patients who progressed after treatment in Part 2 as the primary outcome, with a 24 months timeframe, and also describes tumor response categories under RECIST Version 1.1. The record does not explain how the outcomes were prioritized in the analysis beyond identifying the primary measure, or whether any multiplicity adjustment was planned.
In general, multiplicity arises when several outcomes, comparisons, or time points are tested. If many tests are conducted, the chance of at least one apparently unusual result can increase. Statistical strategies can address that issue, but the choice depends on the analysis plan and research question. The multiplicity strategy for this trial is not reported in the registry record. No adjustment or unadjusted testing approach should be assumed.
The registry does not report the handling of missing outcome measurements, incomplete follow-up, or participants who could not be evaluated. These details are not reported in the registry record. In general, missing-data methods can affect which participants contribute to an estimate and how much uncertainty surrounds it. Without a stated method or outcome data, their influence cannot be assessed.
The analysis population is also not reported. The record does not state whether an intended analysis would include all randomized participants, only participants who received treatment, or another defined group. In Part 2, the registry explicitly reports no randomized subjects, so there is no randomized population for a treatment comparison in the posted results. The absence of these analysis details limits reproducibility and interpretation, but it does not create evidence of a treatment effect or of no effect.
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 |
|---|---|---|---|
| Vigil™ Vaccine (6 Patient run-in) | 1 | 3 | 33.3% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Vigil™ | 0 of 0 |
| Placebo | 0 of 0 |
| Vigil™ Vaccine (6 Patient run-in) | 1 of 3 |
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 |
|---|---|
| Vigil™ | 0 of 0 |
| Placebo | 0 of 0 |
| Vigil™ Vaccine (6 Patient run-in) | 3 of 3 |
Most frequent serious adverse event terms
| Event term | Organ system | Vigil™ | Placebo | Vigil™ Vaccine (6 Patient run-in) |
|---|---|---|---|---|
| Thromboembolic Event | Vascular disorders | 0 of 0 | 0 of 0 | 1 of 3 |
| Urinary Tract Infection | Infections and infestations | 0 of 0 | 0 of 0 | 1 of 3 |
| Fracture | Musculoskeletal and connective tissue disorders | 0 of 0 | 0 of 0 | 1 of 3 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Vigil™ | Placebo | Vigil™ Vaccine (6 Patient run-in) |
|---|---|---|---|---|
| Injection Site Reaction | General disorders | 0 of 0 | 0 of 0 | 3 of 3 |
Serious events with the highest arm-level rate: thromboembolic event (33.3%), urinary tract infection (33.3%), fracture (33.3%).
The safety record separates the planned Vigil™ and placebo arms from the Vigil™ vaccine run-in group. The Vigil™ and placebo entries each list 0 participants at risk for serious adverse events and 0 at risk for other adverse events. Since no participants were randomized in Part 2, those zero-at-risk entries should not be read as evidence that no adverse events occurred among treated randomized participants. The record lists no participants at risk in those groups.
For the run-in group, the registry lists 3 participants at risk for serious adverse events and 1 affected participant. It lists 3 at risk for other adverse events and 3 affected participants. The serious adverse event terms highlighted in the record are Thromboembolic Event, Urinary Tract Infection, and Fracture, each reported as 33.3%. The record also lists 1 death in the run-in group.
These are descriptive safety counts from a small run-in cohort, not comparative estimates. The registry does not report a randomized safety comparison, event timing, or whether the listed events or death were judged related to study treatment. Causality is not established by the counts alone. The safety figures should therefore be read as limited event reporting, with the cohort and the lack of a randomized comparator kept in view.
12. What the Result Does, and Does Not, Mean
The result that can be stated from the registry is about study conduct, not treatment efficacy: the study was terminated, 3 participants were enrolled, and no subjects were randomized in Part 2. The primary endpoint was defined as the percent of patients who progressed after treatment over 24 months, but the registry does not report a primary-endpoint result.
This means there is no randomized estimate comparing Vigil™ with placebo for progression in the posted results. There are no reported treatment-group percentages, response comparisons, confidence intervals, or hypothesis-test results for the planned Part 2 comparison. It is therefore not possible to judge the size, direction, or precision of a comparative treatment effect from this record.
In general, an unavailable or unreported estimate should not be interpreted as evidence that an intervention has no effect, that it has an effect, or that two groups are equivalent. Equivalence or non-inferiority claims require a prespecified framework and analysis; neither is reported. The registry’s description of a randomized design does not change the fact that Part 2 had no randomized participants.
The run-in safety information has a narrower meaning. It records adverse event counts among the 3 participants listed at risk in that group, including 1 participant with a serious adverse event and 3 with other adverse events. Those observations describe the recorded run-in experience only. They do not establish comparative safety against placebo, and the record does not attribute the listed events or death to treatment.
The registry provides useful information about the intended research question, the vaccine and placebo arms, and the outcome definition. It does not provide the randomized outcome evidence needed to answer the question. Readers should distinguish a protocol’s planned comparison from results generated by that comparison and avoid treating enrollment or a design label as a substitute for an endpoint estimate.
13. Why This Trial Matters Statistically
This trial illustrates why the distinction between intended design and completed analysis matters. A randomized label indicates how a comparison was planned, but a treatment-effect estimate requires data from the randomized groups. The registry explicitly states that no subjects were randomized in Part 2. Thus the most important statistical conclusion is not a numerical efficacy finding; it is that the planned randomized comparison is unavailable in the reported record.
The record also separates endpoint specification from endpoint evidence. It defines progression over 24 months and refers to RECIST Version 1.1 response categories, including a confirmation requirement for responses. Such definitions can make an outcome more reproducible, but they do not themselves provide event counts or estimates. The primary outcome result is not reported.
The limited run-in safety counts illustrate a second statistical principle: descriptive observations from one group do not form a comparative treatment effect. Although the record lists participants at risk and affected in the run-in, the planned placebo group has no reported randomized participants. The run-in counts therefore cannot answer whether the groups differ in safety or outcome.
The record further shows why transparent analysis reporting matters. It lists 5 outcome measures and no analysis entries. Details about multiplicity, missing data, analysis populations, and the original sample-size justification are not reported in the registry record. Without those details, readers cannot assess the intended analytical safeguards or reproduce a comparative analysis. Taken together, the record is informative about the question and planned structure, while its reported conduct does not permit the central randomized treatment comparison.
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 central limitation is that the registry states no subjects were randomized in Part 2. The intended comparison between Vigil™ and placebo therefore has no reported randomized outcome data. The record gives no primary progression percentage, response-category results, treatment-effect estimate, or measure of statistical uncertainty.
Overall enrollment is recorded as 3, while Part 1 was planned for 6 patients. The registry safety entries list 3 participants at risk in the run-in group. The record does not explain the full disposition of participants or why enrollment did not reach the planned run-in number. That information is not reported in the registry record and should not be inferred.
The study is marked terminated, but the reason for termination is not reported in the registry record. The record also does not report the detailed analysis plan, original sample-size justification, interim analyses, missing-data procedures, or analysis populations. These gaps limit assessment of the planned statistical approach and the intended handling of incomplete observations.
Safety interpretation is restricted to descriptive run-in counts. The registry does not report randomized safety data for the Vigil™ and placebo groups, and does not state whether the listed events or death were considered related to treatment. The event counts cannot establish comparative risks or causal effects.
Finally, the primary outcome timeframe and response definitions describe the intended assessment, but the record does not provide the observed results needed to evaluate follow-up or endpoint ascertainment. The limitations concern both the absence of randomized outcome data and the lack of reported analytic details; neither should be filled in by assumptions about how the trial might have been conducted.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Randomized Phase II Adjuvant Chemotherapy ± FANG™ in Colorectal Carcinoma With Liver Metastases — NCT01505166.
- Registry record last updated 2021-11-01; status last verified 2021-10.
- Results first posted 2018-05-01.
- 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
The registry describes a randomized, single-masked, parallel Phase II study of post-operative adjuvant chemotherapy with or without Vigil™ in colorectal carcinoma with liver metastases. The planned Part 2 comparison was Vigil™ versus placebo. The primary endpoint was the percent of patients who progressed after treatment over 24 months, with tumor response described using RECIST Version 1.1.
The record lists 3 participants enrolled and states that no subjects were randomized in Part 2. It marks the study terminated and reports no primary outcome estimate or analysis entries. Safety information is listed for the Vigil™ vaccine run-in group, with 3 participants at risk, and does not provide a randomized safety comparison. The reason for termination and detailed statistical analysis plan are not reported in the registry record.