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 EGEN-001 in Treating Patients With Persistent or Recurrent Ovarian Epithelial Cancer, Fallopian Tube Cancer, or Primary Peritoneal Cancer (NCT01118052), a na phase 2 study. The primary outcome is Adverse Events Deemed at Least Possibly Related to Treatment, as Assessed by NCI CTCAE Version 4.0.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01118052 |
| Phase | Phase 2 |
| Condition | Fallopian Tube Carcinoma, Primary Peritoneal Carcinoma, Recurrent Ovarian Carcinoma |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 22 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Gynecologic Oncology Group |
| Results posted | Yes |
2. Clinical Question
Population
Patients with persistent or recurrent ovarian epithelial cancer, fallopian tube cancer, or primary peritoneal cancer. The primary endpoint population is described as eligible and evaluable patients.
Intervention
Intraperitoneal EGEN-001, also described as an IL-12 DNA plasmid vector in a PEG-PEI-cholesterol lipopolymer. Patients were scheduled to receive treatment on days 1, 8, 15, and 22, with courses repeating every 4 weeks unless disease progressed or toxicity became unacceptable.
Comparator
There was no separate comparator group. This was a single-group trial, and allocation is recorded as NA. The treatment group therefore cannot be compared with a randomized control group in this trial.
Primary question
How many eligible and evaluable patients experienced adverse events deemed at least possibly related to treatment, with events assessed using NCI CTCAE Version 4.0? The primary endpoint is a count of participants affected, rather than a direct measure of tumor response or survival.
The study was a phase II evaluation in people whose cancer was persistent or had recurred. Its stated focus included side effects and how well EGEN-001 works, but the primary endpoint recorded in the registry concerns treatment-related adverse events. Overall survival and progression-free survival are listed as secondary outcomes. Their outcome results are not reported in the registry record, so those outcomes cannot be assessed from the information recorded.
The clinical question is consequently narrower than whether treatment changes the course of cancer. The posted safety counts describe participants with serious and other adverse events, but they do not give the overall count for the primary endpoint of events deemed at least possibly related to treatment. The record also does not establish how outcomes would compare with what would have happened without treatment.
3. Trial Design
This was a phase II, single-group trial of EGEN-001, with enrollment recorded as 22 and status recorded as completed. The trial started on 2010-11-01, and the primary completion date is recorded as 2016-07-16. The sponsor was the Gynecologic Oncology Group. Allocation is recorded as NA and masking as none; no random assignment or blinded treatment assessment is described in the registry record.
Patients in the treatment group received intraperitoneal EGEN-001 on days 1, 8, 15, and 22. Courses were scheduled to repeat every 4 weeks in the absence of disease progression or unacceptable toxicity. Laboratory biomarker analysis is also listed as an intervention, but further details about its role are not reported in the registry record.
A single-group design can describe what happened among people who received the study treatment. It does not, by itself, distinguish treatment effects from the natural course of disease, other care, or differences in who entered or remained in the study. There is no like-for-like control comparison in this trial, and the record explicitly cautions that the arms are not a randomized comparison.
The primary endpoint is described as adverse events deemed at least possibly related to treatment, assessed with NCI CTCAE Version 4.0. The stated observation window includes treatment and a period after stopping treatment, described as up to 5 years after stopping study treatment. The wording of the timeframe is not fully clear about the duration for every event, and no clarification is reported in the registry record. Eligible and evaluable patients are the stated population for the primary endpoint, but the criteria for being evaluable are not reported in the registry record.
Overall survival and progression-free survival are listed as secondary outcomes. The registry record does not report outcome analyses for these endpoints. It also does not report a statistical analysis plan, the intended handling of patients who stopped treatment early, or whether any interim analyses were planned.
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 (EGEN-001) |
|---|---|
| STARTED | 22 |
| COMPLETED | 20 |
| NOT COMPLETED | 2 |
Reasons for not completing this period:
| Reason | Treatment (EGEN-001) |
|---|---|
| Never treated | 2 |
Analysis population for the primary outcome, as defined in the registry: Eligible and treated patients
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: Eligible and treated patients
| Characteristic | Unit | Treatment (EGEN-001) |
|---|---|---|
| Participants analysed | 20 | |
| Age, Customized: < 40 years | Participants | 0 |
| Age, Customized: 40 - 49 years | Participants | 1 |
| Age, Customized: 50 - 59 years | Participants | 12 |
| Age, Customized: 60 - 69 years | Participants | 5 |
| Age, Customized: 70 - 79 years | Participants | 2 |
| Sex: Female, Male: Female | Participants | 20 |
| Sex: Female, Male: Male | Participants | 0 |
6. Primary Endpoint
The registry lists the primary outcome as Adverse Events Deemed at Least Possibly Related to Treatment, as Assessed by NCI CTCAE Version 4.0, measured over All Adverse Events (AEs) deemed at least possibly related to study treatmetn occurring during treatment and up to 30 days after stopping the study treatment. for up to 5 years after stopping study treatment.
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint counts participants with adverse events deemed at least possibly related to study treatment. The events were assessed using NCI CTCAE Version 4.0, and the record says that adverse events are listed by event and grade, with the number of affected participants reported. The endpoint population is described as eligible and evaluable patients.
Counting affected participants provides a direct descriptive summary of how many people experienced an event meeting the specified relatedness threshold. It can help characterize treatment tolerability, while the event grades provide information about severity. General statistical context: a participant count is different from a count of event episodes. A person may experience more than one event, so participant counts should not be interpreted as total event counts unless the reporting makes that distinction clear.
The registry record does not report the primary endpoint's overall participant count or a breakdown by event and grade. The serious and other adverse-event counts recorded elsewhere are not identified as the complete primary endpoint result, nor are they stated to include only events deemed at least possibly related. They should therefore not be substituted for the primary endpoint.
The endpoint timeframe covers adverse events during treatment and after stopping treatment, with the record describing follow-up up to 5 years after stopping study treatment. The exact interpretation of that timeframe is not reported in the registry record. The endpoint is a safety measure; it is not a direct measure of tumor response, progression, or survival.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record identifies a single treatment group and an enrollment of 22. The adverse-event fields report 20 participants at risk for serious adverse events, of whom 9 were affected, and 20 at risk for other adverse events, of whom 20 were affected. These are descriptive safety counts. The record does not explain how the at-risk groups relate to the enrolled population, so the difference between the enrollment figure and the safety denominators cannot be interpreted from the registry record.
For serious adverse events, the record lists Small Intestinal Obstruction and Creatinine Increased at 15.0% each. Anemia, Colonic Obstruction, Vomiting, and Ileal Fistula are each listed at 5.0%. These percentages describe the listed serious-event terms; the record does not provide participant counts alongside each term in this summary. They should not be added together: people may have experienced more than one event, and the registry record does not state that the categories are mutually exclusive.
The overall primary endpoint is not reported as an analyzed result. Although the endpoint concerns adverse events deemed at least possibly related to treatment, the posted counts for serious and other events are not explicitly identified as that endpoint. The record does not report the overall number of participants with at least one event meeting the endpoint definition, a grade-specific endpoint summary, or an uncertainty interval. As a result, the primary endpoint cannot be interpreted as a complete estimate of treatment-related adverse-event frequency from these fields alone.
Overall survival and progression-free survival are listed as secondary outcomes, but no analyses are recorded. There are no posted estimates, event counts, follow-up summaries, or uncertainty intervals for those outcomes in the registry record. The listed outcomes therefore do not support conclusions about survival or disease progression.
Because there is no randomized comparator, the safety counts describe the treatment group rather than a difference between treatments. They cannot establish how these events compare with another treatment or with no study treatment. General statistical context: estimates from a single group can be useful for describing observed experience, but a comparison is needed to separate treatment-associated differences from background risks and other influences. No such comparative estimate is reported for this trial.
The registry records 22 enrolled participants but does not explain which participants contributed to each outcome denominator, how missing outcome data were handled, or whether the primary analysis included everyone enrolled. Those omissions limit interpretation of both the observed safety counts and the absence of reported survival analyses. The record also provides no confidence intervals or p-values for the adverse-event results. The available information is therefore descriptive and incomplete for assessing the stated primary and secondary outcomes.
9. Statistical Methods Explained
The design is recorded as single-group, with allocation listed as NA and masking listed as none. Those design features matter when interpreting estimates: there is no randomized control arm against which to compare the treatment group, and the record does not describe blinded assessment. The trial can describe observations among participants receiving EGEN-001, but it cannot estimate a randomized between-group treatment contrast.
What does a participant count represent?
The outcome type is recorded as binary, with the meaning described as participants with an event. In general, a binary outcome records whether each participant experienced the defined event or did not. A count of affected participants summarizes the number meeting that definition in the stated population. It does not necessarily equal the number of event episodes, because one participant can experience multiple events.
The primary endpoint is a count of participants with adverse events deemed at least possibly related to treatment. The registry specifies NCI CTCAE Version 4.0 and says events are listed by event and grade. The record does not post the overall endpoint result or its event-by-grade breakdown. The separate safety fields report serious and other adverse-event counts, but do not state that those fields represent the primary endpoint definition. Keeping those summaries distinct avoids treating a relatedness-based endpoint as if it were interchangeable with a seriousness-based count.
Why does the single-group design affect interpretation?
In general, a randomized comparison estimates how outcomes differ between assigned groups, subject to the trial's analysis and data quality. Here, there is no such comparison group. A count observed in the treatment group does not reveal what the same participants' outcomes would have been under a different treatment or without study treatment. Differences in disease status, other care, and who remained under observation can also shape descriptive results. The registry record does not report an adjustment strategy for these issues.
Masking is recorded as none. In general, knowledge of treatment assignment can influence reporting, assessment, or decisions about whether an event is related to treatment. CTCAE grading supplies a framework for classifying adverse-event severity, but the registry record does not describe procedures for independent review or blinded attribution. No such procedures should be assumed.
What can uncertainty measures add?
In general, a confidence interval gives a range of values compatible with the observed data under a statistical model and conveys how precisely an estimate is determined. A p-value, when tied to a defined hypothesis and analysis, describes how unusual data at least as extreme would be under that hypothesis and model. Neither measure alone establishes clinical importance or causality. The registry record does not report confidence intervals or p-values for the primary endpoint, serious adverse events, or other adverse events. Without them, precision cannot be assessed from the posted summary counts.
General statistical context: counts from a small study can be sensitive to the experience of individual participants, and estimates may have substantial uncertainty. This is not a calculated uncertainty statement for this trial; the record does not give the inferential estimates needed to quantify it. The enrolled number and the at-risk counts should not be treated as interchangeable denominators without a documented analysis population.
How should planning illustrations be read?
The registry's stated sample-size assumptions describe 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. The effect to detect is described as a stated fraction of the observed effect. Those assumptions describe hypothetical two-arm planning, whereas this trial is single-group. Any resulting sample-size illustration is not the trial's observed power and does not establish that the trial was designed around those assumptions. Numerical sample-size illustration results and a trial-specific sample-size justification are not reported in the registry record.
General statistical context: over-dispersion matters when observed variability exceeds what a model assumes. A model that underestimates variability can make uncertainty appear smaller than it is. The registry record does not describe a dispersion model or an analysis model for these outcomes, so there is no basis to say whether over-dispersion was assessed or relevant to an actual fitted model.
The record indicates 5 outcome measures and 0 analyses. It lists overall survival and progression-free survival as secondary outcomes, but their estimates and analysis details are not reported. The statistical plan, including any model specifications, adjustment for baseline factors, treatment of repeated events, and interim analysis procedures, is not reported in the registry record. These absences prevent reconstruction of an inferential analysis from the posted information.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists 5 outcome measures and records 0 analyses. Overall survival and progression-free survival are identified as secondary outcomes; the analysis results for those outcomes are not reported in the registry record. The primary endpoint is also not accompanied by a posted analysis result. Although multiple outcomes can raise questions about multiplicity, the record does not report a statistical plan describing which outcomes were confirmatory, how hypotheses were prioritized, or whether any adjustment for multiple testing was planned. No multiplicity procedure should be inferred.
The stated primary endpoint population is eligible and evaluable patients. The criteria for eligibility and evaluability in the analysis, and the number included in that analysis, are not reported in the registry record. Enrollment is recorded as 22, while the safety fields give at-risk counts of 20. The relationship between these populations is not explained. The record does not say whether the difference reflects eligibility, evaluability, follow-up, missing observations, or another reason.
Missing-data handling is not reported in the registry record. There is no description of how participants without complete follow-up were treated, whether missing outcome information was imputed, or how censoring was handled for the listed survival outcomes. General statistical context: missingness can affect estimates when the likelihood or pattern of missing data is related to participant characteristics or outcomes. Without details on follow-up and analysis populations, the direction or magnitude of any such effect cannot be assessed.
The record also does not report an intention-to-treat population, a per-protocol population, or a sensitivity analysis. These omissions leave uncertainty about which participants and observations would contribute to each outcome estimate, if analyses were conducted.
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 (EGEN-001) | 9 | 20 | 45.0% |
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 (EGEN-001) | 20 of 20 |
Most frequent serious adverse event terms
| Event term | Organ system | Treatment (EGEN-001) |
|---|---|---|
| Small Intestinal Obstruction | Gastrointestinal disorders | 3 of 20 |
| Creatinine Increased | Investigations | 3 of 20 |
| Anemia | Blood and lymphatic system disorders | 1 of 20 |
| Colonic Obstruction | Gastrointestinal disorders | 1 of 20 |
| Vomiting | Gastrointestinal disorders | 1 of 20 |
| Ileal Fistula | Gastrointestinal disorders | 1 of 20 |
| Death Nos | General disorders | 1 of 20 |
| Fracture | Injury, poisoning and procedural complications | 1 of 20 |
| White Blood Cell Decreased | Investigations | 1 of 20 |
| Hypoglycemia | Metabolism and nutrition disorders | 1 of 20 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Treatment (EGEN-001) |
|---|---|---|
| Anemia | Blood and lymphatic system disorders | 15 of 20 |
| Nausea | Gastrointestinal disorders | 15 of 20 |
| Fatigue | General disorders | 13 of 20 |
| Constipation | Gastrointestinal disorders | 9 of 20 |
| Diarrhea | Gastrointestinal disorders | 9 of 20 |
| Abdominal Pain | Gastrointestinal disorders | 9 of 20 |
| Vomiting | Gastrointestinal disorders | 8 of 20 |
| Platelet Count Decreased | Investigations | 7 of 20 |
| Pain | General disorders | 6 of 20 |
| Creatinine Increased | Investigations | 6 of 20 |
Serious events with the highest arm-level rate: small intestinal obstruction (15.0%), creatinine increased (15.0%), anemia (5.0%), colonic obstruction (5.0%), vomiting (5.0%).
The adverse-event record reports 9 affected participants among 20 at risk for serious adverse events, and 20 affected among 20 at risk for other adverse events. These are separate categories in the registry record. They are not the primary endpoint result: the primary endpoint is defined by adverse events deemed at least possibly related to treatment, whereas these counts are presented under serious and other adverse-event fields.
The serious-event terms listed include Small Intestinal Obstruction and Creatinine Increased at 15.0% each, and Anemia, Colonic Obstruction, Vomiting, and Ileal Fistula at 5.0% each. These percentages should be read as the registry's reported values for the listed terms. They are not a complete account of all safety outcomes, and the record does not give participant counts for each term in this summary. Because participants may have more than one event, the term percentages should not be summed.
The safety denominators do not match the enrollment figure, and the record does not explain the difference. It also does not state whether each adverse-event category includes all events or only events judged related to treatment. These counts describe what was recorded in the treated group; without a randomized comparator, they do not establish a relative safety difference. The primary endpoint's overall result and grade-specific counts are not reported in the registry record.
12. What the Result Does, and Does Not, Mean
The posted information documents a completed phase II, single-group evaluation of intraperitoneal EGEN-001 in persistent or recurrent ovarian epithelial, fallopian tube, or primary peritoneal cancer. It records enrollment of 22 and provides some descriptive safety information, including serious and other adverse-event counts and selected serious-event terms. It does not provide a reported analysis of the primary endpoint as defined in the registry.
The safety information can be read as observations in the treatment group, not as proof that EGEN-001 caused every event or that the event frequency differs from another treatment. The primary endpoint's wording includes an attribution threshold of at least possibly related, but the posted serious and other adverse-event counts are not explicitly presented as the endpoint's relatedness-based result. The record therefore does not permit a complete assessment of the primary outcome.
Overall survival and progression-free survival are listed as secondary outcomes, but their estimates and analyses are not reported in the registry record. No conclusion about survival or disease progression can be drawn from the information recorded. The study's stated interest in how well treatment works does not substitute for posted outcome results.
General statistical context: a single-group study can describe outcomes observed during treatment and follow-up. It cannot show what those outcomes would have been for the same participants under an alternative. A control group, defined analysis methods, and sufficiently complete follow-up would be needed to support comparative conclusions. These details are not available in the registry record.
Accordingly, the record supports a limited descriptive reading of safety observations. It does not establish comparative benefit, comparative harm, or a causal treatment effect. The lack of reported endpoint analyses and uncertainty estimates also means that the precision of the posted safety summaries cannot be judged from the registry record.
13. Why This Trial Matters Statistically
This trial illustrates why the design and the endpoint definition must be read together. The primary outcome concerns participants with adverse events deemed at least possibly related to treatment, while the posted safety fields separately describe serious and other adverse events. Treating these summaries as interchangeable would change the question being answered. A registry entry can contain safety counts without supplying the complete result for its stated primary endpoint.
The single-group design is also central to statistical interpretation. Descriptive counts can characterize the treatment group, but there is no randomized comparator to provide a reference for relative risk or treatment difference. The record itself notes that there is no like-for-like control comparison. Even a carefully measured event count in the treated group cannot determine whether the observed experience differs from what would occur under another treatment.
The at-risk counts and enrollment figure highlight the importance of defining analysis populations. The registry records enrollment as 22 and safety denominators as 20, but does not explain the relationship. Without that explanation, readers cannot determine who contributed to each safety summary or assess how exclusions or missing observations might affect the results.
Finally, the record lists survival outcomes but does not report analyses for them, and it does not provide the primary endpoint analysis. This distinction between registered outcomes and reported results is statistically important: naming an outcome does not establish its observed result, precision, or interpretation. The entry is useful for identifying the intended clinical question and available descriptive safety information, while leaving major inferential questions unanswered.
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.
Several trial-specific limitations follow from the registry record. The single-group design has no randomized comparator, so it cannot estimate a treatment contrast. Allocation is recorded as NA and masking as none. The record does not describe blinded outcome assessment, independent event review, or a statistical adjustment strategy.
Enrollment is recorded as 22, while the adverse-event fields use at-risk counts of 20. The record does not explain who was included in each safety population or why those denominators differ from enrollment. The primary endpoint population is described as eligible and evaluable patients, but its size and evaluability criteria are not reported in the registry record.
The overall result for the primary endpoint is not reported. Serious and other adverse-event counts are present, but their relationship to the endpoint's requirement that events be deemed at least possibly related is not specified. The record also does not provide the endpoint's event-by-grade results, confidence intervals, or p-values. Serious-event terms and percentages alone do not resolve these gaps.
Overall survival and progression-free survival are listed, but no analyses are recorded. Follow-up completeness, censoring, missing-data handling, and analysis populations are not reported in the registry record. The treatment timeframe is described as including events during treatment and up to 5 years after stopping study treatment, but the exact application of that period is unclear from the wording.
The registry record does not report a trial-specific sample-size justification or statistical analysis plan. The stated planning assumptions concern a hypothetical two-arm comparison, which differs from the single-group design. These limitations restrict both the completeness of the safety interpretation and any assessment of efficacy.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: EGEN-001 in Treating Patients With Persistent or Recurrent Ovarian Epithelial Cancer, Fallopian Tube Cancer, or Primary Peritoneal Cancer — NCT01118052.
- Registry record last updated 2018-01-11; status last verified 2017-08.
- Results first posted 2017-11-30.
- 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 record describes a completed phase II, single-group study of intraperitoneal EGEN-001 for persistent or recurrent ovarian epithelial, fallopian tube, or primary peritoneal cancer. Enrollment is recorded as 22. Treatment was scheduled on days 1, 8, 15, and 22, with courses repeating every 4 weeks unless disease progressed or toxicity became unacceptable.
The primary endpoint counts participants with adverse events deemed at least possibly related to treatment, assessed using NCI CTCAE Version 4.0. The record does not report the overall primary endpoint analysis. It reports serious adverse events in 9 of 20 at risk and other adverse events in 20 of 20 at risk; selected serious-event terms are also listed with percentages.
Overall survival and progression-free survival are listed as secondary outcomes, but analyses are not reported. With no randomized comparator and no posted endpoint analyses or uncertainty estimates, the available results support only a limited descriptive account of safety observations.