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 Vaccine Therapy in Patients With Stage II, III, or IV Epithelial Ovarian, Fallopian Tube, or Peritoneal Cancer (NCT00112957), a na phase 2 study. The primary outcome is Percentage of Patients in Remission at 1 Year.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00112957 |
| Phase | Phase 2 |
| Condition | Fallopian Tube Cancer, Ovarian Cancer, Peritoneal Cavity Cancer |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 23 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Ludwig Institute for Cancer Research |
| Results posted | Yes |
2. Clinical Question
Population
Patients with epithelial ovarian, fallopian tube, or primary peritoneal carcinoma who had a complete response to standard therapy and whose tumors expressed NY-ESO-1 or LAGE-1 antigen. The registry describes the Full Analysis Set as patients who received at least one vaccine injection and met the cancer entry criterion. It also notes that one patient with breast cancer was treated under compassionate use.
Intervention
A vaccine regimen using recombinant vaccinia-NY-ESO-1 (rV-NY-ESO-1), followed by recombinant fowlpox-NY-ESO-1 (rF-NY-ESO-1). The registry describes an intradermal injection on Day 1 and later subcutaneous injections on scheduled days, or until treatment-related toxicity or disease progression.
Comparator
There was no separate comparison group: the study is described as single-group, with allocation listed as NA and masking as none. The record therefore does not describe a randomized, like-for-like control comparison.
Primary question
What percentage of patients was in remission at 12 months? The primary endpoint was defined as the crude proportion among patients with known status at that time, with a Kaplan-Meier cumulative estimate also calculated. The registry record does not report the numerical result for either estimate.
3. Trial Design
This was a Phase II, single-center, open-label, single-group study of rV-NY-ESO-1 and rF-NY-ESO-1. The registry lists enrollment as 23 and describes the study as completed. The recorded start was 2004-12 and primary completion was 2009-05. These dates describe the registry record and do not, by themselves, explain the time each participant was followed.
Eligible participants had epithelial ovarian, fallopian tube, or primary peritoneal carcinoma, had achieved a complete response to standard therapy, and had tumors expressing NY-ESO-1 or LAGE-1 antigen. The registry defines the Full Analysis Set as patients who received at least one injection with either vaccine and met the cancer entry criterion. It separately notes that one patient with breast cancer was treated under compassionate use. The record does not clarify how that patient was handled in each reported analysis.
The regimen began with one intradermal rV-NY-ESO-1 injection on Day 1. Subcutaneous rF-NY-ESO-1 injections were scheduled on Days 29, 57, 85, 113, 141, and 169, or until observation of treatment-related toxicity of at least grade 3 or disease progression. These are the schedule and stopping conditions recorded for the intervention; they do not establish that every participant received every scheduled injection.
The study had no masking and no listed allocation to separate groups. Consequently, its design addresses outcomes observed during a vaccine regimen in the enrolled study population, not a treatment contrast against a concurrent control group. The registry lists the sponsor as Ludwig Institute for Cancer Research. It does not report additional design details such as a separate comparator or a statistical analysis plan.
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 | rV- and rF-NY-ESO-1 |
|---|---|
| STARTED | 23 |
| COMPLETED | 4 |
| NOT COMPLETED | 19 |
Reasons for not completing this period:
| Reason | rV- and rF-NY-ESO-1 |
|---|---|
| Progressive disease | 18 |
| Physician Decision | 1 |
Analysis population for the primary outcome, as defined in the registry: The Full Analysis Set includes all patients who received at least 1 injection with rV-NY-ESO-1 or rF-NY-ESO-1 and met the entry criterion of having epithelial ovarian, fallopian tube, or primary peritoneal carcinoma. One patient with breast cancer was treated in the protocol under compassionate use and is excluded from the Full Analysis Set.
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: The Safety Analysis Set includes all patients who received at least 1 injection with rV-NY-ESO-1 or rF-NY-ESO-1.
| Characteristic | Unit | rV- and rF-NY-ESO-1 |
|---|---|---|
| Participants analysed | 23 | |
| Age, Continuous | years | 55 (37 to 89) |
| Sex: Female, Male: Female | Participants | 23 |
| Sex: Female, Male: Male | Participants | 0 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 0 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 23 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 |
| Race (NIH/OMB): Asian | Participants | 0 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 |
| Race (NIH/OMB): Black or African American | Participants | 0 |
| Race (NIH/OMB): White | Participants | 23 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Region of Enrollment: United States | participants | 23 |
| Karnofsky Performance Status: 70 | Participants | 1 |
| Karnofsky Performance Status: 80 | Participants | 3 |
| Karnofsky Performance Status: 90 | Participants | 3 |
| Karnofsky Performance Status: 100 | Participants | 16 |
| Body mass index | kg/m^2 | 29.5 (SD 4.7) |
6. Primary Endpoint
The registry lists the primary outcome as Percentage of Patients in Remission at 1 Year, measured over 12 months.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | 95% Confidence Interval |
| Unit | percentage of participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is the percentage of patients in remission at 12 months. It is a status-based measure at a specified follow-up time: the registry defines the crude proportion as the number in remission at one year divided by the number whose status was known at one year, expressed as a percentage. The record also says that a Kaplan-Meier cumulative estimate of the proportion in remission at that time was calculated.
These approaches address follow-up differently. The crude proportion uses only patients with known status at the specified time. The Kaplan-Meier approach is generally used to describe time-to-event experience while accounting for differing follow-up and censoring, subject to assumptions about how censoring relates to the outcome. This distinction matters when status at the target time is not known for every participant. The registry record does not report how many had known status at 12 months, the crude percentage, the Kaplan-Meier estimate, or either estimate's confidence interval.
A remission measure at a fixed time can make an outcome clinically interpretable as the share of the defined population still in remission at that milestone. It is not identical to the time until disease progression or another defined failure event. The study summary also identifies time to failure as an objective, but the numerical one-year remission result is not reported in the registry record. The endpoint definition alone therefore explains what was intended to be measured, not what value was observed.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry describes a study designed to evaluate maintenance of remission at 12 months after a vaccine regimen in patients who had already achieved a complete response to standard therapy. Its primary endpoint was a remission percentage, with both a crude calculation among patients with known one-year status and a Kaplan-Meier estimate specified. The registry record does not report the numerical result for either calculation. Accordingly, the endpoint definition cannot establish the observed remission proportion or how precisely it was estimated.
Enrollment is listed as 23. That figure does not establish how many participants met every analysis-population criterion, received each scheduled injection, had known status at one year, or contributed to any particular endpoint analysis. The record defines a Full Analysis Set based on receipt of at least one injection and meeting the cancer entry criterion, but does not report its size. It also notes a compassionate-use patient with breast cancer without explaining the handling of that patient in each analysis.
The study is single-group, open-label, and has no listed allocation to a comparator. Thus, even if the outcome estimates were available, the design would describe outcomes in the study group rather than isolate a difference attributable to the vaccines. Remission after prior standard therapy is also part of the population context: the study enrolled patients who had already achieved a complete response. Without a concurrent comparison group, outcomes cannot be separated from the course of disease or other care during follow-up.
The record names cellular and humoral immunity, time to failure, safety, and remission maintenance among the study objectives. It lists secondary outcomes concerning tumor response, cancer antigen-125 values, immune responses, and treatment-emergent adverse events. The registry record does not report numerical results for these measures in the registry record for this analysis. The listed objectives and outcomes should therefore be read as planned areas of assessment, not as evidence of a particular finding.
The safety summary records 4 participants with serious adverse events among 23 at risk, 23 with other adverse events among 23 at risk, and 0 deaths. Six serious-event terms are listed, each with a frequency of 4.3%. These summaries describe reported safety counts and terms; the registry entries available do not provide event-level timing, duration, attribution, or severity beyond the serious-event classification. They should not be used to infer whether an individual event was caused by the vaccine regimen.
Overall, the record gives the design, endpoint definition, and selected safety summaries, but not the primary remission estimate or its precision. The statistical interpretation must therefore remain focused on what was measured and how the study was structured, rather than on an unreported treatment effect.
9. Statistical Methods Explained
The primary endpoint combines a fixed follow-up milestone with a binary status: in remission or not in remission at 12 months. The registry calls the endpoint a percentage of participants and defines the crude calculation by dividing the number in remission at one year by the number with known status at one year. A percentage is a way to express the frequency of an outcome relative to a stated denominator. For interpretation, the numerator and denominator matter as much as the percentage, because they show how many observations contributed and how much information supports the estimate. Those counts and the numerical estimate are not reported in the registry record.
What does the confidence interval describe?
A confidence interval expresses statistical uncertainty around an estimate under the method used to construct it. It gives a range of values compatible with the data and method at the stated confidence level; it is not a guarantee that the true value lies in that particular interval. The endpoint specification names a 95% confidence interval, but its limits are not reported in the registry record. Without the estimate and interval, readers cannot assess the reported precision of the one-year remission result.
For a percentage, precision depends in part on the amount of information in the denominator and on how common the outcome is. A smaller effective sample generally permits less precise estimates than a larger one, although the exact interval also depends on the method and observed data. Enrollment alone cannot substitute for the endpoint denominator: the record's crude endpoint explicitly uses patients with known status at one year, while the size of that group is not stated.
Why are both crude and Kaplan-Meier estimates described?
The crude proportion uses participants whose status is known at the target time. It is straightforward to understand but does not directly use the timing of events or account for participants whose follow-up ends before the milestone with status unknown. The Kaplan-Meier method is a general time-to-event approach that updates the estimated event-free share over observed follow-up and accounts for censoring. In general, censoring means that the event time is not observed for a participant during the available follow-up. The method relies on assumptions, including that the censoring process is appropriately handled and is not informative in a way that biases the estimate.
The registry states that a Kaplan-Meier cumulative estimate of remission at one year was calculated, but does not report the estimate, its interval, the event definition in enough detail to assess implementation, or the handling of censoring. The method label alone is not enough to check whether its assumptions were plausible or whether the estimate was stable.
What does a single-group design permit?
A single-group study can describe outcomes observed in people receiving a regimen under the study conditions. It does not estimate a randomized contrast against a control group, because no separate comparison group is listed. General statistical interpretation distinguishes an outcome observed after treatment from a causal effect of treatment. A causal comparison requires a basis for evaluating what would have happened to a comparable group without the intervention; the registry design does not supply that concurrent comparison.
The study was open-label, meaning the record does not describe masking. In general, lack of masking can affect subjective outcome assessment, reporting, care decisions, or follow-up. Whether any of those issues affected this study cannot be determined from the registry record. The primary endpoint is a remission status, but the record does not provide enough detail to evaluate how status was assessed or adjudicated.
How should the listed safety statistics be read?
The registry lists serious adverse events for one group, with participants at risk and participants affected, and separately lists other adverse events and deaths. A participant count is not the same as an event count: one participant can experience more than one event. The serious-event terms each have a listed percentage, but the record does not state whether terms overlap within participants or provide the total number of events for each term. It is therefore not appropriate to add those percentages or treat the terms as mutually exclusive categories.
For general interpretation, adverse-event summaries describe events recorded during a study, not necessarily events caused by the intervention. Attribution requires clinical and methodological information beyond a count. The available record does not report event timing, duration, or attribution for the listed serious-event terms. Its safety summaries can be reported as recorded, but do not establish causation or characterize the full clinical context.
No analysis results are listed in the registry record. This means there is no numerical primary estimate, interval, or inferential statistic in the available record on which to base a conclusion about the magnitude or precision of the remission outcome. Statistical methods can clarify how an estimate would be interpreted, but they cannot recover an unreported estimate.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists 9 outcome measures: the primary remission measure and secondary measures covering time to failure, tumor response, cancer antigen-125 values, immune responses, delayed-type hypersensitivity reactions, and treatment-emergent adverse events. Considering multiple outcomes raises a general statistical issue: when many hypotheses are tested, the chance of at least one apparently notable result can increase if each is assessed separately. Methods such as prespecifying a primary outcome or adjusting for multiple comparisons can address that issue, but their use and details are not reported in the registry record. The record lists 0 analyses, so no outcome-level analysis results are available to evaluate.
The primary endpoint definition specifies a denominator of patients with known status at one year for the crude proportion. This makes missing status consequential: participants without known status at the milestone do not enter that crude denominator. The size of the known-status group, the amount and reasons for missing status, and any approach to missing data are not reported in the registry record. The Kaplan-Meier estimate is described as an additional calculation, but the registry entries do not report the censoring information or analytic details needed to assess it.
The Full Analysis Set is described as including patients who received at least one injection and met the cancer entry criterion. The registry does not state how many participants met that definition or identify the populations used for each endpoint. It also notes one patient with breast cancer treated under compassionate use, but does not explain whether or how that patient contributed to the Full Analysis Set or any particular outcome analysis. These details limit assessment of whether the endpoint population matches the intended study population.
Prespecified handling of missing data, definitions of analysis populations, and a clear plan for multiple outcomes help readers interpret estimates without selectively emphasizing results. Those details are not reported in the registry record. No assumptions about the trial's analysis plan, sample-size justification, or interim analyses can be made from the outcome definitions alone.
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 |
|---|---|---|---|
| rV- and rF-NY-ESO-1 | 4 | 23 | 17.4% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| rV- and rF-NY-ESO-1 | 0 of 23 |
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 |
|---|---|
| rV- and rF-NY-ESO-1 | 23 of 23 |
Most frequent serious adverse event terms
| Event term | Organ system | rV- and rF-NY-ESO-1 |
|---|---|---|
| Urinary tract infection | Infections and infestations | 1 of 23 |
| Pathological fracture | Musculoskeletal and connective tissue disorders | 1 of 23 |
| Small intestinal obstruction | Gastrointestinal disorders | 1 of 23 |
| Lung neoplasm | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | 1 of 23 |
| Abdominal pain | Gastrointestinal disorders | 1 of 23 |
| Cough | Respiratory, thoracic and mediastinal disorders | 1 of 23 |
| Mental status changes | Psychiatric disorders | 1 of 23 |
| Pneumonia | Infections and infestations | 1 of 23 |
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | 1 of 23 |
| Septic shock | Infections and infestations | 1 of 23 |
Most frequent non-serious adverse event terms
| Event term | Organ system | rV- and rF-NY-ESO-1 |
|---|---|---|
| Skin test positive | Investigations | 20 of 23 |
| Abdominal pain | Gastrointestinal disorders | 4 of 23 |
| Abdominal pain upper | Gastrointestinal disorders | 4 of 23 |
| Nausea | Gastrointestinal disorders | 4 of 23 |
| Nasopharyngitis | Infections and infestations | 4 of 23 |
| Back pain | Musculoskeletal and connective tissue disorders | 4 of 23 |
| Cough | Respiratory, thoracic and mediastinal disorders | 4 of 23 |
| Vomiting | Gastrointestinal disorders | 3 of 23 |
| Chest pain | General disorders | 3 of 23 |
| Pyrexia | General disorders | 3 of 23 |
Serious events with the highest arm-level rate: urinary tract infection (4.3%), pathological fracture (4.3%), small intestinal obstruction (4.3%), lung neoplasm (4.3%), abdominal pain (4.3%).
The safety summary lists 23 participants at risk for serious adverse events, with 4 affected. It lists 23 participants at risk for other adverse events, with 23 affected, and records 0 deaths. The serious-event terms listed are urinary tract infection, pathological fracture, small intestinal obstruction, lung neoplasm, abdominal pain, and cough; each is reported at 4.3%.
These are participant-level summaries as presented in the registry record. They do not establish how many separate events occurred, whether a participant experienced more than one term, or whether any event was judged related to the vaccine regimen. The listed percentages should not be added: the record does not say that the terms represent mutually exclusive participants. The registry entries available do not give timing, duration, event-level severity, or attribution for the terms.
Serious adverse events and other adverse events are distinct categories in the registry summary. The fact that 23 participants are listed as affected by other adverse events does not mean all experienced the same event or severity. Likewise, a listed serious event is not, by itself, evidence of causation. The counts describe the safety data as recorded; the registry record does not provide enough detail to make a causal assessment.
12. What the Result Does, and Does Not, Mean
The study addresses an important descriptive question: among patients with selected cancers who had achieved a complete response to standard therapy and whose tumors expressed specified antigens, what was remission status at 12 months during a vaccine study? The registry makes the endpoint definition clear enough to identify the intended quantity, including a crude percentage and a Kaplan-Meier estimate. But it does not report either numerical estimate or a confidence interval. As a result, the posted information does not show the observed one-year remission proportion or its precision.
The study's single-group design also defines what can be inferred. It can provide a description of outcomes and safety events recorded in the study group, but it does not offer a randomized control comparison. Any remission observed in this setting cannot, from this design alone, be attributed to the vaccine regimen rather than other influences or the course of illness after standard therapy. This is a limitation of the comparison structure, not a judgment about the value of the research question.
The registry lists 23 enrolled participants, but the endpoint denominator and the size of the defined Full Analysis Set are not reported. Enrollment should therefore not be mistaken for the number contributing to the one-year calculation. The registry also does not give the numbers or reasons for missing one-year status, the primary endpoint estimate, or the associated interval. Without these details, readers cannot gauge how much follow-up supports the endpoint.
The listed secondary outcomes and immune objectives describe additional topics evaluated by the study, but numerical results for them are not reported in the registry record. Nor does the registry record give enough statistical analysis detail to assess multiplicity, missing-data methods, or the handling of the compassionate-use patient. The safety summary can be described as recorded, but does not establish that a listed event was caused by vaccination.
Thus, the registry record supports a description of the trial's population, intervention schedule, design, endpoint definition, and selected safety summaries. It does not support a numerical conclusion about the primary remission endpoint, a comparative treatment effect, or a causal safety claim. The distinction between a clearly defined outcome and a reported outcome is central to interpreting this record.
13. Why This Trial Matters Statistically
Statistically, this study is useful for illustrating how the design and denominator shape the meaning of an outcome. A one-year remission percentage sounds simple, but the registry defines the crude measure among patients whose status was known at that milestone. The denominator therefore depends on follow-up completeness, not just the enrollment figure. The record also specifies a Kaplan-Meier estimate, underscoring that a fixed-time proportion and a time-to-event estimate can address incomplete follow-up in different ways.
The study also highlights the boundary between describing outcomes and estimating treatment effects. A single-group, open-label design can document what happened in the enrolled group, but it has no concurrent comparator for a controlled contrast. That distinction is especially important when participants enter after a complete response to standard therapy: the registry's endpoint concerns remission during later follow-up, while the design does not isolate the contribution of the vaccine regimen to that course.
Another statistical lesson is the value of complete reporting. The endpoint definition names a confidence interval and two estimation approaches, yet the numerical endpoint results are not reported in the registry record. Without estimates, intervals, denominators, and missing-status information, readers cannot assess precision or evaluate the relationship between the crude and Kaplan-Meier results. Outcome names and methods are not substitutes for results.
Finally, the registry lists several secondary outcomes and safety measures. Their presence illustrates why analysis populations, multiplicity decisions, and transparent reporting matter when a study examines multiple kinds of outcomes. Those details are not reported in the record available for this interpretation. The study therefore offers a clear example of how a registry can describe a research question and study structure while leaving key information needed for statistical evaluation unavailable.
14. Limitations
- The posted summary statistics are the only data these results rest on; patient-level data, full protocols and statistical analysis plans are not available.
- Recalculated results are simple, unadjusted calculations from posted summaries. They do not replace the sponsor's prespecified analysis.
The main trial-specific limitation is the absence of a concurrent comparison group. The registry describes a single-group study with no allocation to a comparator, so observed remission and safety outcomes cannot be interpreted as differences from an otherwise comparable control group. The open-label design also means masking was absent; the registry does not report whether outcome assessment was independently reviewed or whether any assessment procedures reduced potential observer influence.
The primary endpoint's crude calculation depends on known status at 12 months, but the registry record does not report the number with known status, the number missing, or reasons for missing status. It also does not report the crude estimate, Kaplan-Meier estimate, or confidence interval. The definition of the Full Analysis Set is stated, but its size and its relationship to the endpoint population are not reported. The handling of the noted compassionate-use patient with breast cancer is likewise unclear.
The record lists multiple secondary outcomes but does not report numerical results or a statistical analysis plan. It does not report multiplicity procedures, missing-data methods, interim analyses, or sample-size justification. These should be treated as unreported rather than inferred from the Phase II designation or endpoint description.
Safety reporting is limited in the available details: the record provides participant counts, selected serious-event terms, and their reported percentages, but does not give timing, duration, attribution, or event-level detail. The registry entries also do not supply enough information to assess how treatment exposure varied across participants. The scheduled regimen should not be taken as proof that every participant completed it. Together, these reporting limits prevent a detailed assessment of endpoint precision, missingness, or safety context.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Vaccine Therapy in Patients With Stage II, III, or IV Epithelial Ovarian, Fallopian Tube, or Peritoneal Cancer — NCT00112957.
- Registry record last updated 2023-10-04; status last verified 2023-10.
- Results first posted 2018-03-19.
- 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 NCT00112957 as a completed Phase II, single-center, open-label, single-group study sponsored by Ludwig Institute for Cancer Research. It lists enrollment as 23, a start of 2004-12, and primary completion in 2009-05. Participants with selected ovarian, fallopian tube, or primary peritoneal cancers, a complete response to standard therapy, and NY-ESO-1 or LAGE-1 expression received an rV-NY-ESO-1 and rF-NY-ESO-1 vaccine regimen.
The primary outcome was the percentage in remission at 12 months, defined by both a crude proportion among those with known status and a Kaplan-Meier estimate. The numerical remission results and confidence interval are not reported in the registry record. Safety summaries list 4 participants with serious adverse events, 23 with other adverse events, and 0 deaths. The record lists 9 outcome measures and 0 analyses.