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 Two-part Phase IIb Trial of Vigil (Bi-shRNAfurin and GMCSF Augmented Autologous Tumor Cell Immunotherapy) in Ewing's Sarcoma (NCT02511132), a randomized phase 2 study. The primary outcome is Number of Participants With Adverse Events Determined by Laboratory Assessments and Physical Examinations.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02511132 |
| Phase | Phase 2 |
| Condition | Ewing's Sarcoma |
| Allocation | Randomized |
| Design model | Parallel |
| Masking | None (open label) |
| Enrollment | 22 |
| Arms in the primary outcome | 3 |
| Lead sponsor | Gradalis, Inc. |
| Results posted | Yes |
2. Clinical Question
Population
The registry describes patients with metastatic Ewing's sarcoma. Part 2 was intended for patients whose disease was refractory or who were intolerant to at least 1 prior line of systemic chemotherapy. The specific eligibility details and characteristics of participants in each part are not reported in the registry record.
Intervention
Part 1 evaluated Vigil immunotherapy alone. Part 2 evaluated Vigil in combination with temozolomide and irinotecan, with peg-filgrastim also described in the regimen. The stated primary objective for the combination was to determine its safety profile.
Comparator
In Part 1, the comparator was gemcitabine and docetaxel. Part 2 had a combination-treatment arm; a comparator for that part is not reported in the registry record. The registry identifies the trial as randomized, unmasked, and parallel, but does not report allocation details for each part.
Primary question
The listed primary outcome is the number of participants with adverse events determined by laboratory assessments and physical examinations, assessed through 30 days of last treatment dosing. The registry's description assigns a safety objective to Vigil combined with irinotecan and temozolomide. Separately, Part 1's stated objective was to compare overall survival between Vigil and gemcitabine and docetaxel. Although progression-free survival and overall survival are listed as secondary outcomes, comparative survival estimates are not reported in the registry record.
3. Trial Design
NCT02511132 is listed as a randomized, unmasked, parallel Phase 2 trial, with the title describing it as a two-part Phase IIb trial. The registry reports enrollment of 22 participants and status as completed. The trial record lists Ewing's sarcoma as the condition and Gradalis, Inc. as the sponsor. The recorded start date is 2016-02-10, and the primary completion date is 2018-11-12.
Part 1 randomized participants to Vigil alone or gemcitabine and docetaxel. The posted arm counts are 5 participants for Vigil alone and 8 for gemcitabine and docetaxel. Vigil was described as 1.0 x 107 cells per injection, with a minimum of 4 and maximum of 12 administrations every 28 days. The chemotherapy regimen was described as gemcitabine 675 mg/m2 intravenously on Day 1 and docetaxel 75 mg/m2 intravenously starting on Day 8, on a 21-day schedule.
Part 2 enrolled participants to Vigil with temozolomide and irinotecan; the posted arm count is 9. Temozolomide was described as oral dosing on Days 1–5. The record describes irinotecan on Days 1–5, with oral and intravenous options, and lists peg-filgrastim on Day 6. Further details of the Part 2 regimen are truncated in the registry record. The record does not report a comparator for Part 2 or allocation details that would establish whether the randomized design applied to that part.
The trial's stated objectives differ by part. Part 1 was intended to compare overall survival between its treatment arms. Part 2 was intended to assess the safety profile of the combination. The listed primary outcome is a count of participants with adverse events based on laboratory assessments and physical examinations, through 30 days of last treatment dosing. Progression-free survival and overall survival are listed as secondary outcomes. Outcome estimates for those survival measures 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 | Part 1: Vigil Alone | Part 1: Gemicitabine and Docetaxel | Part 2: Vigil in Combination With Temozolomide and Irinotecan |
|---|---|---|---|
| STARTED | 5 | 8 | 9 |
| COMPLETED | 5 | 7 | 9 |
| NOT COMPLETED | 0 | 1 | 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.
Population: All subjects registered to treatment
| Characteristic | Unit | Part 1: Vigil Alone | Part 1: Gemicitabine and Docetaxel | Part 2: Vigil in Combination With Temozolomide and Irinotecan | Total |
|---|---|---|---|---|---|
| Participants analysed | 5 | 8 | 9 | 22 | |
| Age, Categorical: <=18 years | Participants | 0 | 4 | 3 | 7 |
| Age, Categorical: Between 18 and 65 years | Participants | 5 | 4 | 6 | 15 |
| Age, Categorical: >=65 years | Participants | 0 | 0 | 0 | 0 |
| Sex: Female, Male: Female | Participants | 3 | 6 | 4 | 13 |
| Sex: Female, Male: Male | Participants | 2 | 2 | 5 | 9 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 1 | 1 | 1 | 3 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 4 | 7 | 8 | 19 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Asian | Participants | 0 | 1 | 0 | 1 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Black or African American | Participants | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): White | Participants | 5 | 7 | 9 | 21 |
| Race (NIH/OMB): More than one race | Participants | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 | 0 | 0 | 0 |
| Region of Enrollment: United States | participants | 5 | 8 | 9 | 22 |
6. Primary Endpoint
The registry lists the primary outcome as Number of Participants With Adverse Events Determined by Laboratory Assessments and Physical Examinations, measured over 30 days of last treatment dosing.
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is the number of participants with adverse events determined by laboratory assessments and physical examinations, measured through 30 days of last treatment dosing. It is a count of participants, rather than a measure of the number of separate events or their duration. A participant may have more than one adverse event, but a participant count does not convey the total event burden or distinguish repeated events.
A safety endpoint is used to describe undesirable effects observed during treatment and the stated follow-up period. Laboratory assessments and physical examinations are ways to identify events that may not be captured by participant reports alone. General statistical context: a count is straightforward to interpret as the number of participants meeting the endpoint definition, while interpretation also depends on the number assessed and the consistency of event collection.
The registry posts counts of 5 participants in the Vigil-alone arm, 6 in the gemcitabine-and-docetaxel arm, and 9 in the Vigil-combination arm for the listed primary outcome. The corresponding arm counts are 5, 8, and 9. These are descriptive results; comparative estimates and uncertainty intervals are not reported in the registry record. The listed primary outcome does not itself answer the stated Part 1 objective of comparing overall survival. Overall survival and progression-free survival appear among the secondary outcomes, but their results are not reported in the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The posted primary outcome is a participant count for adverse events identified through laboratory assessments and physical examinations within 30 days of last treatment dosing. The registry reports 5 participants in Part 1's Vigil-alone arm, 6 in the gemcitabine-and-docetaxel arm, and 9 in the Part 2 combination arm. The corresponding posted arm counts are 5, 8, and 9. These figures describe the recorded outcome counts; they do not provide a treatment-effect estimate or quantify uncertainty.
The trial has distinct objectives across its parts. Part 1 was described as a randomized comparison of overall survival between Vigil and gemcitabine and docetaxel. Part 2 was intended to assess the safety profile of Vigil with temozolomide and irinotecan. The primary outcome shown in the results is a safety count, while overall survival and progression-free survival are listed as secondary outcomes. Comparative survival results are not reported in the registry record. Therefore the posted primary outcome counts cannot be interpreted as answering the Part 1 survival question.
The adverse-event record gives additional descriptive counts by arm. Serious adverse events are reported for 2 of 5 participants with Vigil alone, 5 of 8 with gemcitabine and docetaxel, and 5 of 9 with the combination. Other adverse events are reported for 2 of 5, 5 of 8, and 9 of 9, respectively. Deaths are recorded for 4, 8, and 9 participants, respectively. These arm-level records are not adjusted comparisons, and the registry does not report an analysis estimating differences between treatment groups.
The serious-event terms with listed percentages include vomiting at 22.2%, edema extremities at 20.0%, disease progression at 20.0%, back pain at 20.0%, dyspnea at 20.0%, and brain mass at 12.5%. The record does not provide enough detail alongside these terms to treat them as a complete account of event severity, timing, or attribution. A term's listed percentage should not be read as a comparative treatment effect.
Overall, the posted results are descriptive counts from a small completed trial. They do not establish whether one Part 1 treatment produced longer survival, nor do they establish comparative safety. Survival estimates, confidence intervals, hypothesis tests, and adjusted analyses are not reported in the registry record. The counts should be read in the context of the distinct parts, their different objectives, and the limited inferential information recorded.
9. Statistical Methods Explained
General explanation: statistical methods determine how an endpoint is summarized and how uncertainty is assessed. For a participant-count endpoint, the basic descriptive result is the number of participants meeting the event definition in each arm. A comparative analysis would then need to specify the effect measure, its uncertainty, and the method used to account for the trial design. The registry reports counts, but comparative estimates and uncertainty intervals are not reported in the registry record.
What can a count tell us?
A count communicates how many participants were recorded as meeting an outcome definition. It does not, on its own, say how likely an event is in a broader population or how precisely that likelihood has been estimated. The arm sizes matter for interpreting counts: the posted groups include 5, 8, and 9 participants. Because the outcome is reported as a count of participants, it should not be confused with a count of all separate events. A participant with multiple events may still contribute a single participant to a participant-level outcome, depending on the endpoint definition.
The primary result lists 5 participants in the Vigil-alone arm, 6 in the gemcitabine-and-docetaxel arm, and 9 in the Vigil-combination arm. The registry does not report confidence intervals for these counts or a statistical comparison. A confidence interval, as a general concept, describes a range of values compatible with the observed data under a specified statistical method. It conveys uncertainty that a point estimate alone cannot show. Without intervals or another stated measure of uncertainty, readers should treat the posted counts as descriptive rather than as precise estimates of comparative effects.
What would a treatment comparison require?
Part 1 was intended to compare overall survival between Vigil and gemcitabine and docetaxel. A survival analysis generally uses information about how long participants are followed and when events occur, while accounting for participants whose event time is not observed during follow-up. General statistical context: censoring means that the exact event time is not known for some participants, even though information up to their last observation can be used. The registry does not report survival estimates, event times, censoring information, a survival-analysis method, or a comparative result. Consequently, no conclusion about the magnitude or precision of a survival difference can be drawn from the posted results.
Progression-free survival and overall survival are listed as secondary outcomes, but outcome estimates and analyses for them are not reported in the registry record. The record lists 0 analyses. It does not report a statistical analysis plan, model specification, hypothesis tests, or adjustment for baseline characteristics. Those details should not be inferred from the randomized designation or from the trial's stated objectives.
How should planning illustrations be understood?
General explanation: sample-size planning asks how many participants may be needed to detect a specified effect under chosen assumptions. The registry's planning description states a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation; it describes the target effect as a stated fraction of the observed effect. For time-to-event outcomes, the stated convention is a total number of events, while other outcomes use participants per arm. These are planning assumptions, not features of the observed results.
A planning illustration is not an observed treatment effect, and it is not observed power. Its interpretation depends on the effect used as a target and the assumptions chosen for the calculation. The registry does not report a protocol-based sample-size justification. The record also states that values are missing or not estimable for a two-arm comparison of the binary endpoint. Thus there is no basis in the registry record for reading a comparative sample-size result as a trial finding or as evidence that the study had a particular probability of detecting an effect.
Why can small groups produce uncertain estimates?
General statistical context: estimates based on small groups can be sensitive to the outcome status of individual participants. Confidence intervals, when available, help show this uncertainty; a point estimate without its uncertainty can appear more definitive than the data warrant. The posted arm counts are small, and the record provides no comparative confidence intervals or tests. It is therefore appropriate to describe the observed numbers without treating differences in counts as established treatment differences.
General explanation: over-dispersion occurs when observed variation is greater than a statistical model expects. It can make standard errors and confidence intervals too optimistic if the model does not account for the extra variation. The registry does not report a model for the posted adverse-event counts, and there is no reported analysis from which to assess over-dispersion. This concept is relevant as a general consideration in count-data analysis, not as a finding about this trial.
The adverse-event results also include serious-event counts, other-event counts, deaths, and selected serious-event terms. These categories are descriptive and are not substitutes for an explicitly defined comparative model. The record does not describe how event timing, recurrent events, exposure duration, or treatment attribution were incorporated into statistical analyses. Without those details, no modeled or adjusted interpretation should be added to the posted counts.
10. Exact Estimates for Each Arm
No two-arm comparison is shown for this trial, but each arm's result can still be summarized with exact interval estimates computed from the posted counts. The Clopper-Pearson interval is exact and conservative; the Wilson interval is a common closer-to-nominal alternative; the Bayesian credible interval uses a uniform Beta(1, 1) prior. For small groups the three can differ noticeably.
| Arm | Participants | Events | Observed % | Clopper-Pearson 95% CI | Wilson 95% CI | Bayesian 95% credible interval |
|---|---|---|---|---|---|---|
| Part 1: Vigil Alone | 5 | 5 | 100.0% | 47.8% to 100.0% | 56.6% to 100.0% | 54.1% to 99.6% |
| Part 1: Gemcitabine and Docetaxel | 8 | 6 | 75.0% | 34.9% to 96.8% | 40.9% to 92.9% | 40.0% to 92.5% |
| Part 2: Vigil in Combination With Temozolomide and | 9 | 9 | 100.0% | 66.4% to 100.0% | 70.1% to 100.0% | 69.2% to 99.7% |
R code and output
arm <- c("Part 1: Vigil Alone", "Part 1: Gemcitabine and Docetaxel", "Part 2: Vigil in Combination With Temozolomide and")
n <- c(5, 8, 9)
events <- c(5, 6, 9)
for (i in seq_along(n)) {
ci <- binom.test(events[i], n[i])$conf.int # Clopper-Pearson exact interval
cr <- qbeta(c(0.025, 0.975), 1 + events[i], 1 + n[i] - events[i]) # Bayesian, uniform Beta(1,1) prior
wi <- (function(x, n) { p <- x / n; z <- qnorm(0.975); d <- 1 + z^2 / n
c((p + z^2 / (2 * n) - z * sqrt(p * (1 - p) / n + z^2 / (4 * n^2))) / d, (p + z^2 / (2 * n) + z * sqrt(p * (1 - p) / n + z^2 / (4 * n^2))) / d) })(events[i], n[i])
cat(sprintf("ARM|%s|%d|%d|%.1f|%.1f|%.1f|%.1f|%.1f|%.1f|%.1f\n", arm[i], n[i], events[i], 100 * events[i] / n[i],
100 * ci[1], 100 * ci[2], 100 * wi[1], 100 * wi[2], 100 * cr[1], 100 * cr[2]))
}
ARM Part 1: Vigil Alone 5 5 100.0 47.8 100.0 56.6 100.0 54.1 99.6
ARM Part 1: Gemcitabine and Docetaxel 8 6 75.0 34.9 96.8 40.9 92.9 40.0 92.5
ARM Part 2: Vigil in Combination With Temozolomide and 9 9 100.0 66.4 100.0 70.1 100.0 69.2 99.711. Multiplicity, Missing Data and Analysis Populations
The registry lists 3 outcome measures, including the primary adverse-event count and the secondary outcomes of progression-free survival and overall survival. It does not report a multiplicity procedure, such as a prespecified strategy for controlling the chance of false-positive conclusions across several outcomes or comparisons. No hierarchy or testing sequence is reported in the registry record. This matters because interpretation of multiple statistical tests depends partly on whether and how they were planned and adjusted.
The record lists 0 analyses. Comparative estimates, confidence intervals, p-values, and analysis methods for the secondary survival outcomes are not reported in the registry record. The registry's stated Part 1 objective of comparing overall survival should therefore be distinguished from a posted statistical result: an objective does not itself provide an estimate or establish a treatment difference.
Missing-data methods and analysis populations are not reported in the registry record. The record does not specify how participants with missing assessments, incomplete follow-up, treatment discontinuation, or other departures from the intended treatment would be handled. It also does not identify an intention-to-treat, per-protocol, or other analysis population. These omissions limit the ability to determine which participants contributed to each outcome and how incomplete observations may affect interpretation.
General statistical context: multiplicity concerns arise when many outcomes or comparisons are tested, while missing-data assumptions concern how unobserved measurements are treated. Both can influence inferential conclusions. Without the corresponding analysis details, the posted counts are best read as descriptive registry results, not as evidence from a fully specified comparative analysis.
12. 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 |
|---|---|---|---|
| Part 1: Vigil Alone | 2 | 5 | 40.0% |
| Part 1: Gemicitabine and Docetaxel | 5 | 8 | 62.5% |
| Part 2: Vigil Plus Temozolomide and Irinotecan | 5 | 9 | 55.6% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Part 1: Vigil Alone | 4 of 4 |
| Part 1: Gemicitabine and Docetaxel | 8 of 8 |
| Part 2: Vigil Plus Temozolomide and Irinotecan | 9 of 9 |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 3% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Part 1: Vigil Alone | 2 of 5 |
| Part 1: Gemicitabine and Docetaxel | 5 of 8 |
| Part 2: Vigil Plus Temozolomide and Irinotecan | 9 of 9 |
Most frequent serious adverse event terms
| Event term | Organ system | Part 1: Vigil Alone | Part 1: Gemicitabine and Docetaxel | Part 2: Vigil Plus Temozolomide and Irinotecan |
|---|---|---|---|---|
| Disease progression | General disorders | 1 of 5 | 0 of 8 | 1 of 9 |
| Back pain | Musculoskeletal and connective tissue disorders | 1 of 5 | 0 of 8 | 1 of 9 |
| Headache | Nervous system disorders | 0 of 5 | 1 of 8 | 1 of 9 |
| Vomiting | Gastrointestinal disorders | 0 of 5 | 0 of 8 | 2 of 9 |
| Fever | General disorders | 0 of 5 | 1 of 8 | 1 of 9 |
| Pain | General disorders | 0 of 5 | 1 of 8 | 1 of 9 |
| Edema extremities | General disorders | 1 of 5 | 0 of 8 | 0 of 9 |
| Dyspnea | Respiratory, thoracic and mediastinal disorders | 1 of 5 | 0 of 8 | 0 of 9 |
| Brain mass | Nervous system disorders | 0 of 5 | 1 of 8 | 0 of 9 |
| Blisters | Skin and subcutaneous tissue disorders | 0 of 5 | 1 of 8 | 0 of 9 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Part 1: Vigil Alone | Part 1: Gemicitabine and Docetaxel | Part 2: Vigil Plus Temozolomide and Irinotecan |
|---|---|---|---|---|
| Anemia | Blood and lymphatic system disorders | 0 of 5 | 3 of 8 | 6 of 9 |
| Nausea | Gastrointestinal disorders | 1 of 5 | 3 of 8 | 5 of 9 |
| Fatigue | General disorders | 1 of 5 | 3 of 8 | 5 of 9 |
| Vomiting | Gastrointestinal disorders | 0 of 5 | 2 of 8 | 6 of 9 |
| Thrombocytopenia | Blood and lymphatic system disorders | 0 of 5 | 3 of 8 | 3 of 9 |
| Anorexia | Metabolism and nutrition disorders | 0 of 5 | 2 of 8 | 4 of 9 |
| Diarrhea | Gastrointestinal disorders | 0 of 5 | 0 of 8 | 5 of 9 |
| Fever | General disorders | 1 of 5 | 1 of 8 | 3 of 9 |
| Arthralgia | Musculoskeletal and connective tissue disorders | 1 of 5 | 1 of 8 | 3 of 9 |
| Neutropenia | Blood and lymphatic system disorders | 0 of 5 | 0 of 8 | 4 of 9 |
Serious events with the highest arm-level rate: vomiting (22.2%), edema extremities (20.0%), disease progression (20.0%), back pain (20.0%), dyspnea (20.0%).
The registry reports serious adverse events for 2 of 5 participants in the Vigil-alone arm, 5 of 8 in the gemcitabine-and-docetaxel arm, and 5 of 9 in the Vigil-combination arm. Other adverse events are reported for 2 of 5, 5 of 8, and 9 of 9 participants, respectively. Deaths are recorded for 4, 8, and 9 participants. These are separate descriptive categories in the adverse-event record; they should not be added together, because the record does not establish that they represent mutually exclusive groups.
The listed serious-event terms include vomiting (22.2%), edema extremities (20.0%), disease progression (20.0%), back pain (20.0%), dyspnea (20.0%), and brain mass (12.5%). The record does not report the arm-specific distribution for these terms or provide enough context to interpret the percentages as comparative risks. It also does not report event-level timing, duration, attribution, or whether participants could be counted under multiple terms.
General statistical context: serious-event counts describe observed safety reporting, not a causal comparison by themselves. The different arm sizes and the absence of comparative uncertainty estimates mean that raw counts should not be treated as evidence that one regimen is safer or less safe. The record's serious-event figures are descriptive and should be interpreted separately from the primary endpoint count.
13. What the Result Does, and Does Not, Mean
The registry results show participant counts for the listed primary adverse-event outcome and additional descriptive adverse-event categories. They establish what was recorded in each arm, including the counts of participants with serious adverse events, other adverse events, and deaths. They do not provide a statistical estimate of how outcomes differ between treatments.
The trial's two parts address different questions. Part 1 was designed to compare overall survival between Vigil and gemcitabine and docetaxel. Part 2 was intended to assess the safety profile of Vigil combined with temozolomide and irinotecan. The posted primary outcome is a safety count, and the registry does not report comparative survival results. The safety counts therefore cannot answer the Part 1 survival question.
General statistical context: a difference between observed counts can arise from treatment effects, random variation, differences in follow-up, or other features of data collection. A statistical comparison with an uncertainty estimate can help assess how compatible the data are with different possible effects. Such estimates are not reported in the registry record. It is not appropriate to treat the raw counts as proof of benefit, lack of benefit, or comparative harm.
The registry record also does not report the statistical analysis plan, missing-data methods, analysis populations, or details of how outcomes were analyzed. These limitations prevent a fuller assessment of precision and robustness. The results are best understood as registry-reported descriptive findings from a completed trial with 22 enrolled participants, not as a definitive comparative analysis.
14. Why This Trial Matters Statistically
This trial is statistically notable because its two parts have different purposes: a randomized comparison of overall survival in Part 1 and a safety assessment of a combination regimen in Part 2. The registry's listed primary outcome is a participant count for adverse events, while the survival outcomes are listed as secondary. Keeping these objectives and endpoints distinct is essential when interpreting the results.
The posted results illustrate the difference between descriptive data and inferential evidence. Counts show how many participants were recorded in each category, but without comparative estimates and uncertainty measures they do not establish the size or precision of a treatment effect. This distinction is especially important for small arm sizes, where results can be sensitive to individual outcomes.
The record also shows why registry results need to be read alongside information about analysis methods. A randomized designation and a stated objective do not specify how endpoints were analyzed, how missing observations were handled, or how multiple outcomes were addressed. Those methods are not reported in the registry record. As a result, readers can describe the posted counts but cannot reconstruct a complete inferential analysis of the trial.
More generally, the trial highlights that a study may contain separate safety and efficacy questions, and that results for one should not be used as a substitute for results on the other. Here, the reported adverse-event counts do not resolve the stated Part 1 survival question. Recognizing that boundary is a central part of statistical interpretation.
15. 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 registry record reports a completed trial with enrollment of 22 participants, distributed across three posted arms. The arm sizes are 5 for Vigil alone, 8 for gemcitabine and docetaxel, and 9 for Vigil with temozolomide and irinotecan. These groups are small, and the record does not report comparative uncertainty estimates. Consequently, the posted counts offer limited precision for judging differences between arms.
The record combines two parts with different objectives and treatment structures. Part 1 was intended to compare overall survival, while Part 2 was intended to assess the safety of a combination. The record does not report a comparator for Part 2. This means that the Part 2 findings are descriptive and do not support a direct randomized comparison within that part.
The listed primary outcome is an adverse-event count through 30 days of last treatment dosing, but survival outcomes are also listed as secondary measures. Comparative results for progression-free survival and overall survival are not reported in the registry record. Statistical methods, analysis populations, missing-data handling, and multiplicity procedures are also not reported in the registry record.
The Part 2 regimen description is incomplete in the registry record. The serious-event terms are listed with percentages but without arm-specific distributions or fuller event-level context. The record also does not report how adverse events were attributed to treatment or how recurrent events were analyzed. These limitations constrain interpretation of both safety and comparative efficacy. They do not negate the descriptive counts, but they make it important not to extend those counts into conclusions that the posted results do not support.
16. Related Tutorials
17. Related Statistical Calculators
18. Sources
- ClinicalTrials.gov: A Two-part Phase IIb Trial of Vigil (Bi-shRNAfurin and GMCSF Augmented Autologous Tumor Cell Immunotherapy) in Ewing's Sarcoma — NCT02511132.
- Registry record last updated 2022-12-22; status last verified 2022-12.
- Results first posted 2022-07-22.
- 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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19. Record Summary
NCT02511132 is a completed, randomized, unmasked, parallel Phase 2 trial titled as a two-part Phase IIb study of Vigil in Ewing's sarcoma. The registry reports enrollment of 22 participants and two distinct objectives: a Part 1 overall-survival comparison of Vigil with gemcitabine and docetaxel, and a Part 2 safety assessment of Vigil with temozolomide and irinotecan.
The posted primary outcome is the number of participants with adverse events determined by laboratory assessments and physical examinations through 30 days of last treatment dosing. The recorded counts are 5 for Vigil alone, 6 for gemcitabine and docetaxel, and 9 for the Part 2 combination. Serious adverse-event counts, other adverse-event counts, and deaths are also reported descriptively.
Comparative survival estimates, inferential analyses, confidence intervals, missing-data methods, analysis populations, and a multiplicity approach are not reported in the registry record. The posted results therefore describe recorded participant counts but do not establish comparative survival or safety effects.